NCT06614569

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

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 7 (estimated)
• Sponsor: Terence Flotte · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• GM2 Gangliosidosis
• Tay Sachs Disease
• Sandhoff Disease

Interventions

Type	Name	Description
DRUG	AXO-AAV-GM2	Participants who received AXO-AAV-GM2 will be tracked in long term follow up

Timeline

• Start date: 2024-09-17
• Primary completion: 2028-12-01
• Completion: 2028-12-01
• First posted: 2024-09-26
• Last updated: 2025-10-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study