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  • About Us
    • Who We Are
    • Mission Statement
    • Board of Directors
    • Medical Advisory Board
    • Partners
  • Patients & Families
    • About Proteus Syndrome
    • Clinical Trials
    • Genetic Testing
    • Resources
      • Videos
      • Documentary
      • Glossary
  • Researchers & Physicians
    • Proteus 101
    • Clinical Trials & Research Pipeline
    • Genetic Testing
    • Resources
  • Ways to Help
    • Donate
    • Fundraise
    • Family Assistance Program
    • The Jackie Johnson Sparkle Grant
  • Contact Us
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Clinical Trials

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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. 
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Genetic mutations can lead to many different diseases. Massachusett’s-based biotechnology company ArQule is currently conducting the MOSAIC clinical trial (Miransertib in Overgrowth Syndromes in Adults and Children) for patients who have been diagnosed with overgrowth disorders, including Proteus syndrome.

ArQule has designed the MOSAIC clinical trial to determine whether its drug candidate, miransertib, can effectively treat individuals with Proteus syndrome. Miransertib is a small molecule drug candidate that inhibits the AKT pathway. Miransertib is administered by mouth once a day. ArQule believes that by inhibiting the AKT pathway, miransertib can reduce cell overgrowth caused by the AKT1 mutation and therefore become a transformative treatment option for those individuals with Proteus syndrome and other overgrowth disorders.

Visit here to learn more. If you are interested in participating in the ArQule MOSAIC study, please email mosaicstudy@arqule.com.  


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The Proteus Syndrome Foundation, a 501c3 not-for-profit organization, is dedicated improving the lives of Proteus patients by funding AKT1 research. We focus on providing family support in the form of education and networking individuals living with Proteus syndrome with other families and medical professionals.

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Alex Hoag
In Loving Memory of
Alexander Hoag
7/5/90 - 9/20/99
"He did a lot in 9 short years."
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  • PSF Home
  • About Us
    • Who We Are
    • Mission Statement
    • Board of Directors
    • Medical Advisory Board
    • Partners
  • Patients & Families
    • About Proteus Syndrome
    • Clinical Trials
    • Genetic Testing
    • Resources
      • Videos
      • Documentary
      • Glossary
  • Researchers & Physicians
    • Proteus 101
    • Clinical Trials & Research Pipeline
    • Genetic Testing
    • Resources
  • Ways to Help
    • Donate
    • Fundraise
    • Family Assistance Program
    • The Jackie Johnson Sparkle Grant
  • Contact Us
  • Subscribe