Ignite Creation Date:
2025-12-25 @ 3:43 AM
Ignite Modification Date:
2026-06-26 @ 5:09 AM
Study NCT ID:
NCT03362502
Status:
TERMINATED
Last Update Posted:
2025-09-15
First Post:
2017-11-29
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ClinicalTrials.gov_Inquiries@pfizer.com', 'phone': '1-800-718-1021', 'title': 'Pfizer ClinicalTrials.gov Call Center', 'organization': 'Pfizer Inc.'}, 'certainAgreement': {'otherDetails': 'Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to the end of 1-year follow-up (approximately 360 days)', 'description': 'The same event may appear as both an AE and an SAE. However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as non-serious in another participant, or 1 participant may have experienced both a serious and non-serious event during the study. Total number at risk below refers to the number of participants evaluable for SAEs or AEs.', 'eventGroups': [{'id': 'EG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.', 'otherNumAtRisk': 16, 'deathsNumAtRisk': 16, 'otherNumAffected': 16, 'seriousNumAtRisk': 16, 'deathsNumAffected': 0, 'seriousNumAffected': 3}, {'id': 'EG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 3, 'seriousNumAtRisk': 3, 'deathsNumAffected': 1, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Haemolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Leukocytosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Lymphopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Cardiomyopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Androgen deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Lacrimation increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Abdominal pain lower', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Inguinal hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 13}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Infusion site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Liver injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Molluscum contagiosum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Parainfluenzae virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Tooth abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Joint dislocation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Joint injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Post procedural contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Amylase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Complement factor C4 decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Cystatin C increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Glutamate dehydrogenase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Hepatic enzyme increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Lipase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Troponin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 6}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Hyperuricaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Osteopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 7}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Abnormal behaviour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Affect lability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Mood altered', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Chronic kidney disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Glycosuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Ketonuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Ecchymosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}], 'seriousEvents': [{'term': 'Haemolytic uraemic syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Cardiogenic shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 3, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 3, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA v25.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'TEAEs', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Severe AEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a treatment-emergent AE (TEAE). A serious AE (SAE) was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With All-Causality TEAEs by System Organ Class (SOC) and Preferred Term (PT) for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Anaemia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolysis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolytic uraemic syndrome', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Leukocytosis', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Lymphopenia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Thrombocytopenia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, PT: Cardiogenic shock', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, PT: Cardiomyopathy', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: ENDOCRINE DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: ENDOCRINE DISORDERS, PT: Androgen deficiency', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: EYE DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: EYE DISORDERS, PT: Lacrimation increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, Total', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Abdominal pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Abdominal pain lower', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Abdominal pain upper', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Constipation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Diarrhoea', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Gastrooesophageal reflux disease', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Inguinal hernia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Nausea', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Toothache', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Vomiting', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Chest pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Fatigue', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Infusion site reaction', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Mass', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Pyrexia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: HEPATOBILIARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: HEPATOBILIARY DISORDERS, PT: Liver injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Cellulitis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Conjunctivitis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Influenza', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Molluscum contagiosum', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Nasopharyngitis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Parainfluenzae virus infection', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Sinusitis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Tooth abscess', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Urinary tract infection', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Viral upper respiratory tract infection', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Contusion', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Fall', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Joint dislocation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Joint injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Ligament sprain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Post procedural contusion', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Procedural pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INJURY, POISONING AND PROCEDURAL COMPLICATIONS, PT: Skin abrasion', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Amylase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Complement factor C4 decreased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Cystatin C increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Glutamate dehydrogenase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Hepatic enzyme increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Lipase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Troponin increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Decreased appetite', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Dehydration', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Hyperuricaemia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, PT: Arthralgia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, PT: Back pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, PT: Myalgia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, PT: Osteopenia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: MUSCULOSKELETAL AND CONNECTIVE TISSUE DISORDERS, PT: Pain in extremity', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Dizziness', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Headache', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Lethargy', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Migraine', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Abnormal behaviour', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Affect lability', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Anxiety', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Irritability', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Mood altered', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: PSYCHIATRIC DISORDERS, PT: Suicidal ideation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Acute kidney injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Chronic kidney disease', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Glycosuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Haematuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Ketonuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Proteinuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS, PT: Cough', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS, PT: Epistaxis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS, PT: Oropharyngeal pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RESPIRATORY, THORACIC AND MEDIASTINAL DISORDERS, PT: Rhinorrhoea', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: SKIN AND SUBCUTANEOUS TISSUE DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: SKIN AND SUBCUTANEOUS TISSUE DISORDERS, PT: Ecchymosis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: SKIN AND SUBCUTANEOUS TISSUE DISORDERS, PT: Rash', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: SKIN AND SUBCUTANEOUS TISSUE DISORDERS, PT: Urticaria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: VASCULAR DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: VASCULAR DISORDERS, PT: Hypertension', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to Medical Dictionary for Regulatory Activities (MedDRA) version 25.0 SOC and PT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Related TEAEs for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'TEAEs', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Severe AEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Related TEAEs by SOC and PT for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Anaemia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolysis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolytic uraemic syndrome', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Leukocytosis', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Lymphopenia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Thrombocytopenia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, PT: Cardiogenic shock', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Abdominal pain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Abdominal pain upper', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Diarrhoea', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Nausea', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GASTROINTESTINAL DISORDERS, PT: Vomiting', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Fatigue', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Infusion site reaction', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: GENERAL DISORDERS AND ADMINISTRATION SITE CONDITIONS, PT: Pyrexia', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: HEPATOBILIARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: HEPATOBILIARY DISORDERS, PT: Liver injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Amylase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Complement factor C4 decreased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Cystatin C increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Glutamate dehydrogenase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Hepatic enzyme increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Lipase increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INVESTIGATIONS, PT: Troponin increased', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Decreased appetite', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Dehydration', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Hyperuricaemia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Dizziness', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Headache', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'SOC: NERVOUS SYSTEM DISORDERS, PT: Lethargy', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Acute kidney injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Chronic kidney disease', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Glycosuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Haematuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Ketonuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Proteinuria', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: VASCULAR DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: VASCULAR DISORDERS, PT: Hypertension', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs were determined by the investigator.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With All-Causality SAEs by SOC and PT for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolytic uraemic syndrome', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Thrombocytopenia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, PT: Cardiogenic shock', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: INFECTIONS AND INFESTATIONS, PT: Urinary tract infection', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Dehydration', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Acute kidney injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Related SAEs by SOC and PT for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Haemolytic uraemic syndrome', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: BLOOD AND LYMPHATIC SYSTEM DISORDERS, PT: Thrombocytopenia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: CARDIAC DISORDERS, PT: Cardiogenic shock', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: METABOLISM AND NUTRITION DISORDERS, PT: Dehydration', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, Total', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'SOC: RENAL AND URINARY DISORDERS, PT: Acute kidney injury', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Hematology', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'Hemoglobin (g/dL) <0.8x Lower Limit of Normal (LLN)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Hematocrit (%)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Erythrocytes (10^6/mm3)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Platelets (10^3/mm3)<0.5x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Erythrocyte Cell Morphology (No Unit)>0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Leukocytes (10^3/mm3)>1.5x Upper Limit of Normal (ULN)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Lymphocytes (10^3/mm3)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Lymphocytes (10^3/mm3)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Lymphocytes Atypical (10^3/mm3)>0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Neutrophils (10^3/mm3)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Neutrophils (10^3/mm3)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Neutrophils Band Form (10^3/mm3)>0.27', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Basophils (10^3/mm3)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Eosinophils (10^3/mm3)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Monocytes (10^3/mm3)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Activated Partial Thromboplastin Time (sec)>1.1x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Prothrombin Time (sec)>1.1x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Neutrophils -TOTAL COUNT (10^3/mm3)<1.35', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Neutrophils -TOTAL COUNT (10^3/mm3)>8.15', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Myelocytes (10^3/mm3)>0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Haptoglobin (mg/dL)<30', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Haptoglobin (mg/dL)>200', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following hematologic parameters were analyzed for laboratory examination: hemoglobin, hematocrit, red blood cell (RBC) count, platelet count, white blood cell count, total neutrophils, absolute neutrophils (ANC), eosinophils, monocytes, basophils, lymphocytes, red blood cell indices, and haptoglobin. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Clinical Chemistry', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'Bilirubin (mg/dL)>1.5x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Direct Bilirubin (mg/dL)>1.5x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Aspartate Aminotransferase (U/L)>3.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Alanine Aminotransferase (U/L)>3.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Gamma Glutamyl Transferase (U/L)>3.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Protein (g/dL)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Albumin (g/dL)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Urea Nitrogen (mg/dL)>1.3x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Creatinine (mg/dL)>1.3x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Urate (mg/dL)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Sodium (mEq/L)<0.95x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Potassium (mEq/L)<0.9x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Calcium (mg/dL)<0.9x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Phosphate (mg/dL)<0.8x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Bicarbonate (mEq/L)<0.9x LLN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Bicarbonate (mEq/L)>1.1x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Glucose (mg/dL)>1.5x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Creatine Kinase (U/L)>2.