Cancer drug shows promise for rare vascular conditions in kids and young adults
NCT ID NCT07543822
First seen Apr 23, 2026 · Last updated May 09, 2026 · Updated 3 times
Summary
This study tests the drug alpelisib (already approved for some breast cancers) in people aged 2 months to 30 years with rare blood vessel growth disorders caused by specific gene changes. Participants take the drug for at least 2 years, and researchers measure improvement using scans, symptom reports, and quality-of-life questionnaires. The goal is to see if the drug can shrink abnormal vessels and ease symptoms, though lifelong management may still be needed.
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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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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Conditions
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