This study is testing N-acetyl-L-leucine (a modified amino acid) in people aged 4 and older with a genetically confirmed CACNA1A disorder, which includes conditions like Spinocerebellar Ataxia Type 6, Episodic Ataxia Type 2, and Familial Hemiplegic Migraine-1. The goal is to see if N-acetyl-L-leucine is safe and effective compared to a placebo (an inactive substance). Researchers will measure changes in ataxia (problems with coordination and balance) using a special scale over 24 weeks. This study is currently unclear regarding its recruitment status, but plans to enroll 60 participants.

Study design

This is a randomized, double-blind, placebo-controlled study where participants will receive N-acetyl-L-leucine and a placebo at different times. It is a multinational, cross-over Phase III study.

What's involved

You will participate in a baseline period of about 2 weeks, followed by two 12-week treatment periods where you will receive either N-acetyl-L-leucine or placebo. You will be assessed twice during each study period.

Compensation

Not stated in the trial record.

Follow-up

After the main study, participants may be offered the chance to join an Extension Phase to continue receiving N-acetyl-L-leucine.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.