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Triacylglycerol Lipase (U/L)>1.5x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'C-Reactive Protein High Sens (mg/dL)>1.1x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Cystatin C (mg/L)>1.2x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Creatine Kinase MB (ug/L)>2.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Glutamate Dehydrogenase (U/L)>1.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '10', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Glutamate Dehydrogenase (U/L)>2.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Glutamate Dehydrogenase (U/L)>3.0x ULN', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}]}, {'title': 'Ferritin (ng/mL)<23.9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Ferritin (ng/mL)>336.2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following parameters for clinical chemistry were analyzed for laboratory examination: blood urea nitrogen (BUN) and creatinine, glucose, calcium, sodium, potassium, chloride, total CO2 (bicarbonate), aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (direct and indirect bilirubin), alkaline phosphatase, uric acid, albumin, total protein, serum phosphorus, cystatin C, creatine kinase, creatine kinase myocardial b fraction (CK-MB), amylase, lipase, gamma-glutamyl transferase (GGT), C-reactive protein (CRP), and cardiac troponin I. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Urinalysis', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'URINE Ketones (No Unit)>=1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'URINE Protein (Scalar)>=1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'URINE Hemoglobin (No Unit)>=1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'URINE Nitrite (No Unit)>=1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'URINE Erythrocytes (/high-powered field [HPF])>=20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '6', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'URINE Leukocytes (/HPF)>=20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Granular Casts (/low-power field [LPF])>1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Hyaline Casts (/LPF)>1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Uric Acid Crystals (/LPF)>=0.5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Calcium Oxalate Crystals (/LPF)>=0.5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Amorphous Crystals (/LPF)>=0.5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Urine Microscopic Exam (Scalar)>=1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following parameters for urinalysis were analyzed for laboratory examination: pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, and microscopy. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline on Left Ventricular Ejection Fraction as Measured by Cardiac Magnetic Resonance Imaging (MRI) for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.7', 'spread': '3.2', 'groupId': 'OG000'}, {'value': '-1.0', 'spread': '4.3', 'groupId': 'OG001'}, {'value': '-3.1', 'spread': '1.8', 'groupId': 'OG002'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, 1 year post dose of PF-06839926 (Day 360)', 'description': 'Cardiac MRI was performed after thigh and upper limb MRI or on separate days within the required visit window. Change in LVEF is presented as percentages, and was calculated as \\[(stroke volume) / (end-diastolic volume)\\]\\*100%. Baseline is defined as the last pre-dose measurement. Change from baseline on Day 360 (ie, 1 year post dose) was reported for this outcome measure.', 'unitOfMeasure': 'Percentage', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population and had reportable data for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Electrocardiograms (ECGs) Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Triplicate ECG was performed after the participant had rested quietly for at least 10 minutes in a supine position. The pre-defined categorical criteria include PR interval aggregate (msec): value\\>=300, baseline\\>200 and percent change\\>=25%, baseline\\<=200 and percent change\\>=50%; QRS duration aggregate (msec): value\\>=140, percent change\\>=50%; QTcF interval aggregate (msec): 450\\<=value\\<480, 480\\<=value\\<500, value\\>=500, 30\\<=change\\<60, change\\>=60. Only the category(ies) with at least 1 participant meeting the pre-defined criterion is/are reported for this outcome measure. Baseline for ECG was defined as the measurement before the Day 1 study drug administration.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Positive Responses on Columbia-Suicide Severity Rating Scale (C-SSRS) According to the Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'Baseline: Lifetime <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Lifetime <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Lifetime <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Lifetime <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Lifetime <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Since Last Visit <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Since Last Visit <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Since Last Visit <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Since Last Visit <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Baseline: Since Last Visit <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 7: Since Last Visit <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 7: Since Last Visit <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 7: Since Last Visit <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 7: Since Last Visit <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 7: Since Last Visit <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 14: Since Last Visit <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 14: Since Last Visit <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 