New drug trial offers hope for rare brain disease
NCT ID NCT05757141
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 33 times
Summary
This study tests an experimental drug called fosigotifator in people with Vanishing White Matter disease, a rare genetic brain disorder. About 50 adults, children, and infants will receive the drug to see if it is safe and how the body processes it. Researchers will also look for early signs that the drug might help control the disease.
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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.
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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Amsterdam UMC, locatie VUmc /ID# 270955
RECRUITINGAmsterdam, North Holland, 1081 HV, Netherlands
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Massachusetts General Hospital /ID# 270960
RECRUITINGBoston, Massachusetts, 02114, United States
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McGill University Health Centre - Glen Site
RECRUITINGMontreal, Quebec, H3H2L9, Canada
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University of Utah /ID# 255624
RECRUITINGSalt Lake City, Utah, 84112-5339, United States
Conditions
Explore the condition pages connected to this study.