Ignite Creation Date:
2025-12-25 @ 1:13 AM
Ignite Modification Date:
2025-12-25 @ 11:23 PM
Study NCT ID:
NCT03454893
Status:
TERMINATED
Last Update Posted:
2024-01-05
First Post:
2017-12-20
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Canada'], 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-10-19', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@avrobio.com', 'phone': '617-914-8419', 'title': 'AVROBIO MedInfo', 'organization': 'AVROBIO, Inc'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'SF-36 data at W24 was inadvertently not collected due to omission in the Protocol Schedule of Assessments.\n\nInvasive sampling, such as bone marrow aspirate, was only provided by one subject at Week 48.\n\nCentral laboratory only reported albumin levels in urine and not urine total protein data.'}}, 'adverseEventsModule': {'timeFrame': 'Up to week 48', 'description': 'Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.', 'eventGroups': [{'id': 'EG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.', 'otherNumAtRisk': 11, 'deathsNumAtRisk': 11, 'otherNumAffected': 11, 'seriousNumAtRisk': 11, 'deathsNumAffected': 0, 'seriousNumAffected': 8}], 'otherEvents': [{'term': 'Anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Febrile neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Leukopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 11, 'numAffected': 9}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lymphopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 14, 'numAffected': 9}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 12, 'numAffected': 9}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Sinus tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': "Fabry's disease", 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Ear congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Primary hypogonadism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry eye', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Anal fissure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dental caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Haemorrhoids', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hiatus hernia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 15, 'numAffected': 10}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Odynophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Stomatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tooth impacted', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 10, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Face oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Feeling abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mucosal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nodule', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Oedema peripheral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vaccination site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hepatic steatosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypertransaminasaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Immunisation reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Escherichia urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Folliculitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gingivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Post-acute COVID-19 syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Postoperative wound infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tinea cruris', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tooth infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary tract infection bacterial', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular access site infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Citrate toxicity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Meniscus cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Post procedural constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Procedural pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 9, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tooth fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tooth injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Traumatic haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular access site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular access site haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vascular access site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood alkaline phosphatase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood follicle stimulating hormone increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood lactate dehydrogenase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood phosphorus increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Blood potassium decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'CD4 lymphocytes decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cardiac murmur', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gamma-glutamyltransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urine analysis abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abnormal loss of weight', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abnormal weight gain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyperlipidaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypomagnesaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyponatraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypophosphataemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypovolaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vitamin D deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Bone pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Joint swelling', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dysgeusia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Neuralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Parosmia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Depressed mood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Acute kidney injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dysuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Renal haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Urinary hesitation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Azoospermia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Erectile dysfunction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gynaecomastia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Oligospermia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Scrotal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Scrotal erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Choking', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Chronic obstructive pulmonary disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry throat', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hiccups', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Increased viscosity of upper respiratory secretion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lung infiltration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Painful respiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pharyngeal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dermatosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dry skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hair growth abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lichen planus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nail ridging', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Neurodermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pain of skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pigmentation disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Skin plaque', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Orthostatic hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Febrile neutropenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Thrombocytopenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Odynophagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mucosal inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cholelithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Choking', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Lung infiltration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'AE', 'categories': [{'measurements': [{'value': '354', 'groupId': 'OG000'}]}]}, {'title': 'SAE', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. The AE/SAE are also inclusive of any abnormalities in Clinical Laboratory Tests, Vital Signs and in Electrocardiographs (ECGs). Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.', 'unitOfMeasure': 'Number of events', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety population consists of all enrolled subjects who received any preparatory medication.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in Immunogenicity of AVR-RD-01', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Number of subjects with changes in anti-AGA antibodies from Baseline to post infusion timepoints. Unite of measure: Number of subjects negative at baseline but positive at post-treatment timepoints. A negative or zero result (titer lower or unchanged at post-infusion timepoints compare to Baseline) indicates no immune response to the therapeutic protein.', 'unitOfMeasure': 'Number of positive subjects', 'reportingStatus': 'POSTED', 'populationDescription': 'The infused population includes all enrolled subjects who received preparatory medications and AVR-RD-01. Four tested positive for anti-AGA antibodies post-AVR-RD-01 infusion at Week 48 or Early Termination visit.'}, {'type': 'PRIMARY', 'title': 'Presence of Replication Competent Lentivirus (RCL)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'The "Presence of RCL" is a theoretical risk of lentiviral gene therapy treatment based on the theory that it may be possible for inadvertent generation of RCL caused either by recombination of the lentiviral vector plasmids during the vector production process or by mobilization of proviral DNA in vivo by infectious retroviruses (HIV). The absence of RCL is a positive indicator of safety.', 'unitOfMeasure': 'Number of positive subjects', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'PRIMARY', 'title': 'Evaluation of Aberrant Clonal Expansion', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Integration Site Analysis (ISA) uses next generation sequencing to identify junction sites between the integrated therapeutic transgene and the host genome. Samples are analyzed for the emergence of clonality (defined as (a single clone accounting for greater than 20% of the population) and whether any integration site is within or near a known oncogene.', 'unitOfMeasure': 'number of abnormal clonal proliferations', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'PRIMARY', 'title': 'Change From Baseline in the Average Number of Gb3 Inclusions (ie, Myelinosomes) Per Kidney Peritubular Capillary (PTC) Per Subject', 'denoms': [{'units': 'Participants', 'counts': [{'value': '2', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '-3.555', 'groupId': 'OG000', 'lowerLimit': '-4.020', 'upperLimit': '-3.090'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) Inclusions in Peritubular Capillaries (PTC) on Kidney Biopsy. Electron microscopic images of kidney biopsy samples were taken and read centrally by two independent renal pathologists, each of whom scored the average number of Gb3 inclusions per kidney PTC per subject using a quantification method. Healthy renal tissue would have no Gb3 inclusions. A reduction from baseline is desirable.', 'unitOfMeasure': 'Number of inclusions', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. Of the nine subjects, only 2 subjects had baseline and Week 48 results to measure change from baseline. The remaining seven subjects either did not provide both samples or the samples provided were not evaluable.'