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Dose Finding Trial of MK-7075 in Children and Adults With Proteus Syndrome

NCT02594215 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2022-06-30

No results posted yet for this study

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.

...

Conditions

  • Proteus Syndrome

Interventions

DRUG

MK-7075 (miransertib)

MK-7075 or miransertib (formerly ARQ 092) is small molecule that effectively inhibits AKT. Proteus syndrome is caused by mosaic activating mutations in AKT1. This is a phase I study to determine a recommended dose for subsequent trials, which will determine the efficacy of miransertib in Proteus syndrome.

Sponsors & Collaborators

  • National Human Genome Research Institute (NHGRI)

    lead NIH

Principal Investigators

  • Leslie G Biesecker, M.D. · National Human Genome Research Institute (NHGRI)

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-11-16
Primary Completion
2017-12-31
Completion
2022-06-24
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02594215 on ClinicalTrials.gov