Hope for rare overgrowth disorder: daily pill shows promise in early trial
NCT ID NCT04316546
First seen Jun 26, 2026 · Last updated Jul 17, 2026 · Updated 4 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.