Hope for rare overgrowth disorder: daily pill shows promise in early trial
NCT ID NCT04316546
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This phase 2 trial tests whether a daily pill called miransertib can slow or stop the abnormal tissue overgrowth seen in Proteus syndrome, a rare genetic condition. About 38 people aged 3 and older will take the drug for up to 4 years. Researchers will measure changes in foot overgrowth, pain, and quality of life to see if the treatment is safe and effective.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
miransertib (MK-7075), a daily pill
What this could lead to
If it works, this could point toward a treatment that slows or stops the abnormal overgrowth in Proteus syndrome, improving quality of life.
What could go wrong
This is a small, early-phase trial with only 38 participants. The drug may not work, could have side effects, and results may not apply to everyone with the condition.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States