}, {'type': 'SECONDARY', 'title': 'Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.383', 'spread': '0.4628', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.313', 'spread': '0.3447', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Week 24 and Week 48 post gene therapy', 'description': 'Vector Copy Number (VCN) is a measurement of the number of copies of the therapeutic transgene found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one \\[working\\] copy of the therapeutic transgene per cell.', 'unitOfMeasure': 'copies/diploid genome', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'CFB Plasma W24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.973', 'spread': '2.585', 'groupId': 'OG000'}]}]}, {'title': 'CFB Plasma W48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.548', 'spread': '2.152', 'groupId': 'OG000'}]}]}, {'title': 'CFB PBL Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '29.524', 'spread': '39.194', 'groupId': 'OG000'}]}]}, {'title': 'CFB PBL Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.973', 'spread': '32.800', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Treatment-naïve Fabry patients are deficient in alpha-galactosidase A (AGA) enzyme activity due to mutations in the GLA gene. Any therapeutic option offered should aim to increase the amount of available AGA enzyme. This assay measured the AGA enzyme activity levels in plasma and PBLs. It should be noted that the measurement in plasma reflects the amount of "free" AGA enzyme that has been released from cells into the extracellular space and is therefore considered a more indirect measure of AGA enzyme activity, compared to the result in PBLs which is more of a direct measure of enzyme within cells. In both cases, enzyme activity is expected to increase from Baseline to the post-infusion timepoints.', 'unitOfMeasure': 'nmol/h/mg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Plasma', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3334.0', 'spread': '3846.8', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5067.8', 'spread': '4067.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in plasma. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of circulating Gb3 in plasma.', 'unitOfMeasure': 'nM', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Urine', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24', 'categories': [{'measurements': [{'value': '-11.73', 'spread': '309.78', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'categories': [{'measurements': [{'value': '-188.73', 'spread': '262.90', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in urine. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of excreted Gb3 in urine.', 'unitOfMeasure': 'nmol/mmol creatinine', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Myelinosomes in superficial dermal capillary endothelial cells (Week 24)', 'categories': [{'measurements': [{'value': '-0.4', 'spread': '1.3', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in superficial dermal capillary endothelial cells (Week 48)', 'categories': [{'measurements': [{'value': '-0.5', 'spread': '1.0', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in deep dermal capillary endothelial cells (Week 24)', 'categories': [{'measurements': [{'value': '0', 'spread': '1.3', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in deep dermal capillary endothelial cells (Week 48)', 'categories': [{'measurements': [{'value': '-0.5', 'spread': '1.0', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in vascular smooth muscle cells (Week 24)', 'categories': [{'measurements': [{'value': '0.1', 'spread': '1.2', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in vascular smooth muscle cells (Week 48)', 'categories': [{'measurements': [{'value': '-0.5', 'spread': '1.0', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in perineurium (Week 24)', 'categories': [{'measurements': [{'value': '0', 'spread': '1.4', 'groupId': 'OG000'}]}]}, {'title': 'Myelinosomes in perineurium (Week 48)', 'categories': [{'measurements': [{'value': '-0.5', 'spread': '1.0', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in tissue samples. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of measured Gb3 in tissues.', 'unitOfMeasure': 'number of myelinosomes', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Renal Function as Assessed by Measured Glomerular Filtration Rate (mGFR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.358', 'spread': '14.502', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'mGFR is a measure of the time the kidney takes to filter products that the body does not naturally produce.', 'unitOfMeasure': 'mL/min/1.73m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Renal Function as Assessed by Estimated Glomerular Filtration Rate (eGFR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24', 'categories': [{'measurements': [{'value': '-6.386', 'spread': '9.699', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'categories': [{'measurements': [{'value': '-3.561', 'spread': '7.332', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'eGFR is the measure to evaluate kidney function. It is the estimated amount of blood that is filtered through all glomeruli in a given time.', 'unitOfMeasure': 'mL/min/1.73 m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Renal Function as Assessed by Urine Total Protein Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. No data were collected for any subjects since samples were not analyzed in order to produce data (Refer to "Limitations and Caveat" section.")'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Renal Function as Assessed by Urine Albumin Levels', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24', 'categories': [{'measurements': [{'value': '0.915', 'spread': '6.704', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'categories': [{'measurements': [{'value': '0.643', 'spread': '5.789', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'A healthy kidney only allows very small amounts of albumin to pass from the blood into the urine. An increased level of albumin in urine (albuminuria) is a marker of renal damage.', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Left Ventricular Mass Index (LVMI) as Assessed by Cardiac Magnetic Resonance Imaging (MRI)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '7.270', 'spread': '6.055', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'LVMI is a surrogate of left ventricular hypertrophy. An increase in LVMI is an independent risk factor for cardiovascular morbidity and mortality.', 'unitOfMeasure': 'g/m^2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Abdominal Pain Week 24', 'categories': [{'measurements': [{'value': '-0.445', 'spread': '0.583', 'groupId': 'OG000'}]}]}, {'title': 'Abdominal Pain Week 48', 'categories': [{'measurements': [{'value': '-0.497', 'spread': '0.626', 'groupId': 'OG000'}]}]}, {'title': 'Stool Consistency Week 24', 'categories': [{'measurements': [{'value': '-0.373', 'spread': '0.216', 'groupId': 'OG000'}]}]}, {'title': 'Stool Consistency Week 48', 'categories': [{'measurements': [{'value': '-0.530', 'spread': '0.104', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'DIBSS-D assesses bowel habits and abdominal symptoms over a period of time. It was administered daily for 14 days commencing at each study visit.\n\nStool Consistency scale has been converted to a numeric rating scale for ease of analysis, where 1=Very hard; 2=Hard; 3=Neither too hard nor too soft; 4=Loose but not lumpy; 5=Very loose and watery. The median of each 14-day period was derived per patient, per visit before deriving the group median. Group median at Baseline (pre-treatment) was 3.540 (n=9). Change from baseline (CFB) is presented below. An increase CFB indicates softening of the stools, and a decrease CFB indicates hardening of the stools. Abdominal Pain measure asked the patient to rate the worst level of pain within the past 24hrs (0=no pain; 10=worst possible pain). A mean score was derived for each 14-day period per patient, per visit, and these means used to derive a group mean. An increase CFB indicates more abdominal pain; a decrease CFB indicates less abdominal pain.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Week 24 (pain severity)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.188', 'spread': '0.874', 'groupId': 'OG000'}]}]}, {'title': 'Week 48 (pain severity)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.500', 'spread': '0.612', 'groupId': 'OG000'}]}]}, {'title': 'Week 24 (pain interference)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.073', 'spread': '1.007', 'groupId': 'OG000'}]}]}, {'title': 'Week 48 (pain interference)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.573', 'spread': '0.996', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'The short version of the BPI (Short form) includes 9 items: Q1 - Q9, Question 9 includes 7 sub-items (Q9a - Q9g). It uses a 0 to 10 numeric rating scales for item rating. The pain severity score is calculated as the average of questions answered: Q3 (worst pain), Q4 (least pain), Q5 (average pain) and Q6 (current pain). The pain interference score is calculated as the average of the answered Q9 sub-items, which represents pain interference with general activity (Q9a), mood (Q9b), walking ability (Q9c), normal work (Q9d), relations with other people (Q9e), sleep (Q9f), and enjoyment of life (Q9g). A reduction in score from baseline indicates less pain.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Physical and Mental Functioning as Assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'title': 'Physical Functioning Week 48', 'categories': [{'measurements': [{'value': '3.235', 'spread': '4.810', 'groupId': 'OG000'}]}]}, {'title': 'Mental Functioning Week 48', 'categories': [{'measurements': [{'value': '-1.580', 'spread': '4.652', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'The original version of the SF-36 was administered to the participants and consisted of eight subscales (Vitality, Physical Functioning, Bodily Pain, General Health Perceptions, Physical Role Functioning, Emotional Role Functioning, Social Role Functioning and Mental Health) each scored from 0 (worst health) to 100 (best heath). These scores were normalized (re-scaled) against mean scores obtained in the US general population (Mean=50, Standard deviation 10). The summary health components PCS and MCS are derived from the eight subscales mentioned above and summarize information from all eight subscales but with different weights. For PCS, highest weights are given to the physical subscales while some mental subscales are given negative weights. For MCS, highest weights are given to the mental subscales while some physical subscales are given negative weights. An increase in the normalized score from baseline indicates improvement in physical and mental functioning.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. For Week 24 data, please see comment in "Limitations and Caveats" section.'}, {'type': 'SECONDARY', 'title': 'Average Vector Copy Number (VCN) in Bone Marrow / Progenitor Cells as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '1', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.14', 'groupId': 'OG000', 'lowerLimit': '1.14', 'upperLimit': '1.14'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'At Week 48 post gene therapy', 'description': 'VCN is defined as the average number of copies of the therapeutic gene (transgene) in a sample of cells and is a measurement of the number of copies of the vector found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one \\[working\\] copy of the therapeutic transgene per cell. This measurement was for VCN in a sample of Bone marrow progenitor cells obtained from an aspirate.', 'unitOfMeasure': 'copies/diploid genome', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'The Infused population consists of all enrolled subjects who received all preparatory medications and AVR-RD-01. No analysis performed (refer to "Limitations and Caveats" section)'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'Safety Population', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}]}, {'type': 'Infused Population', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '9'}]}], 'dropWithdraws': [{'type': 'Study Terminated by the Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Did not pass screening', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}]}], 'recruitmentDetails': 'Total number of subjects who signed the ICF was 16 (one subject who re-screened was not considered enrolled initially), as a result there were 15 distinct subjects who signed ICF. There were 4 participants who failed screening and were not rescreened.