Ignite Creation Date:
2025-12-24 @ 1:04 PM
Ignite Modification Date:
2025-12-27 @ 10:00 PM
Study NCT ID:
NCT06488261
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-22
First Post:
2023-11-20
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Follow-Up for Safety of AVR-RD-02
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005776', 'term': 'Gaucher Disease'}], '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': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-02-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2037-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-17', 'studyFirstSubmitDate': '2023-11-20', 'studyFirstSubmitQcDate': '2024-06-27', 'lastUpdatePostDateStruct': {'date': '2025-07-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-07-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2037-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Change from baseline over time in hemoglobin concentration', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Change from baseline over time in platelet count', 'timeFrame': 'Baseline to Year 14'}], 'secondaryOutcomes': [{'measure': 'Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Change from baseline over time in anti-GCase total antibodies and subsequent titers', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood', 'timeFrame': 'Baseline to Year 14'}, {'measure': 'Presence of replication competent lentivirus (RCL)', 'timeFrame': 'Baseline to Year 14'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Gaucher Disease']}, 'descriptionModule': {'briefSummary': 'The goal of this observational study is to assess the safety and tolerability of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).', 'detailedDescription': 'Subjects who received AVR-RD-02 in the preceding treatment study (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects with Gaucher disease who previously received the investigational gene therapy product AVR-RD-02', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria\n\nSubjects must meet all of the following inclusion criteria for participation in this study:\n\n1. Subject must be willing and able to provide written informed consent for the JAB-GD-001 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.\n2. Subject must have been enrolled in the preceding AVROBIO AVRO-RD-02-201 treatment study and have received AVR-RD-02 treatment\n\nExclusion Criteria\n\nSubject 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."}, 'identificationModule': {'nctId': 'NCT06488261', 'briefTitle': 'Long Term Follow-Up for Safety of AVR-RD-02', 'organization': {'class': 'OTHER', 'fullName': 'University of Iowa'}, 'officialTitle': 'A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02', 'orgStudyIdInfo': {'id': '202309221'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Subjects who received AVR-RD-02', 'description': 'Subjects who received AVR-RD-02 in a separate parent trial', 'interventionNames': ['Biological: AVR-RD-02']}], 'interventions': [{'name': 'AVR-RD-02', 'type': 'BIOLOGICAL', 'description': 'No study drug is administered in this study. Subjects who received AVR-RD-02 in a separate parent trial will be evaluated in this trial for long-term safety.', 'armGroupLabels': ['Subjects who received AVR-RD-02']}]}, 'contactsLocationsModule': {'locations': [{'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa Hospitals and Clinics', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}], 'overallOfficials': [{'name': 'John A. Bernat, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Iowa'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'John Bernat', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Clinical Associate Professor', 'investigatorFullName': 'John Bernat', 'investigatorAffiliation': 'University of Iowa'}}}}