Current Research Studies

Vascular Anomalies: Miransertib in PI3KCA Overgrowth Syndromes in Adults and Children - MOSAIC

Condition or Therapy:

PI3KCA overgrowth, lymphatic malformation with overgrowth, Proteus Syndrome 

Category:

Vascular Anomalies

What is the goal of this study?

This is an open-label study to determine whether miransertib can effectively treat individuals with Proteus syndrome (PS) and PI3KCA related overgrowth (PROS).

Who can join the study?

This study may be good for individuals who:

  • Are over 2 years of age
  • Have a clinical diagnosis of PROS or PS
  • Have a documented mutation in either the AKT1 or PI3KCA gene

What will happen if my child takes part in this study?

If you/your child agree to take part in this study, then you/your child can expect to:

  • Take a pill by mouth once daily
  • Come into the hospital 7 times for the first 6 months, every other month for the rest of the first year, and every 3 months for as long as treatment is being given
  • Fill out surveys about quality of life at certain visits
  • Give blood samples at visits to monitor safety

Who can I contact for more information?

To learn more, call 206-987-1927 or email the study coordinator.


Study Location(s):

Seattle Children's Main Hospital Campus and Bellevue Clinic and Surgery Center

Principal Investigator:

Dr. Jonathan Perkins
Research Center: Center for Clinical and Translational Research