Active — Not RecruitingNot Applicable

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

United States7 participantsStarted 2024-09-17

Plain-language 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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Long term Safety of AXO-AAV-GM2

Timeframe: Every 6 months, up to five years

Trial details

NCT IDNCT06614569

SponsorTerence Flotte

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2028-12

View on ClinicalTrials.gov More GM2 Gangliosidosis trials