14: Since Last Visit <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 14: Since Last Visit <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 14: Since Last Visit <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '13', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 180: Since Last Visit <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 180: Since Last Visit <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 180: Since Last Visit <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 180: Since Last Visit <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Day 180: Since Last Visit <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '14', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 360: Since Last Visit <1> Completed Suicide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 360: Since Last Visit <2> Suicide Attempt', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 360: Since Last Visit <3> Preparatory Acts towards Imminent Suicidal Behavior', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 360: Since Last Visit <4> Suicidal Ideation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Day 360: Since Last Visit <7> Self-injurious behavior, no suicidal intent', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Day 7, Day 14, Day 180, and Day 360', 'description': 'C-SSRS is a participant rated questionnaire to assess whether participant experienced the following: completed suicide(1), suicide attempt(2)("Yes" on "actual attempt"), preparatory acts toward imminent suicidal behavior(3)("Yes" on "preparatory acts or behavior"), suicidal ideation(4)("Yes" on "wish to be dead","non-specific active suicidal thoughts","active suicidal ideation with methods without intent to act or some intent to act, without specific plan or with specific plan and intent), any suicidal behavior or ideation, self-injurious behavior(7)("Yes" on "Has subject engaged in non-suicidal self-injurious behavior"). Yes/No responses are mapped to C-CASA categories. C-SSRS was conducted with the participant\'s care giver/legal guardian on the participant\'s behalf throughout the study for participants\\<=12 years of age at screening. Baseline is defined as the last predose measurement. Number of participants with responses of "Yes" is reported for this outcome measure.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Vital Signs Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'OG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'OG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'classes': [{'title': 'Respiratory Rate: value<14 breaths/min for aged 6-12 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Respiratory Rate: value>22 breaths/min for aged 6-12 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}]}, {'title': 'Respiratory Rate: value<12 breaths/min for aged 13-18 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Respiratory Rate: value>20 breaths/min for aged 13-18 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '3', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG002'}]}]}, {'title': 'Supine Diastolic Blood Pressure: value<50 mmHg', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Supine Diastolic Blood Pressure: increase>=20 mmHg', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Supine Diastolic Blood Pressure: decrease>=20 mmHg', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Supine Heart Rate: value<40 beats per minute (bpm)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Supine Heart Rate: value>120 bpm', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}]}]}, {'title': 'Supine Systolic Blood Pressure: value<70 mmHg + 2*age for aged 2-10 years', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Supine Systolic Blood Pressure: value<90 mmHg for aged 11 years and older', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Supine Systolic Blood Pressure: increase from baseline>=30 mmHg', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}, {'title': 'Supine Systolic Blood Pressure: decrease from baseline>=30 mmHg', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}]}], 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Vital signs consisted of blood pressure, respiratory rate, and heart rate, was obtained with these measurements at the following timepoints: within 30 minutes before and approximately 30 minutes, 1, 2, 4, 8, and 24 hours after the start of the infusion, and on Days 4, 7, 10, 14, 30, 90, 180, and 360. Baseline was defined as the last pre-dose recording. Number of participants who had at least 1 vital sign measurement at the specified timepoints meeting the pre-defined criteria from baseline up to the end of 1-year follow-up is reported for this outcome measure.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Includes all enrolled participants who received PF-06939926. Participants were analyzed according to the intervention they actually received. Number of participants analyzed represents the total number of participants in each treatment group in the analysis population for this outcome measure. Number analyzed represents the number of participants who had reportable data for this outcome measure.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'FG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'FG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '16'}, {'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'comment': 'Completed indicates participants completed the 1-year follow-up as of data cutoff on 30 Sep 2022.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '16'}, {'groupId': 'FG002', 'numSubjects': '2'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study reached its primary completion date (PCD) on 28 Mar 2022. The data cutoff for PCD was on 30 Sep 2022. As of 30 Sep 2022, a total of 22 participants, including 19 ambulatory participants and 3 non-ambulatory participants, were enrolled and assigned to treatment.', 'preAssignmentDetails': 'One participant was enrolled but not dosed due to a pause in enrollment. The participant was considered as an early termination due to screening window running out. The participant was subsequently re-screened and assigned to study intervention with another participant ID. Therefore, a total of 22 unique participants were enrolled as of 30 Sep 2022.