Could a simple amino acid ease rare brain disorders?

NCT ID NCT07221292

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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Bambino Gesu' Children's Research Hospital

    Rome, Italy

    Contact Email: •••••@•••••

    Contact

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

    Contact Email: •••••@•••••

    Contact

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Medical University of Innsbruck

    Innsbruck, Austria

    Contact Email: •••••@•••••

    Contact

  • The University of Texas Health (UT Health)

    Houston, Texas, 77030, United States

  • University Hospital Bern Inselspital

    Bern, 3010, Switzerland

  • University of Athens

    Athens, Greece

    Contact Email: •••••@•••••

    Contact

  • University of Cologne

    Cologne, 50937, Germany

  • University of Milan

    Milan, Italy

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

    Contact

  • University of Oxford - John Radcliffe Hospital

    Oxford, United Kingdom

    Contact Email: •••••@•••••

    Contact