New drug shows promise for rare overgrowth disorder in kids and adults
NCT ID NCT04589650
First seen Jan 03, 2026 · Last updated May 08, 2026 · Updated 23 times
Summary
This study tests a drug called alpelisib in children and adults with PROS, a rare condition that causes abnormal growth of body parts. The goal is to see if the drug can shrink these overgrowths by at least 20%. Participants are randomly assigned to receive the drug or a placebo for 16 weeks, followed by longer-term treatment periods.
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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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Contacts and locations
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Locations
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Baylor College Of Medicine
Houston, Texas, 77030, United States
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CHOP Abramson Pediatric Resch Ctr
Philadelphia, Pennsylvania, 19104, United States
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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States
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Childrens Hospital and Regional Medical Center
Seattle, Washington, 98105, United States
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Cincinnati Children s Hospital Medical Center
Cincinnati, Ohio, 45229-3039, United States
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Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45206, United States
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Fink Childrens Ambulatory Care Ctr
New York, New York, 10016, United States
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Novartis Investigative Site
Montreal, Quebec, H2W 1T8, Canada
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Novartis Investigative Site
Montreal, Quebec, H3T 1C5, Canada
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Novartis Investigative Site
Beijing, 100730, China
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Novartis Investigative Site
Shanghai, 200011, China
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Novartis Investigative Site
Bordeaux, 33076, France
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Novartis Investigative Site
Dijon, 21000, France
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Novartis Investigative Site
Paris, 75015, France
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Novartis Investigative Site
Tours, 37044, France
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Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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Novartis Investigative Site
Düsseldorf, North Rhine-Westphalia, 40225, Germany
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Novartis Investigative Site
Leipzig, Saxony, 04103, Germany
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Novartis Investigative Site
Hamburg, 22149, Germany
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Novartis Investigative Site
Heidelberg, 69120, Germany
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Novartis Investigative Site
Hong Kong, 999077, Hong Kong
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Novartis Investigative Site
Roma, RM, 00165, Italy
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Novartis Investigative Site
Torino, TO, 10126, Italy
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Novartis Investigative Site
Nijmegen, Gelderland, 6500HB, Netherlands
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Novartis Investigative Site
Oslo, 0372, Norway
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Novartis Investigative Site
Esplugues, Barcelona, 08950, Spain
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Novartis Investigative Site
Madrid, 28046, Spain
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Novartis Investigative Site
Bern, 3010, Switzerland
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Novartis Investigative Site
Zurich, 8032, Switzerland
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Novartis Investigative Site
West Midlands, Birmingham, B4 6NH, United Kingdom
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Novartis Investigative Site
London, SW17 0QT, United Kingdom
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Novartis Investigative Site
Manchester, M13 9WL, United Kingdom
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UCSF Birthmarks and Vascular Center
San Francisco, California, 94158, United States
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UNC Chapel Hill
Chapel Hill, North Carolina, 27599, United States
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Unv of TX Southwestern Medical Center
Dallas, Texas, 75235, United States
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Washington Univ School Of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.