Study of fosigotifator for vanishing white matter disease
Part of Brain & nervous system clinical trials.
This early-phase study tests fosigotifator in people with vanishing white matter disease (VWM). The goal is to see whether the treatment is safe and could affect how the disease works in the body and brain.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your legally authorized representative) can give informed consent, and there is a caregiver who can complete caregiver questionnaires
- You are age 6 months or older
- You have a confirmed VWM diagnosis from a doctor plus genetic testing and MRI results that match VWM
- Your symptoms or abilities fit the study’s VWM-related criteria (history of possible VWM symptoms, walking difficulty, or certain thinking test scores)
- If you can become pregnant or someone can become pregnant from sexual activity, you agree to effective birth control during the study and for a set time after
- If you recently changed VWM symptom medicines, are on other experimental treatments, have uncontrolled seizures, or are on breathing support (young children), you may not qualify
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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