Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

Active, not recruiting

• Conditions: GM2 Gangliosidosis; Tay Sachs Disease; Sandhoff Disease
• Interventions: Drug: AXO-AAV-GM2

• Registration Number: NCT06614569
• Lead Sponsor: Terence Flotte

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Status Verified: 2024-09
• Study Start: 2024-09-17
• Study First Submitted: 2024-09-23
• Study First Submitted QC: 2024-09-25
• Last Update Submitted: 2024-09-25
• Study First Posted: 2024-09-26
• Last Update Posted: 2024-09-26
• Primary Completion: 2028-12
• Study Completion: 2028-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 7

Inclusion Criteria

• 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."

Exclusion Criteria

• Subjects who were not previously enrolled under the initial protocol.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease	AXO-AAV-GM2	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.

Primary Outcome Measures

Name	Time	Method
Long term Safety of AXO-AAV-GM2	Every 6 months, up to five years	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.

Secondary Outcome Measures

Name	Time	Method
Long term Impact of AXO-AAV-GM2	Every 6 months, up to five years	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.

Trial Locations

Locations (1)

UMass Chan Medical School; 🇺🇸 Worcester, Massachusetts, United States