Could a simple amino acid ease rare brain disorders?

NCT ID NCT07221292

Not yet recruiting Symptom relief Sponsor: IntraBio Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement and balance. The goal is to find a safe treatment that eases symptoms, not to cure the condition.

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Conditions

The condition(s) this trial relates to.

CACNA1A-related complex neurodevelopmental disorder developmental and epileptic encephalopathy, 42 episodic ataxia type 2 migraine, familial hemiplegic, 1 spinocerebellar ataxia type 6 Spinocerebellar Ataxias

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CACNA1A EPISODIC ATAXIA TYPE 2 FAMILIAL HEMIPLEGIC MIGRAINE-1 SPINOCEREBELLAR ATAXIA TYPE 6

Contacts and locations

Locations

Bambino Gesu' Children's Research Hospital

Rome, Italy
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Medical University of Innsbruck

Innsbruck, Austria
The University of Texas Health (UT Health)

Houston, Texas, 77030, United States
University Hospital Bern Inselspital

Bern, 3010, Switzerland
University of Athens

Athens, Greece
University of Cologne

Cologne, 50937, Germany
University of Milan

Milan, Italy
University of Oxford - John Radcliffe Hospital

Oxford, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.