MK-7075 (Miransertib) in Proteus Syndrome
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|ClinicalTrials.gov Identifier: NCT04316546|
Recruitment Status : Recruiting
First Posted : March 20, 2020
Last Update Posted : January 28, 2022
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.
The learn if miransertib is a safe and effective treatment for Proteus syndrome.
People ages 3 and older with Proteus syndrome
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.
|Condition or disease||Intervention/treatment||Phase|
|Proteus Syndrome||Drug: MK-7075 (miransertib)||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||45 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multi-Cohort Phase 2 Dose-Escalation Study of MK-7075 (Miransertib) in Proteus Syndrome|
|Estimated Study Start Date :||February 2, 2022|
|Estimated Primary Completion Date :||March 31, 2025|
|Estimated Study Completion Date :||March 31, 2028|
Experimental: MK-7075 (miransertib)
This is a single-arm study. All study participants will be taking the experimental drug, MK-7075 (miransertib).
Drug: MK-7075 (miransertib)
MK-7075 (miransertib) is a small molecule developed by ArQule Inc., a wholly owned subsidiary of Merck & Co., that effectively inhibits AKT. Proteus syndrome is caused by mosaic activating mutations in AKT1. This is a Phase 2 trial investigating the efficacy of miransertib as a treatment for adult and pediatric patients with Proteus syndrome.
- CCTN [ Time Frame: baseline, two years ]Change in CCTN involvement of the plantar surface from baseline will be used to classify each subject as either a responder or non-responder (binary) in the treated population. The primary endpoint is response rate (defined as (=< 5% increase in plantar involvement from baseline over two years). This will be assessed by blinded central photography review.
- Quality of life [ Time Frame: Periodically throughout the study (described in schedule of activities) ]Change from baseline in pain score (NRS-11), physical functioning (PROMIS), and quality of life (PedsQL)
- Long-term safety and tolerability [ Time Frame: Periodically throughout the study (described in schedule of activities) ]Periodic safety (e.g., physical examination, vital sign measurements, clinical laboratory tests, use of concomitant medications and collection of AE information) assessments.
- Duration of response [ Time Frame: Periodically throughout the study (described in schedule of activities) ]Duration of response is defined as the amount of time from first response signal to progression of CCTN involvement >5% over rolling two year intervals.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04316546
|Contact: Christopher A Ours, M.D.||(301) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Contact: Research Assistant Not Listed email@example.com|
|Principal Investigator:||Leslie G Biesecker, M.D.||National Human Genome Research Institute (NHGRI)|