Dose Finding Trial of MK-7075 in Children and Adults With Proteus Syndrome
Status:
Active, not recruiting
Trial end date:
2025-12-01
Target enrollment:
Participant gender:
Summary
Background:
Proteus syndrome (PS) is caused by a mutation in the AKT1 gene. This gene makes a protein
that communicates with other proteins in the body to make cells grow. The AKT1 mutation
changes chemical signals in the body and causes overgrowth. PS can be fatal. The drug MK-7075
reduces signals from the AKT1 protein. This may reduce or stabilize some of the overgrowth in
people with PS. Researchers want to find the best dose of MK-7075 based on its effect on
tissues in people with PS.
Objective:
To determine the safety, tolerability, and recommended dose of MK-7075 in people with PS.
Eligibility:
People ages 6 and older with PS
Design:
Participants will be screened with medical history, physical exam, and blood and urine tests.
Participants will take MK-7075 by mouth once daily for up to 12 28-day cycles.
Participants must stay near the NIH Clinical Center (CC) during the whole first cycle, for
weekly visits to the CC. For cycle 2, they will have visits every 2 weeks. They will have 1
visit before cycles 3 and 4, and once before every other cycle for cycles 5 11. The final
visit will be at the end of cycle 12. Visits may include:
Small skin samples taken.
ECG: Soft electrodes on the skin record heart signals.
Echocardiogram: A small probe held to the chest takes pictures of the heart.
MRI: Participants will lie in a machine that takes pictures of the body.
Joint and mobility function tests.
Participants will complete surveys by phone and in person.
Participants will keep a daily medication and symptom diary.
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