New hope for rare brain disorder: phase 3 trial tests potential treatment
NCT ID NCT07221292
Summary
This study aims to see if a medicine called N-Acetyl-L-Leucine (IB1001) is safe and can help improve movement, balance, and quality of life for people with rare genetic disorders caused by a CACNA1A gene mutation. About 60 participants, aged 4 and older, will take either the study medicine or a placebo for periods of 12 weeks each. Researchers will measure changes in symptoms like walking, speech, and hand coordination.
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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.