Dose Finding Trial of ARQ 092 in Children and Adults With Proteus Syndrome
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|ClinicalTrials.gov Identifier: NCT02594215|
Recruitment Status : Enrolling by invitation
First Posted : November 3, 2015
Last Update Posted : August 31, 2018
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 ARQ 092 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 ARQ 092 based on its effect on tissues in people with PS.
To determine the safety, tolerability, and recommended dose of ARQ 092 in people with PS.
People ages 6 and older with PS
Participants will be screened with medical history, physical exam, and blood and urine tests.
Participants will take ARQ 092 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.
|Condition or disease||Intervention/treatment||Phase|
|Proteus Syndrome||Drug: ARQ 092||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase 1 Dose Finding Trial of ARQ 092 in Children and Adults With Proteus Syndrome|
|Study Start Date :||October 31, 2015|
|Estimated Primary Completion Date :||July 1, 2025|
|Estimated Study Completion Date :||December 1, 2025|
Drug: ARQ 092
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 ARQ 092 in Proteus syndrome.
- Tissue drug levels [ Time Frame: End of cycle 3 ]Tissue drug levels are assessed at baseline, cycle 1 /day 14, and cycle 4/day 1.
- Tissue phospho-AKT level [ Time Frame: End of cycle 3 ]Tissue tissue phospho-AKT is assessed at baseline, cycle 1/day 14, and cycle 4/day 1.
- Tolerabilty/Side Effects [ Time Frame: Ongoing ]Tolerability and side effects are assessed on an ongoing basis.
- Tolerability and side effects [ Time Frame: Ongoing ]Tolerability and side effects are assessed on an ongoing basis.
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): NCT02594215
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Leslie G Biesecker, M.D.||National Human Genome Research Institute (NHGRI)|