RecruitingPhase 3NCT07221292

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

Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

Sponsor

IntraBio Inc

Enrollment

60 participants

Start Date

Aug 17, 2026

Study Type

INTERVENTIONAL

Conditions

Spinocerebellar Ataxia Type 6 CACNA1A Episodic Ataxia Type 2 Familial Hemiplegic Migraine-1

Summary

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.

Eligibility

Min Age: 4 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called N-Acetyl-L-Leucine and Placebo for people with cacna1a, episodic ataxia type 2, and other related conditions. The study is currently recruiting participants at 10 locations. People eligible for this study include aged 4 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGN-Acetyl-L-Leucine

N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)

OTHERPlacebo

Matching Placebo Sachet

Locations(11)

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

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, United Kingdom

University of Oxford - John Radcliffe Hospital

Oxford, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07221292

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