- Conditions
- GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2
- Drug
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Worcester, Massachusetts
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–2
of 2
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Tay-Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
- Biological
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Worcester, Massachusetts