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Active Not RecruitingNCT06614569

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

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 year...

Lead sponsor: Terence Flotte•1 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Terence Flotte

Subjects will be contacted bi-annually for a total of five years following the administration of AXO-AAV-GM2 for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders. This will be done to comply with FDA Recommendations and NIH Guidelines for long-term follow-up for research involving gene therapy with AAV Vectors.

Age

All ages

Sex

ALL

Healthy Volunteers

No

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.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: October 16, 2025Terms

Trial snapshot

StatusACTIVE_NOT_RECRUITING

Phase—

Enrollment7

Start dateSep 2024

Last updatedOct 16, 2025

Condition

GM2 Gangliosidosis Tay Sachs Disease Sandhoff Disease

Locations shown

UMass Chan Medical School

Worcester, Massachusetts

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 16, 2025Data synced to Clareo: May 10, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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