Ignite Creation Date:
2025-12-24 @ 2:48 PM
Ignite Modification Date:
2026-01-04 @ 8:43 AM
Study NCT ID:
NCT04999059
Status:
TERMINATED
Last Update Posted:
2023-08-31
First Post:
2021-08-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'releaseDate': '2023-11-09', 'unreleaseDate': '2024-01-08'}, {'resetDate': '2024-07-11', 'releaseDate': '2024-01-12'}], 'estimatedResultsFirstSubmitDate': '2023-11-09'}}, '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D012449', 'term': 'Safety'}], 'ancestors': [{'id': 'D000056', 'term': 'Accident Prevention'}, {'id': 'D000059', 'term': 'Accidents'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Blood sample collections including serum chemistry (and electrolytes), hematology, and anti-AGA antibodies, and urinalysis. Bone marrow aspirate for efficacy, and reproductive testing.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}, 'patientRegistry': False}, 'statusModule': {'whyStopped': 'This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety or efficacy issue.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-05-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-10', 'completionDateStruct': {'date': '2023-08-16', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-08-29', 'studyFirstSubmitDate': '2021-08-04', 'studyFirstSubmitQcDate': '2021-08-04', 'lastUpdatePostDateStruct': {'date': '2023-08-31', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-08-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-08-16', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of clinically significant AEs and SAEs', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Number of participants with clinically relevant abnormalities, as assessed by vital signs', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Presence of anti-Alpha-galactosidase A (AGA) antibodies', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Presence of replication competent lentivirus (RCL)', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}], 'secondaryOutcomes': [{'measure': 'Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs)', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'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': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'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': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Change from baseline in eGFR', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'measure': 'Change from baseline in left ventricular mass index (LVMI) as assessed by cardiac magnetic resonance imaging (MRI)', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'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 Year 15 post gene therapy infusion'}, {'measure': 'Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores', 'timeFrame': 'Baseline to Year 15 post gene therapy infusion'}, {'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 Year 15 post gene therapy infusion'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Fabry Disease', 'Cell therapy', 'Gene therapy', 'Long-term follow-up', 'Lysosomal storage disorder', 'Lenti-viral'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': 'This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.', 'detailedDescription': "Participants enrolled in the AVRO-RD-01-201 study will be offered participation in the AVRO-RD-01-LTF01 study. The Baseline visit for the AVRO-RD-01-LTF01 study will coincide with the participant's last visit in the AVRO-RD-01-201 study. Participants confirmed eligible for the AVRO-RD-01-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for 10 years (for a total of 15 years from AVR-RD-01 infusion) during which time continued safety, engraftment, and efficacy of treatment will be assessed."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '16 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects who received AVR-RD-01 in the preceding treatment study, meet all eligibility criteria, and agree to comply with the study visit schedule and procedures.', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1\\. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.\n\nExclusion Criteria:\n\n1. Subject has any medical, psychological, or other condition that, in the opinion of the Investigator:\n\n * Might interfere with the subject's participation in the study (including consenting to procedures); and/or\n * Poses any additional risk to the subject; and/or\n * Might confound the results of any study-required assessments.\n2. Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study."}, 'identificationModule': {'nctId': 'NCT04999059', 'briefTitle': 'Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201', 'organization': {'class': 'INDUSTRY', 'fullName': 'AVROBIO'}, 'officialTitle': 'A Long-Term Follow-Up Study of Subjects With Fabry Disease Who Previously Received Ex-Vivo, Lentiviral Vector-Mediated Gene-Modified Autologous Cell Therapy AVR-RD-01 in Study AVRO-RD-01-201', 'orgStudyIdInfo': {'id': 'AVRO-RD-01-LTF01'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Participants with Fabry Disease', 'description': 'This is a long-term follow-up study of participants who previously received AVR-RD-01 (single dose administration) in the AVRO-RD-01-201 treatment study. No investigational product will be administered in this study.', 'interventionNames': ['Other: Safety and Efficacy Assessments']}], 'interventions': [{'name': 'Safety and Efficacy Assessments', 'type': 'OTHER', 'otherNames': ['Gene Therapy Intervention'], 'description': 'Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.', 'armGroupLabels': ['Participants with Fabry Disease']}]}, 'contactsLocationsModule': {'locations': [{'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}}, {'zip': '90420-020', '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'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AVROBIO', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}, 'annotationSection': {'annotationModule': {'unpostedAnnotation': {'unpostedEvents': [{'date': '2023-11-09', 'type': 'RELEASE'}, {'date': '2024-01-08', 'type': 'UNRELEASE'}, {'date': '2024-01-12', 'type': 'RELEASE'}, {'date': '2024-07-11', 'type': 'RESET'}], 'unpostedResponsibleParty': 'AVROBIO'}}}}