Hope for rare overgrowth disorder: new drug trial launches
NCT ID NCT04316546
First seen Nov 01, 2025 · Last updated May 18, 2026 · Updated 33 times
Summary
This study tests whether the drug miransertib can safely slow or stop the abnormal tissue growth in people with Proteus syndrome, a rare condition that begins in early childhood. About 45 participants aged 3 and older will take a daily pill for up to 4 years, with regular checkups and imaging to track changes. The goal is to see if the drug can control the disease, not cure it, as ongoing management is needed.
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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.
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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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