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

NCT06614569 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 7

Last updated 2025-10-16

No results posted yet for this study

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.

Conditions

  • GM2 Gangliosidosis
  • Tay Sachs Disease
  • Sandhoff Disease

Interventions

DRUG

AXO-AAV-GM2

Participants who received AXO-AAV-GM2 will be tracked in long term follow up

Sponsors & Collaborators

  • Terence Flotte

    lead OTHER

Principal Investigators

  • Terence Flotte, MD · UMass Chan Medical School

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-17
Primary Completion
2028-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06614569 on ClinicalTrials.gov