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '22', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'PF-06939926: Low Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single intravenous (IV) infusion of PF-06939926 1E+14 vector genomes per kilogram bodyweight (vg/kg) on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years.'}, {'id': 'BG001', 'title': 'PF-06939926: High Dose, Ambulatory Cohort', 'description': 'Ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per transgene (TG)-based titer method or 3E+14 vg/kg per the inverted terminal repeats (ITR)-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'BG002', 'title': 'PF-06939926: High Dose, Non-Ambulatory Cohort', 'description': 'Non-ambulatory participants received a single IV infusion of PF-06939926 2E+14 vg/kg per TG-based titer method or 3E+14 vg/kg per the ITR-based titer method on Day 1, followed by at least 24 hours of in-patient monitoring, a 2-week follow-up visit, 1-year follow-up, and long-term follow-up for 4 years. PF-06939926 at 3E+14 vg/kg per the ITR-based titer method is approximately equivalent to 2E+14 vg/kg per the TG-titer method.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'categories': [{'title': '6-<7 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': '7-<8 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}, {'title': '8-<9 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}, {'title': '9-<10 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}]}, {'title': '10-<11 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': '11-<12 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': '>=12 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '4', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Customized', 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000', 'lowerLimit': '7', 'upperLimit': '9'}, {'value': '8', 'groupId': 'BG001', 'lowerLimit': '6', 'upperLimit': '12'}, {'value': '14', 'groupId': 'BG002', 'lowerLimit': '13', 'upperLimit': '16'}, {'value': '8', 'groupId': 'BG003', 'lowerLimit': '6', 'upperLimit': '16'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'Years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '22', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'White', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '17', 'groupId': 'BG003'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}]}, {'title': 'Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Mutation Type', 'classes': [{'categories': [{'title': 'Deletion', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'Exon Deletion', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '10', 'groupId': 'BG003'}]}, {'title': 'Exon Duplication', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '3', 'groupId': 'BG003'}]}, {'title': 'Insertion', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}, {'title': 'Point Mutation', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '6', 'groupId': 'BG003'}]}, {'title': 'Cryptic Splice Donor Site', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Confirmation by genetic testing of the diagnosis of duchenne muscular dystrophy (DMD) was reported from an appropriate regulated laboratory using a clinically validated genetic test.', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Includes all enrolled participants who received PF-06939926 as of the PCD data cutoff (30 Sep 2022). Participants were analyzed according to the intervention they actually received.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-03-07', 'size': 32552784, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-03-27T15:56', 'hasProtocol': True}, {'date': '2022-10-27', 'size': 5113303, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-03-27T15:56', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'whyStopped': 'All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2018-01-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2025-07-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-08-25', 'studyFirstSubmitDate': '2017-11-29', 'resultsFirstSubmitDate': '2023-03-27', 'studyFirstSubmitQcDate': '2017-11-29', 'lastUpdatePostDateStruct': {'date': '2025-09-15', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2024-05-17', 'studyFirstPostDateStruct': {'date': '2017-12-05', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-09-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a treatment-emergent AE (TEAE). A serious AE (SAE) was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities.'}, {'measure': 'Number of Participants With All-Causality TEAEs by System Organ Class (SOC) and Preferred Term (PT) for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to Medical Dictionary for Regulatory Activities (MedDRA) version 25.0 SOC and PT.'}, {'measure': 'Number of Participants With Treatment-Related TEAEs for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Severe AE=an event that prevents normal everyday activities. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator.'}, {'measure': 'Number of Participants With Treatment-Related TEAEs by SOC and PT for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs were determined by the investigator.'}, {'measure': 'Number of Participants With All-Causality SAEs by SOC and PT for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT.'}, {'measure': 'Number of Participants With Treatment-Related SAEs by SOC and PT for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a TEAE. An SAE was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in congenital anomaly/birth defect, or was considered to be an important medical event. Treatment-related AE was any untoward medical occurrence attributed to study treatment in a participant who received study treatment. Treatment-related AEs and SAEs were determined by the investigator. For this outcome measure, TEAEs were reported according to MedDRA version 25.0 SOC and PT.'