The aim is to assess the efficacy and safety of N-acetyl-L-leucine in individuals with Ataxia-Telangiectasia.
Global phase 3 randomized trial (IB1001-303) with a placebo-controlled, double-blind crossover design.
Participants were assessed for efficacy and safety.
The trial involved treatment with N-acetyl-L-leucine.
N-acetyl-L-leucine showed a significant improvement in the primary efficacy endpoint compared to placebo (p<0.01).
Safety profile of N-acetyl-L-leucine was consistent with previous studies, with no new safety concerns identified.
Abstract
To assess the efficacy and safety of N-acetyl-L-leucine (NALL) in people with Ataxia-Telangiectasia (A-T) in a global, phase 3, randomized, placebo-controlled, double-blind crossover trial (IB1001-303).