Ignite Creation Date:
2025-12-24 @ 3:54 PM
Ignite Modification Date:
2026-01-07 @ 8:19 PM
Study NCT ID:
NCT02484092
Status:
COMPLETED
Last Update Posted:
2020-06-16
First Post:
2015-06-18
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Gene Therapy Study for Hemophilia B
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Canada']}, 'conditionBrowseModule': {'meshes': [{'id': 'D002836', 'term': 'Hemophilia B'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}]}}, '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}, 'limitationsAndCaveats': {'description': '15 participants were treated at the first dose planned for this dose escalation study. Requirements for dose escalation were not met as per protocol and the study completed once all participants completed 52 weeks of follow-up at the initial dose.'}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to Week 52', 'eventGroups': [{'id': 'EG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg) IV Infusion', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.', 'otherNumAtRisk': 15, 'deathsNumAtRisk': 15, 'otherNumAffected': 14, 'seriousNumAtRisk': 15, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Microcytic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Normocytic anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Palpitations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Conjunctivitis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Abdominal pain lower', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Aphthous ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Food poisoning', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Catheter site bruise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Face oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Muscle injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Muscle strain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Transaminases increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Calcification of muscle', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Costochondritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Exostosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Joint range of motion decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Tendonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Hypoaesthesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Sinus headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Sinus congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Upper-airway cough syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}, {'term': 'Rash macular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 21.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': "Physical examination included examination of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The examination assessed the participants for any potential changes in general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. Findings were considered to be clinically significant based on investigator's decision.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Clinically Significant Change From Baseline in Vital Signs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': "Vital signs (temperature, respiratory rate, pulse rate, height, weight, systolic and diastolic blood pressure) were obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Clinical significance of vital signs was determined at the investigator's discretion.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': 'Following parameters were analyzed for laboratory examination: hematology (neutrophils, lymphocytes, monocytes, eosinophils, basophils, red blood cell \\[RBC\\] count, hemoglobin, hematocrit, platelet count); liver function (albumin, total bilirubin, total protein, direct bilirubin, indirect bilirubin, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, alkaline phosphatase, GGT); Lipid panel (HDL, VLDL, triglycerides, total cholesterol); clinical chemistry (sodium, potassium, chloride, bicarbonate, glucose, phosphate, serum creatinine, BUN); urinalysis (specific gravity, pH, glucose, protein, blood, ketones; coagulation, immunology, etc. Investigators determined which laboratory abnormalities were reported as treatment-emergent adverse events (TEAEs).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'title': 'Drug-related TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Drug-related Serious TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': "An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and AEs. An AE was regarded as TEAE if the start date was on or after the infusion of SPK-9001 but before participant's last visit on study (or the date of withdrawal/the date of being lost to follow-up). Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': 'Peripheral blood mononuclear cells (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT) to assess cellular immune responses to AAV capsid and to FIX were presented. The ELISPOT is a type of assay that focuses on quantitatively measuring the frequency of cytokine secretion for a single cell. The positive ELISPOT results suggested a T-cell reaction to capsid protein.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': 'Based on non-clinical studies in non-human primates (NHPs), it was not predicted that vector-derived FIX:C activity levels \\>150% of normal would be achieved in this study. However, thrombin antithrombin (TAT) levels as thrombotic potential were to be measured if vector derived FIX:C activity levels \\>150% of normal were achieved in any participant during the study. Blood samples for TAT at Day 0 visit (prior to FIX protein product infusion) were used to establish baseline value.