MK-7075 (Miransertib) in Proteus Syndrome (NCT04316546) | Clinical Trial Compass
RecruitingPhase 2
MK-7075 (Miransertib) in Proteus Syndrome
United States45 participantsStarted 2022-05-20
Plain-language summary
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....
Who can participate
Age range3 Years – 99 Years
SexALL
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Inclusion criteria
✓. Total bilirubin \<=2 x upper limit of normal (ULN)
✓. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<=3 x ULN
✓. Serum creatinine depending on age:
✓Have at least one plantar CCTN that can accurately be measured by standardized photography. The CCTN is defined as a nevus with at least two gyri and three sulci affecting 10% - 70% of the total surface area of the foot.
✓Male or female participants age greater than or equal to 3 and less than or equal to 17 years old and BSA of greater than or equal to 0.33 m\^2
✓Participants previously treated with miransertib or currently receiving miransertib under Compassionate Use/Expanded Access or an existing trial (i.e., 16-HG-0014)
✓Male or female participants greater than or equal to 3 years old BSA of greater than or equal to 0.33 m\^2
Exclusion criteria
✕History of clinically significant cardiac disorders:
What they're measuring
1
CCTN
Timeframe: Baseline, two years
Trial details
NCT IDNCT04316546
SponsorNational Human Genome Research Institute (NHGRI)
-Myocardial infarction (MI) or congestive heart failure defined as Class II-IV per the New York Heart Association (NYHA) classification within six months of the first dose of miransertib (MI occurring \>6 months of the first dose of miransertib will be permitted)
✕-Grade 2 (per National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTCAE v 5.0\]) or worse conduction defect (e.g., right or left bundle branch block).
✕Major surgery, radiotherapy, chemotherapy, or immunotherapy within four weeks of the first dose of miransertib