New drug trial offers hope for rare brain disease
NCT ID NCT05757141
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times
Summary
This study tests an experimental drug, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 weeks and have regular check-ups, blood tests, and questionnaires.
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This is a summary of
the original study
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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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
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