N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)
Status:
Active, not recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
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.