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Could a pill shrink abnormal growths in PROS? new trial hopes to find out.

NCT ID NCT04589650

Ongoing Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 study tests the drug alpelisib in 206 children and adults with PIK3CA-related overgrowth spectrum (PROS), a rare condition causing abnormal tissue growth. Participants receive either alpelisib or a placebo for 16 weeks to see if the drug can shrink growths by at least 20%. The goal is to find a safe and effective treatment for this condition.

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 drug taken by mouth

What this could lead to

If it works, alpelisib could become the first approved treatment to shrink abnormal tissue growth and improve symptoms in people with PROS.

What could go wrong

This is a mid-stage trial with only 206 participants, so results may not apply to everyone. The drug may cause side effects like high blood sugar or rash, and not all patients may respond.

Disclaimer Read more

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.

PIK3CA-related overgrowth spectrum

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)

Contacts and locations

Locations

  • Baylor College Of Medicine

    Houston, Texas, 77030, United States

  • CHOP Abramson Pediatric Resch Ctr

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Childrens Hospital and Regional Medical Center

    Seattle, Washington, 98105, United States

  • Cincinnati Children s Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Cinn Children Hosp Medical Center

    Cincinnati, Ohio, 45206, United States

  • Fink Childrens Ambulatory Care Ctr

    New York, New York, 10016, United States

  • Novartis Investigative Site

    Montreal, Quebec, H2W 1T8, Canada

  • Novartis Investigative Site

    Montreal, Quebec, H3T 1C5, Canada

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Shanghai, 200011, China

  • Novartis Investigative Site

    Bordeaux, 33076, France

  • Novartis Investigative Site

    Dijon, 21000, France

  • Novartis Investigative Site

    Paris, 75015, France

  • Novartis Investigative Site

    Tours, 37044, France

  • Novartis Investigative Site

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    Düsseldorf, North Rhine-Westphalia, 40225, Germany

  • Novartis Investigative Site

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    Hamburg, 22149, Germany

  • Novartis Investigative Site

    Heidelberg, 69120, Germany

  • Novartis Investigative Site

    Hong Kong, 999077, Hong Kong

  • Novartis Investigative Site

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    Nijmegen, Gelderland, 6500HB, Netherlands

  • Novartis Investigative Site

    Oslo, 0372, Norway

  • Novartis Investigative Site

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    Madrid, 28046, Spain

  • Novartis Investigative Site

    Bern, 3010, Switzerland

  • Novartis Investigative Site

    Zurich, 8032, Switzerland

  • Novartis Investigative Site

    West Midlands, Birmingham, B4 6NH, United Kingdom

  • Novartis Investigative Site

    London, SW17 0QT, United Kingdom

  • Novartis Investigative Site

    Manchester, M13 9WL, United Kingdom

  • UCSF Birthmarks and Vascular Center

    San Francisco, California, 94158, United States

  • UNC Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • Unv of TX Southwestern Medical Center

    Dallas, Texas, 75235, United States

  • Washington Univ School Of Medicine

    St Louis, Missouri, 63110, United States