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

Starting soon · Phase 3 · Has a placebo group

Conditions studied: CACNA1A, Spinocerebellar Ataxia Type 6, Episodic Ataxia Type 2, Familial Hemiplegic Migraine-1

In brief

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.

Key facts

Study ID

NCT07221292

Run by

IntraBio Inc

People needed

60

Starts

2026-09-01

Expected to finish

2028-11-01

Last updated by the study team

2026-06-04

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Johns Hopkins University — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• The University of Texas Health (UT Health) — Houston, Texas, United States
• Medical University of Innsbruck — Innsbruck, Austria
• University of Cologne — Cologne, Germany
• University of Athens — Athens, Greece
• University of Milan — Milan, Italy
• Bambino Gesu' Children's Research Hospital — Rome, Italy
• University Hospital Bern Inselspital — Bern, Switzerland
• University of Oxford - John Radcliffe Hospital — Oxford, United Kingdom

Full record on ClinicalTrials.gov

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