Hope for rare brain disease: Final-Stage trial tests promising pill
NCT ID NCT07221292
Summary
This study is testing if a medication called N-Acetyl-L-Leucine can help control symptoms and improve daily life for people with rare, inherited disorders caused by a faulty CACNA1A gene. These disorders affect coordination, balance, and speech. In this final-stage trial, 60 participants aged 4 and older will try the medication and a placebo in a random order to see which one works better.
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.