\n\nThe Safety population consists of all 11 enrolled subjects who received any preparatory medication.\n\nThe Infused population consists of all 9 enrolled subjects who received all preparatory medications and AVR-RD-01.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.\n\nDrug: AVR-RD-01 Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '31.45', 'spread': '10.98', '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': '11', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Safety population consists of all enrolled subjects who received any preparatory medication.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-04-01', 'size': 8215200, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_002.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-11-08T14:44', 'hasProtocol': True}, {'date': '2023-07-26', 'size': 5528020, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_003.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-11-08T14:45', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'whyStopped': 'AVROBIO has deprioritized its Fabry disease program', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2018-02-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'dispFirstSubmitDate': '2022-11-15', 'completionDateStruct': {'date': '2022-03-14', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-01-03', 'studyFirstSubmitDate': '2017-12-20', 'resultsFirstSubmitDate': '2023-09-25', 'studyFirstSubmitQcDate': '2018-02-27', 'dispFirstPostDateStruct': {'date': '2024-01-05', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2024-01-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-01-03', 'studyFirstPostDateStruct': {'date': '2018-03-06', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-01-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-03-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. The AE/SAE are also inclusive of any abnormalities in Clinical Laboratory Tests, Vital Signs and in Electrocardiographs (ECGs). Of the 13 serious adverse events, no SAEs reported were considered related to AVR RD 01. Of the 354 adverse events, no AEs were considered related to AVR RD 01. The SAEs and AEs reported in the study were attributed to the conditioning agent used, the underlying disease, comorbid conditions, study procedures and concomitant medications.'}, {'measure': 'Change From Baseline in Immunogenicity of AVR-RD-01', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Number of subjects with changes in anti-AGA antibodies from Baseline to post infusion timepoints. Unite of measure: Number of subjects negative at baseline but positive at post-treatment timepoints. A negative or zero result (titer lower or unchanged at post-infusion timepoints compare to Baseline) indicates no immune response to the therapeutic protein.'}, {'measure': 'Presence of Replication Competent Lentivirus (RCL)', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'The "Presence of RCL" is a theoretical risk of lentiviral gene therapy treatment based on the theory that it may be possible for inadvertent generation of RCL caused either by recombination of the lentiviral vector plasmids during the vector production process or by mobilization of proviral DNA in vivo by infectious retroviruses (HIV). The absence of RCL is a positive indicator of safety.'}, {'measure': 'Evaluation of Aberrant Clonal Expansion', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Integration Site Analysis (ISA) uses next generation sequencing to identify junction sites between the integrated therapeutic transgene and the host genome. Samples are analyzed for the emergence of clonality (defined as (a single clone accounting for greater than 20% of the population) and whether any integration site is within or near a known oncogene.'}, {'measure': 'Change From Baseline in the Average Number of Gb3 Inclusions (ie, Myelinosomes) Per Kidney Peritubular Capillary (PTC) Per Subject', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) Inclusions in Peritubular Capillaries (PTC) on Kidney Biopsy. Electron microscopic images of kidney biopsy samples were taken and read centrally by two independent renal pathologists, each of whom scored the average number of Gb3 inclusions per kidney PTC per subject using a quantification method. Healthy renal tissue would have no Gb3 inclusions. A reduction from baseline is desirable.'}], 'secondaryOutcomes': [{'measure': 'Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)', 'timeFrame': 'At Week 24 and Week 48 post gene therapy', 'description': 'Vector Copy Number (VCN) is a measurement of the number of copies of the therapeutic transgene found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one \\[working\\] copy of the therapeutic transgene per cell.'}, {'measure': 'Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs)', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Treatment-naïve Fabry patients are deficient in alpha-galactosidase A (AGA) enzyme activity due to mutations in the GLA gene. Any therapeutic option offered should aim to increase the amount of available AGA enzyme. This assay measured the AGA enzyme activity levels in plasma and PBLs. It should be noted that the measurement in plasma reflects the amount of "free" AGA enzyme that has been released from cells into the extracellular space and is therefore considered a more indirect measure of AGA enzyme activity, compared to the result in PBLs which is more of a direct measure of enzyme within cells. In both cases, enzyme activity is expected to increase from Baseline to the post-infusion timepoints.'}, {'measure': 'Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Plasma', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in plasma. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of circulating Gb3 in plasma.'