Hope for rare overgrowth disorder: new drug trial launches

NCT ID NCT04316546

First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 32 times

Summary

This study tests a daily pill called miransertib in people with Proteus syndrome, a rare condition causing abnormal tissue growth. The goal is to see if the drug can safely slow or stop this overgrowth. About 45 participants aged 3 and older will take the drug for up to 4 years, with regular checkups and scans to track progress.

Disclaimer Read more

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for PROTEUS SYNDROME are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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.