Miransertib for Proteus Syndrome
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does exclude those using certain experimental therapies or those with uncontrolled diabetes requiring regular medication. It's best to discuss your specific medications with the trial team.
What makes the drug Miransertib unique for treating Proteus Syndrome?
Miransertib is unique for treating Proteus Syndrome because it targets a specific pathway involved in the overgrowth of tissues, which is a hallmark of this condition. Unlike other treatments, it directly inhibits the AKT1 enzyme, which is part of the signaling pathway that contributes to the abnormal cell growth seen in Proteus Syndrome.12345
What is the purpose of this trial?
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....
Research Team
Leslie G Biesecker, M.D.
Principal Investigator
National Human Genome Research Institute (NHGRI)
Eligibility Criteria
This trial is for people aged 3 and older with Proteus syndrome, a rare overgrowth disorder. Participants must have specific blood values within normal ranges, not use tobacco or certain drugs, and agree to contraception if of childbearing potential. They can't join if they have uncontrolled diabetes, significant heart issues, active infections, or intolerance to AKT inhibitors like miransertib.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive miransertib in continuous, 28-day cycles, with dose escalation based on tolerance
Follow-up
Participants are monitored for safety and effectiveness after treatment, with a final clinical safety follow-up 30 days after the last dose
Long-term monitoring
Participants may continue to be monitored for long-term safety and effectiveness
Treatment Details
Interventions
- Miransertib (ARQ092)
- MK-7075 (Miransertib)
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Human Genome Research Institute (NHGRI)
Lead Sponsor