}, {'measure': 'Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Urine', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in urine. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of excreted Gb3 in urine.'}, {'measure': 'Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'Globotriaosylceramide (Gb3) is the substrate that accumulates in the lysosomes of patients affected by Fabry Disease as a result of deficiencies in AGA enzyme activity. Treatment-naive patients are expected to have high levels of Gb3 in their lysosomes and correspondingly elevated levels in tissue samples. Treatment with AVR-RD-01 is intended to replace the missing AGA enzymatic activity, which allows degradation of accumulated Gb3 substrate in the lysosomes and reductions in the levels of measured Gb3 in tissues.'}, {'measure': 'Change From Baseline in Renal Function as Assessed by Measured Glomerular Filtration Rate (mGFR)', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'mGFR is a measure of the time the kidney takes to filter products that the body does not naturally produce.'}, {'measure': 'Change From Baseline in Renal Function as Assessed by Estimated Glomerular Filtration Rate (eGFR)', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'eGFR is the measure to evaluate kidney function. It is the estimated amount of blood that is filtered through all glomeruli in a given time.'}, {'measure': 'Change From Baseline in Renal Function as Assessed by Urine Total Protein Levels', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy'}, {'measure': 'Change From Baseline in Renal Function as Assessed by Urine Albumin Levels', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'A healthy kidney only allows very small amounts of albumin to pass from the blood into the urine. An increased level of albumin in urine (albuminuria) is a marker of renal damage.'}, {'measure': 'Change From Baseline in Left Ventricular Mass Index (LVMI) as Assessed by Cardiac Magnetic Resonance Imaging (MRI)', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'LVMI is a surrogate of left ventricular hypertrophy. An increase in LVMI is an independent risk factor for cardiovascular morbidity and mortality.'}, {'measure': 'Change From Baseline in Abdominal Pain and Stool Consistency as Assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D)', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'DIBSS-D assesses bowel habits and abdominal symptoms over a period of time. It was administered daily for 14 days commencing at each study visit.\n\nStool Consistency scale has been converted to a numeric rating scale for ease of analysis, where 1=Very hard; 2=Hard; 3=Neither too hard nor too soft; 4=Loose but not lumpy; 5=Very loose and watery. The median of each 14-day period was derived per patient, per visit before deriving the group median. Group median at Baseline (pre-treatment) was 3.540 (n=9). Change from baseline (CFB) is presented below. An increase CFB indicates softening of the stools, and a decrease CFB indicates hardening of the stools. Abdominal Pain measure asked the patient to rate the worst level of pain within the past 24hrs (0=no pain; 10=worst possible pain). A mean score was derived for each 14-day period per patient, per visit, and these means used to derive a group mean. An increase CFB indicates more abdominal pain; a decrease CFB indicates less abdominal pain.'}, {'measure': 'Change From Baseline in Brief Pain Inventory-Short Form (BPI-SF) Questionnaire Scores', 'timeFrame': 'Baseline to Week 24 and Week 48 post gene therapy', 'description': 'The short version of the BPI (Short form) includes 9 items: Q1 - Q9, Question 9 includes 7 sub-items (Q9a - Q9g). It uses a 0 to 10 numeric rating scales for item rating. The pain severity score is calculated as the average of questions answered: Q3 (worst pain), Q4 (least pain), Q5 (average pain) and Q6 (current pain). The pain interference score is calculated as the average of the answered Q9 sub-items, which represents pain interference with general activity (Q9a), mood (Q9b), walking ability (Q9c), normal work (Q9d), relations with other people (Q9e), sleep (Q9f), and enjoyment of life (Q9g). A reduction in score from baseline indicates less pain.'}, {'measure': 'Change From Baseline in Physical and Mental Functioning as Assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores', 'timeFrame': 'Baseline to Week 48 post gene therapy', 'description': 'The original version of the SF-36 was administered to the participants and consisted of eight subscales (Vitality, Physical Functioning, Bodily Pain, General Health Perceptions, Physical Role Functioning, Emotional Role Functioning, Social Role Functioning and Mental Health) each scored from 0 (worst health) to 100 (best heath). These scores were normalized (re-scaled) against mean scores obtained in the US general population (Mean=50, Standard deviation 10). The summary health components PCS and MCS are derived from the eight subscales mentioned above and summarize information from all eight subscales but with different weights. For PCS, highest weights are given to the physical subscales while some mental subscales are given negative weights. For MCS, highest weights are given to the mental subscales while some physical subscales are given negative weights. An increase in the normalized score from baseline indicates improvement in physical and mental functioning.'}, {'measure': 'Average Vector Copy Number (VCN) in Bone Marrow / Progenitor Cells as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)', 'timeFrame': 'At Week 48 post gene therapy', 'description': 'VCN is defined as the average number of copies of the therapeutic gene (transgene) in a sample of cells and is a measurement of the number of copies of the vector found in a sample, relative to copies of a reference gene in the human genome. This is an estimate of the number of integration sites per cell (on average). A VCN of 1 would signify that a sample of cells evaluated contains on average at least one \\[working\\] copy of the therapeutic transgene per cell. This measurement was for VCN in a sample of Bone marrow progenitor cells obtained from an aspirate.