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With FIX Inhibitor', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 52', 'description': 'FIX inhibitors were measured using the Bethesda assay from the central and local laboratory. The Bethesda assay measures the amount of factor (FIX) inactivated when the plasma from the patient is incubated with an external source of factor for 2 hours at 37ºC. Inhibitor levels are quantified in Bethesda units (BU). An inhibitor titer of ≥ 0.6 BU/ml is to be taken as clinically significant.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received the infusion of SPK-9001.'}, {'type': 'PRIMARY', 'title': 'Incremental Recovery of FIX Product', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'title': 'Day 0', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0100', 'spread': '0.00242', 'groupId': 'OG000'}]}]}, {'title': 'Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.0162', 'spread': '0.01351', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Day 0 and Week 52', 'description': 'Incremental recovery was determined as the peak factor level recorded within the first 3 hours after infusion and was reported as (IU/ml)/(IU/kg), using the formula:(\\[Activity IU/mL peak post infusion\\] - \\[Activity IU/mL pre-infusion\\]) / (IU/kg infused).', 'unitOfMeasure': '[IU/ml]/[IU/kg]', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who had received 100 IU/kg of FIX protein product infusion and completed the blood sample collection within the first 3 hours post infusion for FIX protein product enabling determination of FIX incremental recovery.'}, {'type': 'SECONDARY', 'title': 'FIX:C Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'title': 'Steady-State Level', 'categories': [{'measurements': [{'value': '22.9', 'spread': '9.89', 'groupId': 'OG000'}]}]}, {'title': 'Peak Activity', 'categories': [{'measurements': [{'value': '29.1', 'spread': '11.63', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 52', 'description': 'All samples collected from participants for plasma FIX activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FIX activity levels. The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity levels were characterized by post-treatment population mean. Dose escalation and dose level expansion strategies were employed in the study based on vector-derived FIX activity levels as well as any immune responses against AAV capsid. Steady-state levels were based on 2 separate vector-derived FIX:C activity level measurements (at least 2 weeks apart) starting from Week 8-12 with adequate washout.', 'unitOfMeasure': 'Percentage of Normal', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who had received SPK-9001 and had collected vector-derived FIX:C activity levels enabling acceptable determination of the peak and steady-state derived activity level.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in FIX:C Antigen Level at Steady State', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg)', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'classes': [{'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.4', 'spread': '19.26', 'groupId': 'OG000'}]}]}, {'title': 'Week 14', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.7', 'spread': '23.11', 'groupId': 'OG000'}]}]}, {'title': 'Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.7', 'spread': '19.79', 'groupId': 'OG000'}]}]}, {'title': 'Week 18', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.8', 'spread': '21.81', 'groupId': 'OG000'}]}]}, {'title': 'Week 22', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.6', 'spread': '22.06', 'groupId': 'OG000'}]}]}, {'title': 'Week 26', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.6', 'spread': '20.64', 'groupId': 'OG000'}]}]}, {'title': 'Week 32', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-7.1', 'spread': '20.48', 'groupId': 'OG000'}]}]}, {'title': 'Week 42', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.9', 'spread': '19.87', 'groupId': 'OG000'}]}]}, {'title': 'Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.9', 'spread': '19.70', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 12 up to Week 52', 'description': 'The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity antigen levels were characterized by post-treatment population mean.', 'unitOfMeasure': 'Percentage of Normal', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who had received SPK-9001 and had collected vector-derived FIX:C activity levels enabling acceptable determination of the peak and steady-state derived activity level.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg) IV Infusion', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'A total of 22 participants were screened, 15 participants were assigned to treatment and completed study. All 15 participants received the lowest dose in the study (5 x 10\\^11 vg/kg). No participants were assigned to the 2 higher dose arms. Results presented here are from the lowest dose level.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'SPK-9001 (5 x 10^11 vg/kg) IV Infusion', 'description': 'Participants were infused with 100 IU/kg of their usual FIX protein product over 10 minutes at Day 0 visit. Following the bolus infusion of the usual FIX protein product, the participant was infused with SPK-9001 (5 x 10\\^11 vg/kg) for approximately 60 minutes via infusion pump.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '38.6', 'spread': '14.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White or Caucasian', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': 'Black or African American', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Multiple', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Baseline analysis population included all participants who received at least 1 dose of study treatment.