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PIK3CA Related Overgrowth Syndromes (PROS) Clinical Trial

This is a study to understand how safe and effective a medication (alpelisib) is in patients with PIK3CA Related Overgrowth Syndromes (PROS). We will be studying adults and children who are at least 6 years of age.

Age & Gender

  • 6 years ~ 99 years
  • Male, Female, Gender Inclusive

Visit Availability

  • Standard business hours (M-F, 8-5)
  • Visits can be combined with regular clinical appointments


Georgia, North Carolina (Statewide), South Carolina, Tennessee, Virginia, West Virginia

What will be asked of you

At each visit you (or your child) will meet with members of the study team. Visits for the first year are monthly then every 6 months for the rest of the study. Visits will take anywhere from 2 hrs to 2 days as an outpatient and may include talking, physical examination, questionnaires, blood draws, studies of heart and lung function (EKGs, echocardiograms, lung function tests), MRI scans and photographs of your (or your child's) body, and other X-rays.


Gas and parking assistance, study drug at no cost for the duration of the study, meals for long clinic visits, hotel assistance for 2-day visits.

In-person visits:
Total length of participation:
5 years

Looking for Specific Volunteers

Able to participate:

  • Your (or your child's) doctor has told you that you have PROS
  • You (or your child) has had this confirmed by genetic testing from a biopsy.

Not eligible if:

  • You are a pregnant or nursing female
  • You are a sexually active male and not willing to use contraception
  • You have had alpelisib before or are on other "targeted" drugs for PROS such as sirolimus
  • You are not able to keep frequent appointments

Contact the Team

Visit Location

Contact & Visit Location

Primary Contact

Primary Visit Location

Clinical and Translational Research Center (CTRC)
Burnett-Womack Building (UNC-Chapel Hill)
160 Dental Cir, Chapel Hill, NC 27514, USA

Additional Study Information

Principal Investigator

Julie Blatt
Pediatrics - Hematology/Oncology

Study Type

Clinical or Medical

Study Topics

Rare Diseases

IRB Number




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