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Louis Bar Syndrome clinical trials

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NCT ID: NCT03759678 Recruiting - Clinical trials for Ataxia Telangiectasia

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Start date: January 8, 2020
Phase: Phase 2
Study type: Interventional

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 Ataxia-Telangiectasia (A-T). 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) for the symptomatic treatment of A-T. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.