An observational study in GM2 Gangliosidosis, Tay Sachs Disease and Sandhoff Disease, sponsored by Terence Flotte. Active, not recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-10-16.
Sponsored by Terence Flotte · Observational
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.
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.
26 studies on the registry are indexed under Gangliosidoses, GM2; 6 are open to participants now.
This study's planned enrollment of 7 is below the median of 46 across 10 observational studies indexed under Gangliosidoses, GM2.
Browse Gangliosidoses, GM2 studies →Terence Flotte is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Subjects with Tay-Sachs or Sandhoff Disease who were previously treated with AXO-AAV-GM2
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Exclusion Criteria:
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.
Drug: AXO-AAV-GM2
Participants who received AXO-AAV-GM2 will be tracked in long term follow up
Long term Safety of AXO-AAV-GM2
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.
Time frame: Every 6 months, up to five years
Long term Impact of AXO-AAV-GM2
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.
Time frame: Every 6 months, up to five years
Plan to share: No
This study is active, not recruiting, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.
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Terence Flotte