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2019-11-01', 'size': 869712, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-03-31T14:07', 'hasProtocol': False}, {'date': '2019-06-14', 'size': 2725009, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-04-01T16:15', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-11-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-06', 'completionDateStruct': {'date': '2019-04-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-06-03', 'studyFirstSubmitDate': '2015-06-18', 'resultsFirstSubmitDate': '2020-04-04', 'studyFirstSubmitQcDate': '2015-06-24', 'lastUpdatePostDateStruct': {'date': '2020-06-16', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-05-05', 'studyFirstPostDateStruct': {'date': '2015-06-29', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-05-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-04-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings', 'timeFrame': 'Baseline up to Week 52', 'description': "Physical examination included examination of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The examination assessed the participants for any potential changes in general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. Findings were considered to be clinically significant based on investigator's decision."}, {'measure': 'Number of Participants With Clinically Significant Change From Baseline in Vital Signs', 'timeFrame': 'Baseline up to Week 52', 'description': "Vital signs (temperature, respiratory rate, pulse rate, height, weight, systolic and diastolic blood pressure) were obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Clinical significance of vital signs was determined at the investigator's discretion."}, {'measure': 'Number of Participants With Clinical Laboratory Abnormalities Reported as TEAE', 'timeFrame': 'Baseline up to Week 52', 'description': 'Following parameters were analyzed for laboratory examination: hematology (neutrophils, lymphocytes, monocytes, eosinophils, basophils, red blood cell \\[RBC\\] count, hemoglobin, hematocrit, platelet count); liver function (albumin, total bilirubin, total protein, direct bilirubin, indirect bilirubin, aspartate aminotransferase, alanine aminotransferase, lactate dehydrogenase, alkaline phosphatase, GGT); Lipid panel (HDL, VLDL, triglycerides, total cholesterol); clinical chemistry (sodium, potassium, chloride, bicarbonate, glucose, phosphate, serum creatinine, BUN); urinalysis (specific gravity, pH, glucose, protein, blood, ketones; coagulation, immunology, etc. Investigators determined which laboratory abnormalities were reported as treatment-emergent adverse events (TEAEs).'}, {'measure': 'Number of Participants With Drug -Related TEAEs and Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline up to Week 52', 'description': "An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and AEs. An AE was regarded as TEAE if the start date was on or after the infusion of SPK-9001 but before participant's last visit on study (or the date of withdrawal/the date of being lost to follow-up). Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator."}, {'measure': 'Number of Participants With Positive Immune Reponses Against Adeno-associated Virus Vector (AAV) Capsid', 'timeFrame': 'Baseline up to Week 52', 'description': 'Peripheral blood mononuclear cells (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT) to assess cellular immune responses to AAV capsid and to FIX were presented. The ELISPOT is a type of assay that focuses on quantitatively measuring the frequency of cytokine secretion for a single cell. The positive ELISPOT results suggested a T-cell reaction to capsid protein.'}, {'measure': 'Number of Participants Who Reached > 150% Vector-derived FIX:C Activity Level After SPK-9001 Infusion', 'timeFrame': 'Baseline up to Week 52', 'description': 'Based on non-clinical studies in non-human primates (NHPs), it was not predicted that vector-derived FIX:C activity levels \\>150% of normal would be achieved in this study. However, thrombin antithrombin (TAT) levels as thrombotic potential were to be measured if vector derived FIX:C activity levels \\>150% of normal were achieved in any participant during the study. Blood samples for TAT at Day 0 visit (prior to FIX protein product infusion) were used to establish baseline value.'}, {'measure': 'Number of Participants With FIX Inhibitor', 'timeFrame': 'Baseline up to Week 52', 'description': 'FIX inhibitors were measured using the Bethesda assay from the central and local laboratory. The Bethesda assay measures the amount of factor (FIX) inactivated when the plasma from the patient is incubated with an external source of factor for 2 hours at 37ºC. Inhibitor levels are quantified in Bethesda units (BU). An inhibitor titer of ≥ 0.6 BU/ml is to be taken as clinically significant.'}, {'measure': 'Incremental Recovery of FIX Product', 'timeFrame': 'Day 0 and Week 52', 'description': 'Incremental recovery was determined as the peak factor level recorded within the first 3 hours after infusion and was reported as (IU/ml)/(IU/kg), using the formula:(\\[Activity IU/mL peak post infusion\\] - \\[Activity IU/mL pre-infusion\\]) / (IU/kg infused).'}], 'secondaryOutcomes': [{'measure': 'FIX:C Activity', 'timeFrame': 'Baseline up to Week 52', 'description': 'All samples collected from participants for plasma FIX activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FIX activity levels. The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity levels were characterized by post-treatment population mean. Dose escalation and dose level expansion strategies were employed in the study based on vector-derived FIX activity levels as well as any immune responses against AAV capsid. Steady-state levels were based on 2 separate vector-derived FIX:C activity level measurements (at least 2 weeks apart) starting from Week 8-12 with adequate washout.'