OncoMatch/Clinical Trials/NCT04316546
MK-7075 (Miransertib) in Proteus Syndrome
Is NCT04316546 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies MK-7075 (miransertib) for proteus syndrome.
Treatment: MK-7075 (miransertib) — 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. Participants will have 20-30 visits at the NIH....
Check if I qualifyExtracted eligibility criteria
Biomarker criteria
Required: AKT1 mutation
Prior therapy
Cannot have received: major surgery
Major surgery ... within four weeks of the first dose of miransertib
Cannot have received: radiotherapy
radiotherapy ... within four weeks of the first dose of miransertib
Cannot have received: chemotherapy
chemotherapy ... within four weeks of the first dose of miransertib
Cannot have received: immunotherapy
immunotherapy within four weeks of the first dose of miransertib
Cannot have received: experimental systemic therapy (sirolimus, everolimus, high dose steroids, alpelisib)
Exception: except for participants who were previously or are currently treated with miransertib under a Compassionate Use/Expanded Access program or existing protocol
Any experimental systemic therapy for the purpose of treating Proteus syndrome (e.g., sirolimus, everolimus, high dose steroids, alpelisib) within two weeks of the first dose of miransertib, except for participants who were previously or are currently treated with miransertib under a Compassionate Use/Expanded Access program or existing protocol
Lab requirements
Blood counts
Hemoglobin >=10.0 g/dL; Glycated hemoglobin (HbA1c) <=8%; ANC >=1.5 x 10^9/L; Platelet count >=150 x 10^9/L
Kidney function
Serum creatinine depending on age: 2-5 years <=0.50 mg/dL; 6-10 years <=0.59 mg/dL; 11-15 years <=1.2 mg/dL; >15 years <=1.5 mg/dL
Liver function
Total bilirubin <=2 x ULN; AST and ALT <=3 x ULN
Adequate organ function as indicated by the following laboratory values: Hemoglobin (Hgb): >=10.0 g/dL; Glycated hemoglobin (HbA1c): <=8%; ANC >=1.5 x 10^9/L; Platelet count >=150 x 10^9/L; Total bilirubin <=2 x ULN; AST and ALT <=3 x ULN; Serum creatinine depending on age: 2-5 years <=0.50 mg/dL; 6-10 years <=0.59 mg/dL; 11-15 years <=1.2 mg/dL; >15 years <=1.5 mg/dL; Cholesterol <=400 mg/dL; Triglyceride <=500 mg/dL
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- National Institutes of Health Clinical Center · Bethesda, Maryland
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