GM2 Gangliosidosis, Sandhoff Disease, Tay Sachs Disease
Conditions
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.
Interventions
DRUGAXO-AAV-GM2
Participants who received AXO-AAV-GM2 will be tracked in long term follow up
Sponsors
Terence Flotte
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
Outcome results
None listed