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New drug trial offers hope for rare, disfiguring growth disorder

NCT ID NCT04589650

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

Summary

This study is testing whether the drug alpelisib can safely reduce abnormal tissue overgrowth in people with a rare genetic condition called PROS. It involves about 200 children and adults. For the first 16 weeks, some participants receive the drug while others receive a placebo, and then all receive the active drug for up to five years to see if it shrinks lesions and improves symptoms.

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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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, 45229-3039, 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

Conditions

Explore the condition pages connected to this study.

PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)