A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Status:
Not yet recruiting
Trial end date:
2024-04-01
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 Niemann Pick disease type C (NPC). The
objective of this study is to evaluate the safety, tolerability and efficacy of
N-acetyl-L-leucine (IB1001) compared to standard of care.