Search / Trial NCT06614569

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

Launched by TERENCE FLOTTE · Sep 25, 2024

Trial Information

Current as of December 22, 2024

Active, not recruiting

Keywords

ClinConnect Summary

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.

Gender

ALL

Eligibility criteria

  • 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 Officials

Terence Flotte, MD

Principal Investigator

UMass Chan Medical School

About Terence Flotte

Terence Flotte is a distinguished leader in the field of gene therapy and molecular medicine, serving as a clinical trial sponsor dedicated to advancing innovative therapeutic solutions. With a robust background in biomedical research and a commitment to improving patient outcomes, Flotte's initiatives focus on the development and evaluation of cutting-edge treatments for genetic disorders. His collaborative approach engages multidisciplinary teams and leverages state-of-the-art technologies, ensuring that clinical trials are conducted with the highest standards of scientific rigor and ethical integrity. Through his efforts, Flotte aims to translate groundbreaking research into effective therapies that address unmet medical needs.

Locations

Worcester, Massachusetts, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0