A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2028-06-01
Target enrollment:
Participant gender:
Summary
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.