First drug trial launched for devastating brain disease in children and adults
NCT ID NCT05757141
Summary
This study is testing an experimental drug called fosigotifator for vanishing white matter disease, a rare genetic brain disorder that affects both children and adults. Researchers will give the drug to about 50 participants over 201 weeks to check if it's safe and how the body processes it, while also looking for early signs that it might help control the disease. The study includes participants as young as 6 months old who have been diagnosed with this condition.
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
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