}, {'measure': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Hematology', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following hematologic parameters were analyzed for laboratory examination: hemoglobin, hematocrit, red blood cell (RBC) count, platelet count, white blood cell count, total neutrophils, absolute neutrophils (ANC), eosinophils, monocytes, basophils, lymphocytes, red blood cell indices, and haptoglobin. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.'}, {'measure': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Clinical Chemistry', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following parameters for clinical chemistry were analyzed for laboratory examination: blood urea nitrogen (BUN) and creatinine, glucose, calcium, sodium, potassium, chloride, total CO2 (bicarbonate), aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (direct and indirect bilirubin), alkaline phosphatase, uric acid, albumin, total protein, serum phosphorus, cystatin C, creatine kinase, creatine kinase myocardial b fraction (CK-MB), amylase, lipase, gamma-glutamyl transferase (GGT), C-reactive protein (CRP), and cardiac troponin I. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.'}, {'measure': 'Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Urinalysis', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Following parameters for urinalysis were analyzed for laboratory examination: pH, glucose, protein, blood, ketones, nitrites, leukocyte esterase, and microscopy. Laboratory abnormalities with occurrence in at least 1 participant are reported for this outcome measure. Baseline was defined as the last pre-dose measurement.'}, {'measure': 'Change From Baseline on Left Ventricular Ejection Fraction as Measured by Cardiac Magnetic Resonance Imaging (MRI) for 1-Year Follow-Up', 'timeFrame': 'Baseline, 1 year post dose of PF-06839926 (Day 360)', 'description': 'Cardiac MRI was performed after thigh and upper limb MRI or on separate days within the required visit window. Change in LVEF is presented as percentages, and was calculated as \\[(stroke volume) / (end-diastolic volume)\\]\\*100%. Baseline is defined as the last pre-dose measurement. Change from baseline on Day 360 (ie, 1 year post dose) was reported for this outcome measure.'}, {'measure': 'Number of Participants With Electrocardiograms (ECGs) Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Triplicate ECG was performed after the participant had rested quietly for at least 10 minutes in a supine position. The pre-defined categorical criteria include PR interval aggregate (msec): value\\>=300, baseline\\>200 and percent change\\>=25%, baseline\\<=200 and percent change\\>=50%; QRS duration aggregate (msec): value\\>=140, percent change\\>=50%; QTcF interval aggregate (msec): 450\\<=value\\<480, 480\\<=value\\<500, value\\>=500, 30\\<=change\\<60, change\\>=60. Only the category(ies) with at least 1 participant meeting the pre-defined criterion is/are reported for this outcome measure. Baseline for ECG was defined as the measurement before the Day 1 study drug administration.'}, {'measure': 'Number of Participants With Positive Responses on Columbia-Suicide Severity Rating Scale (C-SSRS) According to the Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories', 'timeFrame': 'Baseline, Day 7, Day 14, Day 180, and Day 360', 'description': 'C-SSRS is a participant rated questionnaire to assess whether participant experienced the following: completed suicide(1), suicide attempt(2)("Yes" on "actual attempt"), preparatory acts toward imminent suicidal behavior(3)("Yes" on "preparatory acts or behavior"), suicidal ideation(4)("Yes" on "wish to be dead","non-specific active suicidal thoughts","active suicidal ideation with methods without intent to act or some intent to act, without specific plan or with specific plan and intent), any suicidal behavior or ideation, self-injurious behavior(7)("Yes" on "Has subject engaged in non-suicidal self-injurious behavior"). Yes/No responses are mapped to C-CASA categories. C-SSRS was conducted with the participant\'s care giver/legal guardian on the participant\'s behalf throughout the study for participants\\<=12 years of age at screening. Baseline is defined as the last predose measurement. Number of participants with responses of "Yes" is reported for this outcome measure.'}, {'measure': 'Number of Participants With Vital Signs Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up', 'timeFrame': 'Baseline up to 1 year post dose of PF-06939926', 'description': 'Vital signs consisted of blood pressure, respiratory rate, and heart rate, was obtained with these measurements at the following timepoints: within 30 minutes before and approximately 30 minutes, 1, 2, 4, 8, and 24 hours after the start of the infusion, and on Days 4, 7, 10, 14, 30, 90, 180, and 360. Baseline was defined as the last pre-dose recording. Number of participants who had at least 1 vital sign measurement at the specified timepoints meeting the pre-defined criteria from baseline up to the end of 1-year follow-up is reported for this outcome measure.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['gene therapy', 'mini-dystrophin', 'AAV', 'fordadistrogene movaparvovec'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'references': [{'pmid': '40753271', 'type': 'DERIVED', 'citation': 'Walsh J, Palandra J, Duriga N, Beidler D, McIntosh A, Binks M, Neubert H. Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography-tandem mass spectrometry after gene therapy for DMD. Gene Ther. 2025 Aug 2. doi: 10.1038/s41434-025-00554-5. Online ahead of print.'}, {'pmid': '40583275', 'type': 'DERIVED', 'citation': 'Byrne BJ, Butterfield RJ, Shieh PB, Smith EC, Licht C, Binks M, Casinghino S, Delnomdedieu M, Ravindra KC, McDonnell T, Ryan K, Schulz M, Shen Q, Shi H, Sirivelu MP, Vaidya VS, Whiteley L, Levy DI. Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy. Mol Ther. 2025 Sep 3;33(9):4226-4238. doi: 10.1016/j.ymthe.2025.06.032. Epub 2025 Jun 28.'}, {'pmid': '40583273', 'type': 'DERIVED', 'citation': 'Sherlock SP, Levy DI, McIntosh A, Shieh PB, Smith EC, McDonnell TG, Ryan KA, Delnomdedieu M, Binks M, Lal AK, Butterfield RJ. Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial. Mol Ther. 2025 Sep 3;33(9):4216-4225. doi: 10.1016/j.ymthe.2025.06.031. Epub 2025 Jun 28.'