A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2022
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.
Epistemonikos ID: 1d0d30b1ea8a97d24e88365745b9da1247bc90f1
First added on: May 09, 2024