TerminatedPhase 1NCT04669535
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Studying Tay-Sachs disease, juvenile form
Last synced from ClinicalTrials.gov
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Report missing dataKey facts
- Sponsor
- Terence Flotte
- Principal Investigator
- Terence Flotte, MDUniversity of Massachusetts Medical Health Center
- Intervention
- AXO-AAV-GM2 Starting Dose(biological)
- Enrollment
- 9 enrolled
- Eligibility
- 12 years · All sexes
- Timeline
- 2021 – 2024
Study locations (2)
- Massachusetts General Hospital, Center for Rare Neurological Diseases, Boston, Massachusetts, United States
- University of Massachusetts Medical Health Center, Worcester, Massachusetts, United States
Collaborators
University of Massachusetts, Worcester · Massachusetts General Hospital
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT04669535 on ClinicalTrials.gov