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

Running, not enrolling

Conditions studied: GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease

In brief

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.

Key facts

Study ID

NCT06614569

Run by

Terence Flotte

People needed

7

Starts

2024-09-17

Expected to finish

2028-12-01

Last updated by the study team

2025-10-16

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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."

You may not qualify if…

• Subjects who were not previously enrolled under the initial protocol.

Where it is running

• UMass Chan Medical School — Worcester, Massachusetts, United States

Full record on ClinicalTrials.gov

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