Could a simple amino acid ease rare brain disorders?
NCT ID NCT07221292
First seen Oct 31, 2025 · Last updated May 15, 2026 · Updated 23 times
Summary
This study tests an experimental drug, N-acetyl-L-leucine, for people with rare genetic conditions caused by changes in the CACNA1A gene. These conditions can cause problems with movement, balance, and severe migraines. The study will include 60 people aged 4 and older, and will compare the drug to a placebo to see if it improves symptoms like walking and coordination.
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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.
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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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The University of Texas Health (UT Health)
Houston, Texas, 77030, United States
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University Hospital Bern Inselspital
Bern, 3010, Switzerland
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University of Cologne
Cologne, 50937, Germany
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University of Giessen
Giessen, 35389, Germany
Conditions
Explore the condition pages connected to this study.