New hope for rare overgrowth disorder: daily pill could tame symptoms
NCT ID NCT06997588
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a daily oral drug called alpelisib in people aged 2 and older with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. The goal is to see if the drug can shrink overgrown tissue and improve symptoms. About 104 participants will take the drug daily, and doctors will monitor changes using MRI scans.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alpelisib (BYL719), a daily oral drug
What this could lead to
If it works, this could provide a treatment to shrink overgrown tissue and manage symptoms in people with PROS, potentially improving daily life.
What could go wrong
This is a mid-stage trial with only 104 participants, so results may not apply to everyone. The drug may cause side effects and might not work for all types of overgrowth.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Baylor College Of Medicine
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Childrens Hosp Pittsburgh UPMC
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Cinn Children Hosp Medical Center
RECRUITINGCincinnati, Ohio, 45229-3039, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Novartis Investigative Site
RECRUITINGSydney, New South Wales, 2010, Australia
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Novartis Investigative Site
RECRUITINGSydney, New South Wales, 2031, Australia
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Novartis Investigative Site
RECRUITINGNorth Adelaide, South Australia, 5066, Australia
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Novartis Investigative Site
RECRUITINGSalzburg, 5020, Austria
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Novartis Investigative Site
RECRUITINGVienna, A 1090, Austria
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Novartis Investigative Site
RECRUITINGGhent, 9000, Belgium
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Novartis Investigative Site
RECRUITINGAngers, 49933, France
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Novartis Investigative Site
RECRUITINGBron, 69677, France
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Novartis Investigative Site
RECRUITINGDijon, 21000, France
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Novartis Investigative Site
RECRUITINGMontpellier, 34295, France
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Novartis Investigative Site
RECRUITINGParis, 75015, France
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Novartis Investigative Site
RECRUITINGToulouse, 31400, France
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Novartis Investigative Site
RECRUITINGTours, 37044, France
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Novartis Investigative Site
RECRUITINGFreiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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Novartis Investigative Site
RECRUITINGDüsseldorf, North Rhine-Westphalia, 40225, Germany
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Novartis Investigative Site
RECRUITINGLeipzig, Saxony, 04103, Germany
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Novartis Investigative Site
RECRUITINGHalle, Saxony-Anhalt, 06120, Germany
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Novartis Investigative Site
RECRUITINGBerlin, 13353, Germany
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Novartis Investigative Site
RECRUITINGStuttgart, 70374, Germany
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Novartis Investigative Site
RECRUITINGRoma, RM, 00168, Italy
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Novartis Investigative Site
RECRUITINGTorino, TO, 10126, Italy
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Novartis Investigative Site
RECRUITINGTrieste, TS, 34137, Italy
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Novartis Investigative Site
RECRUITINGEsplugues, Barcelona, 08950, Spain
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Novartis Investigative Site
RECRUITINGA Coruña, 15006, Spain
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Novartis Investigative Site
RECRUITINGBarcelona, 08035, Spain
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Novartis Investigative Site
RECRUITINGMadrid, 28046, Spain
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Novartis Investigative Site
RECRUITINGLausanne, 1011, Switzerland
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Novartis Investigative Site
RECRUITINGLiverpool, L12 2AP, United Kingdom
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UNC Chapel Hill
RECRUITINGChapel Hill, North Carolina, 27599, United States
Contact
Contact Email: •••••@•••••
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
Contact
Contact Email: •••••@•••••