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Minor2 versionsRe-verified since May 20, 2026, no change to tracked fieldsv617-v618
MinorTrial status changed: Recruiting → Active, Not RecruitingEnrollment reduced: 45 → 38 participants · Site details revisedv616Show 2 more changesShow less
Enrollment reduced: 45 → 38 participants
Site details revised
Minor21 versionsRe-verified since Dec 19, 2025, no change to tracked fieldsv595-v615
MinorEligibility criteria revisedSecondary endpoints: details revised at 1 of 3 entries · Study description shortened, about 23 wordsv594Show 2 more changesShow less
Secondary endpoints: details revised at 1 of 3 entries
Minor5 versionsRe-verified since Nov 22, 2025, no change to tracked fieldsv589-v593
MinorPrimary completion date: Mar 31, 2026 to Jul 1, 2026Completion date: Mar 31, 2028 to Jul 1, 2028v588Show 1 more changeShow less
Completion date: Mar 31, 2028 to Jul 1, 2028
Minor9 versionsRe-verified since Jul 10, 2025, no change to tracked fieldsv579-v587
Stopped: an ongoing trial was terminated, suspended or withdrawn.
Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Background:
Proteus syndrome is a rare overgrowth disorder. Most people begin to have symptoms between 6 months and 2 years of age. There are very few living adults with this disease. There is also no known treatment for it. Researchers want to see if a new drug can slow down or stop overgrowth in people with Proteus syndrome.
Objective:
To learn if miransertib is a safe and effective treatment for Proteus syndrome.
Eligibility:
People ages 3 and older with Proteus syndrome.
Design:
Participants will be screened with a medical checkup. They will answer questions about their medical history and current health. They will have a physical exam with vital signs. They will have an electrocardiogram to measure their heartbeat. They will give blood and urine samples. They will repeat the screening tests during the study.
Participants will take a miransertib pill once a day. They will bring their empty pill bottles with them to the NIH when they visit. If they can t swallow a pill, researchers will try to find other ways for them to take the drug.
Participants will have X-rays, ultrasounds, and imaging scans. Photos may be taken of their feet and other parts of the body that have or develop signs of Proteus syndrome.
Participants will have lung function tests to measure how much and how fast air moves out of their lungs.
Participants will complete surveys about their levels of pain, physical functioning, and quality of life.
Participants may have additional tests performed to assess their individual disease. They may have consultations with other specialists.
Participation lasts about 4 years with possibility of continued treatment beyond 4 years....