}, {'pmid': '40579547', 'type': 'DERIVED', 'citation': 'Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC. AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial. Nat Med. 2025 Aug;31(8):2712-2721. doi: 10.1038/s41591-025-03750-3. Epub 2025 Jun 27.'}], 'seeAlsoLinks': [{'url': 'https://pmiform.com/clinical-trial-info-request?StudyID=C3391001', 'label': 'To obtain contact information for a study center near you, click here.'}]}, 'descriptionModule': {'briefSummary': 'This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function.\n\nA total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '4 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Age as follows, based on ambulatory status:\n\n * FOR AMBULATORY PARTICIPANTS, defined as the ability to walk at least 10 meters unassisted: Between 4 and 12 years, inclusive,\n * FOR NON-AMBULATORY PARTICIPANTS, defined as the inability to walk at least 10 meters unassisted: No age restrictions so long as loss of ambulation occurs prior to the subject's 17th birthday;\n* Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing;\n* Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry;\n* Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications to these procedures;\n* Ability to tolerate muscle biopsies under anesthesia with no contraindications to these procedures;\n* Body weights as follows, based on ambulatory status:\n\n * FOR AMBULATORY PARTICIPANTS: Between 15 kg and 50 kg,\n * FOR NON-AMBULATORY PARTICIPANTS: Less than 75 kg, but which may be managed or adjusted to a lower limit, especially to ensure participant safety;\n* Functional performance as follows, based on ambulatory status:\n\n * FOR AMBULATORY PARTICIPANTS: Ability to rise from floor within seven (7) seconds,\n * FOR NON-AMBULATORY PARTICIPANTS: Percent predicted forced vital capacity greater than 40% as part of pulmonary function tests, as well as adequate upper limb function.\n\nExclusion Criteria:\n\n* Receipt of live attenuated vaccination within 3 months prior to receiving PF-06939926 or exposure to an influenza (or other inactivated) vaccination or systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926;\n* Prior exposure to any gene therapy agent, including exon-skipping agents;\n* Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer;\n* Neutralizing antibodies (NAb) against adeno-associated virus, serotype 9 (AAV9);\n* Compromised cardiac function as indicated by left ventricular ejection fraction on cardiac MRI, as follows, based on ambulatory status:\n\n * FOR AMBULATORY PARTICIPANTS: Less than 55%,\n * FOR NON-AMBULATORY PARTICIPANTS: Less than 35%;\n* Inadequate hepatic or renal function or risk factors for autoimmune disease on screening laboratory assessments.\n* The following genetic abnormalities in the dystrophin gene as confirmed by the investigator based on the review of the DMD genetic testing:\n\n 1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR\n 2. A deletion that affects both exon 29 and exon 30.\n\nSirolimus Cohort\n\nInclusion Criteria\n\n* \\> 8 years of age Exclusion Criteria\n* Hypersensitivity to sirolimus or intolerance to soy, including a history of angioedema\n* Concomitant use with strong CYP3A4/P-gp inducers or inhibitors"}, 'identificationModule': {'nctId': 'NCT03362502', 'briefTitle': 'A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY', 'orgStudyIdInfo': {'id': 'C3391001'}, 'secondaryIdInfos': [{'id': 'NCT03362502', 'type': 'REGISTRY', 'domain': 'ClinicalTrials.gov'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PF-06939926', 'interventionNames': ['Genetic: PF-06939926']}], 'interventions': [{'name': 'PF-06939926', 'type': 'GENETIC', 'description': 'Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a muscle-specific promoter.\n\nSubjects will receive a single intravenous infusion of one of 2 dose levels.', 'armGroupLabels': ['PF-06939926']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'MRI Research Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Reed Neurological Research Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Ronald Reagan UCLA Medical Center (Investigational Drug Section)', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Ronald Reagan UCLA Medical Center - Interventional Radiology', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Ronald Reagan UCLA Medical Center Drug Information Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA (David Geffen School of Medicine)', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "UCLA Children's Heart Center", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "UCLA Mattel Children's Hospital", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA Outpatient Surgery Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Neurology', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': "Duke University Medical Center, Lenox Baker Children's Hospital", 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Biospecimen Repository & Processing Core - BPRC', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Cardiovascular Magnetic Resonance Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': "Duke Children's Hospital & Health Center", 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Hospital Investigational Drug Services (IDS) Pharmacy', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'CCTS Clinical Research Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Imaging and Neurosciences Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospital & Clinics Investigational Drug Services', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospital', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "Primary Children's Hospital", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Clinical Neurosciences Center', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}], 'overallOfficials': [{'name': 'Pfizer CT.gov Call Center', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pfizer'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': "Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\\_trials/trial\\_data\\_and\\_results/data\\_requests."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}