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
Overview
- Phase
- Not Applicable
- Intervention
- AXO-AAV-GM2
- Conditions
- GM2 Gangliosidosis
- Sponsor
- Terence Flotte
- Enrollment
- 7
- Locations
- 1
- Primary Endpoint
- Long term Safety of AXO-AAV-GM2
- Status
- Active, not recruiting
- Last Updated
- 6 months ago
Overview
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.
Investigators
Terence Flotte
Professor and Dean
University of Massachusetts, Worcester
Eligibility Criteria
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.
Arms & Interventions
Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease
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.
Intervention: AXO-AAV-GM2
Outcomes
Primary Outcomes
Long term Safety of AXO-AAV-GM2
Time Frame: 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 Outcomes
- Long term Impact of AXO-AAV-GM2(Every 6 months, up to five years)