First drug trial launched for devastating brain disease

NCT ID NCT05757141

Summary

This study is testing a new drug called fosigotifator for vanishing white matter disease, a rare and serious genetic brain disorder. It will enroll about 50 adults and children to see if the drug is safe, how the body handles it, and if it might help control the disease. This is an early-stage study, so the main goal is to gather information for future research.

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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Amsterdam UMC, locatie VUmc /ID# 270955

    RECRUITING

    Amsterdam, North Holland, 1081 HV, Netherlands

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Massachusetts General Hospital /ID# 270960

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • McGill University Health Centre - Glen Site

    RECRUITING

    Montreal, Quebec, H3H2L9, Canada

  • University of Utah /ID# 255624

    RECRUITING

    Salt Lake City, Utah, 84112-5339, United States

Conditions

Explore the condition pages connected to this study.