The Phase 1/2 MOSAIC trial (NCT03094832) is currently the only ongoing interventional clinical trial for the treatment of Proteus syndrome. Massachusetts-based biotechnology company ArQule has designed the MOSAIC trial to determine whether its small molecule AKT inhibitor, miransertib, can effectively treat individuals with Proteus syndrome and other overgrowth spectrum disorders. To be eligible for the trial, individuals must have a documented mutation in the AKT1 gene. 
 
Preclinical research has demonstrated that miransertib suppresses PIK3CA/AKT1 mutant dependent kinase signaling and demonstrates anti-tumor activity in molecularly defined tumor models. Preliminary clinical data suggests that miransertib improves disease-related symptoms and objective condition assessments.

AIMProteus
             AKT Inhibitor Miransertib on Proteus Syndrome  - Targeted therapy at NIH
Update:  The AIM Proteus Trial is currently in IRB review status.
Approximate patient enrollment date for trial - Late Feb-March 2020

The NIH team is very excited about the opening of this phase 2 clinical trial. They will be hosting an Educational Information Session Webinar on Tuesday, January 21st at 11:00am eastern standard time. concerning:

• trial status 
• the next steps for enrollment 

We will post a link for the webinar soon. 

Questions about the NIH AIMProteus trial email here or call. ​