Drug shows promise for rare overgrowth disorder in long-term study
NCT ID NCT04980833
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at the long-term safety and effectiveness of the drug alpelisib in people with PIK3CA-Related Overgrowth Spectrum (PROS), a rare condition causing abnormal tissue growth. It includes 41 children and adults who previously took alpelisib in an earlier trial. Researchers will track side effects and how well the drug controls overgrowth over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alpelisib
What this could lead to
If successful, this could confirm alpelisib as a long-term treatment option to control overgrowth in PROS, improving quality of life.
What could go wrong
This is a small, phase 2 study with no placebo group, so results may not be definitive. Side effects like high blood sugar or rash are possible.
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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Locations
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Boston Childrens Hospital
Boston, Massachusetts, 02215, United States
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Novartis Investigative Site
Dijon, 21000, France
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Novartis Investigative Site
Montpellier, 34295, France
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Novartis Investigative Site
Paris, 75015, France
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Novartis Investigative Site
Dublin, 12, Ireland
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Novartis Investigative Site
Madrid, 28046, Spain