N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
Completed · Phase 2
Conditions studied: GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
In brief
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the symptomatic treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of GM2 Gangliosidosis. The Extension Phase was considered exploratory.
Key facts
- Study ID
- NCT03759665
- Run by
- IntraBio Inc
- People needed
- 30
- Starts
- 2019-06-07
- Expected to finish
- 2023-01-09
- Last updated by the study team
- 2024-03-12
Who can join
Age: 6 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California - Los Angeles — Los Angeles, California, United States
- Mayo Clinic — Rochester, Minnesota, United States
- NYU Langone School of Medicine — New York, New York, United States
- University of Giessen — Giessen, Germany
- Ludwig Maximilian University of Munich — München, Germany
- Bellvitge University Hospital — Barcelona, Spain
- Salford Trust — Salford, Greater Manchester, United Kingdom
- Royal Manchester Children's Hospital — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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