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

Terence Flotte

Status: Active, not recruiting

Conditions: Tay Sachs Disease; GM2 Gangliosidosis; Sandhoff Disease

Treatments: Drug: AXO-AAV-GM2

Study type: Observational

Funder types: Other

Identifiers

NCT06614569

STUDY00001894

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 7 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

7 participants in 1 patient group

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

Description:

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.

Treatment:

Drug: AXO-AAV-GM2