}, {'measure': 'Change From Baseline in FIX:C Antigen Level at Steady State', 'timeFrame': 'Week 12 up to Week 52', 'description': 'The vector-derived endogenous (not affected by intercurrent FIX product infusions) FIX:C activity antigen levels were characterized by post-treatment population mean.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hemophilia B', 'Blood Coagulation Disorders', 'Gene Therapy', 'Gene Transfer', 'Factor IX Deficiency', 'Factor IX Gene', 'Factor IX Protein', 'Vector', 'AAV'], 'conditions': ['Hemophilia B']}, 'referencesModule': {'references': [{'pmid': '40750723', 'type': 'DERIVED', 'citation': 'Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1.'}, {'pmid': '38863155', 'type': 'DERIVED', 'citation': 'Pittman DD, Carrieri C, Soares H, McKay J, Tan CY, Liang JZ, Rakhe S, Marshall JC, Murphy JE, Gaitonde P, Rupon J. Field Study and Correlative Studies of Factor IX Variant FIX-R338L in Participants Treated with Fidanacogene Elaparvovec. Thromb Haemost. 2024 Oct;124(10):912-921. doi: 10.1055/s-0044-1787734. Epub 2024 Jun 11.'}, {'pmid': '29211678', 'type': 'DERIVED', 'citation': 'George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.'}]}, 'descriptionModule': {'briefSummary': 'A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.', 'detailedDescription': 'Hemophilia B, or Christmas disease, is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes leads to death due to the inability for blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current treatment is intravenous infusion of FIX protein products, either prophylactically or in response to bleeding.\n\nThe approach being tested in this study uses a novel recombinant adeno-associated virus (AAV), which in nature causes no disease, to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made. Recent data of a gene therapy study showed preliminary encouraging results with the approach of using an AAV vector carrying the factor IX gene. This study will seek to determine the safety and kinetics of a single IV infusion of SPK-9001 (a novel AAV vector carrying a high specific activity factor IX variant).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'genderBased': True, 'genderDescription': 'Genetic Males', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Able to provide informed consent and comply with requirements of the study\n* Males ≥18 y.o. with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous factor IX)\n* Received ≥50 exposure days to factor IX products\n* A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions\n* No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein\n* Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences\n\nExclusion Criteria:\n\n* Evidence of active hepatitis B or C\n* Currently on antiviral therapy for hepatitis B or C\n* Have significant underlying liver disease\n* Have serological evidence\\* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (\\* subjects who are HIV+ and stable with CD4 count \\>200/mm3 and undetectable viral load are eligible to enroll)\n* Neutralizing antibodies reactive with AAV-Spark100 above and/or below a defined titre\n* Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 12 weeks\n* Unable or unwilling to comply with study assessments'}, 'identificationModule': {'nctId': 'NCT02484092', 'briefTitle': 'A Gene Therapy Study for Hemophilia B', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': 'Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B', 'orgStudyIdInfo': {'id': 'C0371005'}, 'secondaryIdInfos': [{'id': 'SPK-9001-101', 'type': 'OTHER', 'domain': 'Alias Study Number'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'SPK-9001', 'description': 'Single intravenous (i.v.) infusion of SPK-9001 \\[an adeno-associated viral (AAV) vector with human factor IX gene\\] Intervention: Gene Therapy / Gene Transfer', 'interventionNames': ['Biological: SPK-9001']}], 'interventions': [{'name': 'SPK-9001', 'type': 'BIOLOGICAL', 'description': 'A novel, bioengineered adeno-associated viral vector carrying human factor IX variant', 'armGroupLabels': ['SPK-9001']}]}, 'contactsLocationsModule': {'locations': [{'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Comprehensive Cancer Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis CTSC Clinical Research Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Ellison Ambulatory Care Clinic', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Investigational Pharmacy', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Medical Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '39216', 'city': 'Jackson', 'state': 'Mississippi', 'country': 'United States', 'facility': 'University of Mississippi Medical Center', 'geoPoint': {'lat': 32.29876, 'lon': -90.18481}}, {'zip': '39110', 'city': 'Madison', 'state': 'Mississippi', 'country': 'United States', 'facility': 'Mississippi Center for Advanced Medicine', 'geoPoint': {'lat': 32.46181, 'lon': -90.11536}}, {'zip': '10065', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Weill Cornell Medicine - New York Presbyterian Hospital', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "The Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '2050', 'city': 'Camperdown/Sydney', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Royal Prince Alfred Hospital'}], 'overallOfficials': [{'name': 'Pfizer CT.gov Call Center', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pfizer'}]}, 'ipdSharingStatementModule': {'url': 'https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests', 'ipdSharing': 'YES', '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'}}}}