Pivotal Study of N-acetyl-L-leucine for CACNA1A

Authors
Category Primary study
Registry of TrialsClinicalTrials.gov
Year 2025
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. 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: 2afa3afa328c62e7b17b0e4c93843dba9be3f7f4
First added on: Oct 28, 2025