Last hope: experimental drug tested on single child with rare brain disease
NCT ID NCT07300397
First seen Jan 11, 2026 · Last updated May 24, 2026 · Updated 17 times
Summary
This study gives one child with Cree Leukoencephalopathy (CLE), a severe and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the breakdown of the brain's white matter. Researchers will monitor if the benefits outweigh the risks and if the child's symptoms and quality of life improve.
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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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Locations
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McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
Conditions
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