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Fabry disease', 'Cell therapy', 'Gene therapy', 'Lysosomal storage disorder', 'Lenti-viral'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': 'This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient alpha galactosidase A (AGA) enzyme activity who were considered treatment naïve, i.e., had not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening.', 'detailedDescription': "The duration of each subject's participation in this study was approximately 64 weeks (or 1 year, 12 weeks), comprised of five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up). During the Screening Period (approximately 8 weeks), written informed consent (and assent, if applicable) was obtained and the subject had completed other Screening procedures to confirm study eligibility. Once study eligibility was confirmed, the subjects entered the Baseline Period (up to 3 days) during which time assessments would have been performed to establish a pre-transplant baseline. Once baseline assessments were completed, the subject entered the Pre-transplant Period (approximately 6 weeks) during which time mobilization, apheresis, AVR-RD-01 investigational drug product preparation and testing for release, and conditioning regimen administration to achieve myeloablation took place. Following completion of the Pre-transplant Period, the subject entered the Transplant Period (1 day) during which time AVR-RD-01 infusion took place. After AVR-RD-01 infusion, the subject entered the Post-transplant Follow-up Period (approximately 48 weeks), during which time periodic safety and efficacy assessments were performed to assess measures of engraftment, clinical response, and safety post-transplant.\n\nIn January 2022, the study was terminated early due to a decision by the study sponsor, to deprioritize its Fabry disease development program, and therefore, some subjects (n=5) did not complete the study (i.e., Week 48). Subsequently, in August 2023, the long-term follow-up study (AVRO-RD-01-LTF01), was also terminated early due to the decision by the sponsor to terminate the development program for Fabry disease, and therefore, no subjects completed the 15-year long-term follow-up study. This decision to terminate was not based on any safety or medical reasons."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Subject was male, 16 years of age or older (18 years of age or older in the US), and post pubertal,(minimum age by region)\n2. Subject had a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as \\< 1% of normal).\n\nExclusion Criteria:\n\n1. Subject had a galactosidase alpha (GLA) gene mutation associated with late-onset cardiac variant Fabry disease.\n2. Subject had previously received ERT and/or chaperone therapy within 3 years for treatment of Fabry disease.\n3. Subject had tested positive for anti-AGA antibodies at the time of screening.\n4. Subject had eGFR \\< 60 mL/min/1.73 m² (ie, chronic kidney disease \\[CKD\\] stage ≥ 3) at Screening.\n5. Subject had a prior history of myocardial infarction (MI).\n6. Subject had a history of coronary artery disease (CAD) with angina requiring percutaneous transluminal coronary angioplasty (with or without stent placement) and/or coronary artery bypass graft (CABG).\n7. Subject had a history of moderate to severe valvular heart disease requiring valve replacement.\n8. Subject had a history of heart failure, moderate to severe diastolic dysfunction, and/or left ventricular ejection fraction (LVEF) ≤ 45% on echocardiogram (ECHO) performed at rest at Screening.\n9. Subject had a history of clinically significant cardiac arrhythmia (eg, heart block \\[second or third degree\\], atrial fibrillation requiring therapy, ventricular fibrillation, ventricular tachycardia, supraventricular tachycardia, or cardiac arrest).\n\n Note \\[history of intermittent atrial fibrillation not requiring treatment was allowed\\].\n10. Subject had a prior history of stroke and/or transient ischemic attack (TIA).\n11. Subject had aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening.\n12. Subject had a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma.\n13. Subject had previously received treatment with AVR-RD-01 or any other gene therapy.\n\nOther inclusion/exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT03454893', 'briefTitle': 'Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'AVROBIO'}, 'officialTitle': 'An Open-Label, Multinational Study Of The Efficacy And Safety of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy AVR-RD-01 For Treatment-Naive Subjects With Classic Fabry Disease', 'orgStudyIdInfo': {'id': 'AVRO-RD-01-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Single Assignment AVR-RD-01', 'description': 'AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.', 'interventionNames': ['Drug: AVR-RD-01']}], 'interventions': [{'name': 'AVR-RD-01', 'type': 'DRUG', 'description': 'Single IV infusion of between 3 - 20 x 10\\^6 CD34+ cells/kg.', 'armGroupLabels': ['Single Assignment AVR-RD-01']}]}, 'contactsLocationsModule': {'locations': [{'zip': '07601', 'city': 'Hackensack', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Hackensack University Medical Center', 'geoPoint': {'lat': 40.88593, 'lon': -74.04347}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'city': 'Melbourne', 'state': 'Parkville VIC', 'country': 'Australia', 'facility': 'Royal Melbourne Hospital', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'city': 'Perth', 'country': 'Australia', 'facility': 'Royal Perth Hospital', 'geoPoint': {'lat': -31.95224, 'lon': 115.8614}}, {'city': 'Porto Alegre', 'state': 'Rio Grande do Sul', 'country': 'Brazil', 'facility': 'Hospital de Clinicas de Porto Alegre', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}], 'overallOfficials': [{'name': 'Inderpal Panesar, MRPharmS', 'role': 'STUDY_DIRECTOR', 'affiliation': 'AVROBIO, Inc'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AVROBIO', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}