Clinical Trials / Research Studies

Clinical Trials / Research Studies

MOSAIC - A Phase 1/2 Study of ARQ 092 (Miransertib) in Subjects with PIK3CA-related Overgrowth Spectrum and Proteus Syndrome

Why are we doing this research?

The main reason for this research study is to learn more about PIK3CA-related overgrowth spectrum (PROS) disease or Proteus Syndrome (PS) and the investigational drug ARQ 092 as a possible treatment.

Who can participate?

You may take part in this study if you have PROS or PS.

Conditions

  • Hematology - Hemangioma and Vascular Malformation

What will happen in the study?

You will be treated with a new drug, ARQ 092 (Miransertib), as we assess its safety and efficacy by looking at overall growth and the pain associated with the lesion(s), your physical functioning, and your overall health.

We expect that you will be in this research study for at least 3 cycles (each cycle is 28 days). However, you may be in the study receiving study medication as long as a total of 48 cycles (about 48 months) if you are benefiting from the therapy in the opinion of the research doctor. 30 days after your last dose of the investigational drug, we will request you come in for one last visit for safety.

What are the good things that can happen from this research?

ARQ 092 may limit overgrowth lesion, which may help you feel better; however, taking part in this research study may or may not make your health better. We hope the information learned from this research study will help doctors learn more about ARQ 092 as a treatment in the future for patients with overgrowth diseases with PIK3CA or AKT1 mutations.

What are the bad things that can happen from this research?

The investigational drug used in this study may affect how different parts of your body work such as your liver, kidneys, heart, and blood, causing side effects. These will be followed throughout the study.

Will you/your child be paid to be in this research study?

You will not receive payment for taking part in this study. You may be reimbursed up to $158 per visit for your travel and meals, if needed, while you are in this research study. You may also be reimbursed for any airfare or hotel used throughout the duration of the study, depending on your distance.

Contact

Contact Us.Megan Metcalf and Nicholas Jakubowski
Email: HVMCresearch@cchmc.org
Phone Number: 513-803-4862