Ignite Creation Date:
2025-12-24 @ 10:55 PM
Ignite Modification Date:
2026-01-02 @ 1:39 AM
Study NCT ID:
NCT06614569
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-16
First Post:
2024-09-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020143', 'term': 'Gangliosidoses, GM2'}, {'id': 'D013661', 'term': 'Tay-Sachs Disease'}, {'id': 'D012497', 'term': 'Sandhoff Disease'}], 'ancestors': [{'id': 'D005733', 'term': 'Gangliosidoses'}, {'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': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'May possibly retain frozen blood samples.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 7}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-09-17', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2028-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-15', 'studyFirstSubmitDate': '2024-09-23', 'studyFirstSubmitQcDate': '2024-09-25', 'lastUpdatePostDateStruct': {'date': '2025-10-16', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-09-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Long term Safety of AXO-AAV-GM2', 'timeFrame': 'Every 6 months, up to five years', 'description': 'Participants will be monitored for adverse events, including delayed adverse events, as a result of AXO-AAV-GM2 including de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders.'}], 'secondaryOutcomes': [{'measure': 'Long term Impact of AXO-AAV-GM2', 'timeFrame': 'Every 6 months, up to five years', 'description': 'Participants will be monitored for the impact of treatment with AXO-AAV-GM2 on the expected natural history of their condition in the domains of neurocognitive adaptive, developmental, neurological and motor function.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['GM2 Gangliosidosis', 'Tay Sachs Disease', 'Sandhoff Disease']}, 'referencesModule': {'references': [{'pmid': '40817303', 'type': 'DERIVED', 'citation': 'Eichler F, Cataltepe OI, Daci R, Puri AS, Taghian T, Jiang X, Shazeeb MS, Kuhn A, Hader A, Celik H, Vardar Z, Lewis CJ, Artinian R, Nagy A, Vachha B, Thompson R, Gallagher T, Bateman S, Parzych J, Spanakis SG, Vaughn TA, Pier K, De Boever E, Abbott MA, D Ambrosio E, Kokoski D, Blackwood M, Drummond E, Ratai EM, Townsend EL, McLaughlin H, Tifft CJ, Keeler AM, Sena-Esteves M, Gray-Edwards HL, Flotte TR. Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial. Nat Med. 2025 Sep;31(9):2927-2935. doi: 10.1038/s41591-025-03822-4. Epub 2025 Aug 15.'}]}, 'descriptionModule': {'briefSummary': 'This study is to continue Long-Term Follow-Up of Patients who were previously treated with AXO-AAV-GM2 Gene Therapy as treatment for Tay-Sachs or Sandhoff Disease to follow the subjects through 5 years after their initial gene therapy treatment.', 'detailedDescription': 'Subjects will be contacted bi-annually for a total of five years following the administration of AXO-AAV-GM2 for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders. This will be done to comply with FDA Recommendations and NIH Guidelines for long-term follow-up for research involving gene therapy with AAV Vectors.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects with Tay-Sachs or Sandhoff Disease who were previously treated with AXO-AAV-GM2', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects who were previously enrolled in the study "A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal thalamic and intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease."\n\nExclusion Criteria:\n\n* Subjects who were not previously enrolled under the initial protocol.'}, 'identificationModule': {'nctId': 'NCT06614569', 'briefTitle': 'Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease', 'organization': {'class': 'OTHER', 'fullName': 'University of Massachusetts, Worcester'}, 'officialTitle': 'Long-Term Follow-Up of A Two-Stage Dose-Escalation Study to Evaluate the Safety and Efficacy of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Admin of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease', 'orgStudyIdInfo': {'id': 'STUDY00001894'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease', 'description': 'All subjects have previously received gene therapy and will be followed for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders following AAV gene therapy.', 'interventionNames': ['Drug: AXO-AAV-GM2']}], 'interventions': [{'name': 'AXO-AAV-GM2', 'type': 'DRUG', 'description': 'Participants who received AXO-AAV-GM2 will be tracked in long term follow up', 'armGroupLabels': ['Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease']}]}, 'contactsLocationsModule': {'locations': [{'zip': '01655', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'UMass Chan Medical School', 'geoPoint': {'lat': 42.26259, 'lon': -71.80229}}], 'overallOfficials': [{'name': 'Terence Flotte, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'UMass Chan Medical School'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Terence Flotte', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor and Dean', 'investigatorFullName': 'Terence Flotte', 'investigatorAffiliation': 'University of Massachusetts, Worcester'}}}}