Hope for rare overgrowth disease: first treatment trial

NCT ID NCT04316546

Summary

This study is testing whether a drug called miransertib can safely slow or stop the abnormal tissue overgrowth caused by Proteus syndrome, a rare and serious genetic condition. It will involve about 45 participants aged 3 and older who have a confirmed diagnosis. Participants will take a daily pill for up to 4 years while researchers monitor changes in their condition, pain levels, and quality of life.

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: •••••@•••••

  • Contact

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

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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

Conditions

Explore the condition pages connected to this study.