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

Plain-language summary

This study provides long-term follow-up of participants who previously received a gene therapy called AXO-AAV-GM2 for Tay-Sachs disease or Sandhoff disease. Only people who were enrolled in the earlier study where this therapy was given are eligible. Run by Terence Flotte, this study does not use clinical phases and is currently active but not recruiting new participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Terence Flotte

Intervention

AXO-AAV-GM2

Start date

9/17/2024

Estimated completion

12/1/2028

Countries

United States

Related genes

Locations

• Worcester, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.