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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

7 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-09-17

Study Completion Date

2028-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

NCT04669535

Tay-Sachs Disease Sandhoff Disease

TERMINATED PHASE1

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

NCT03759665

GM2 Gangliosidosis Tay-Sachs Disease Sandhoff Disease

COMPLETED PHASE2

Gene Therapy for Tay-Sachs Disease

NCT01869270

Tay Sachs Disease Sandhoff Disease Late Onset Tay Sachs Disease

COMPLETED

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

NCT04798235

Infantile GM2 Gangliosidosis (Disorder)

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429

Angelman Syndrome

ACTIVE_NOT_RECRUITING PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

GM2 Gangliosidosis Tay Sachs Disease Sandhoff Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Subjects previously treated with AXO-AAV-GM2 gene therapy for Tay-Sachs or Sandhoff Disease

All subjects have previously received gene therapy and will be followed for the collection of clinical information, especially pertaining to de novo cancer, neurologic, rheumatologic, and hematologic/immunologic disorders following AAV gene therapy.

AXO-AAV-GM2

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

AXO-AAV-GM2

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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."