CREE LEUKOENCEPHALOPATHY
Clinical trials for CREE LEUKOENCEPHALOPATHY explained in plain language.
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Last hope drug trial for kids with fatal brain disease
Disease control OngoingThis study gives one child with Cree Leukoencephalopathy (CLE) access to an experimental drug called fosigotifator. CLE is a rare, inherited brain disease that destroys white matter and leads to early death. The drug aims to slow or stop the damage, possibly easing symptoms and i…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Phase: NA • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated Jun 27, 2026 08:11 UTC
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Desperate patients get early access to experimental brain drug
Disease control NO_LONGER_AVAILABLEThis program offers early access to fosigotifator, an oral drug, for people with Vanishing White Matter or Cree Leukoencephalopathy—two rare, serious brain diseases. A doctor must decide if the potential benefit outweighs the risks for each patient. The drug is not yet approved, …
Matched conditions: CREE LEUKOENCEPHALOPATHY
Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 08:11 UTC
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Last hope: experimental drug tested in single child with rare brain disease
Disease control OngoingThis study gives one patient with Cree Leukoencephalopathy, a rare and fatal brain disease, access to an experimental drug called fosigotifator. The drug aims to slow or stop the brain damage that causes severe disability and early death. Researchers will track whether the patien…
Matched conditions: CREE LEUKOENCEPHALOPATHY
Phase: EARLY_PHASE1 • Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC