Study of fosigotifator for vanishing white matter disease
Part of Brain & nervous system clinical trials.
This study tests a new medicine called fosigotifator for people with vanishing white matter disease (VWM), a rare genetic condition that affects the brain and movement. It aims to see if the medicine is safe and helpful for patients from infancy to adulthood.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Must be at least 6 months old (or younger for a special Canada-only group)
- Must have a diagnosis of vanishing white matter disease (VWM) confirmed by a doctor, genetic test, and brain MRI
- Must have had at least one symptom related to VWM (like trouble walking or thinking)
- Must have a caregiver who can attend visits and report symptoms
- All sexually active participants must agree to use birth control during the study
- Must not be on breathing support (for children under 6)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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