PIK3CA-related overgrowth spectrum
MONDO:1040002Any overgrowth syndrome resulting from pathogenic gain-of-function variants in the PIK3CA gene. The variants can be germline or somatic
Also known as: overgrowth syndrome and/or cerebral malformations due to abnormalities in MTOR pathway genes
19 clinical trials for this condition and its sub-types.
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Broader categories
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Could a pill shrink abnormal growths in PROS? new trial hopes to find out.
Disease control OngoingThis 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 leas…
Phase: PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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Drug shows promise for rare overgrowth disorder in long-term study
Disease control OngoingThis 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. Resear…
Phase: PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Sound waves instead of surgery: new device tested for vein disease
Disease control OngoingThis study tests whether a device called Sonablate can safely use high-intensity focused ultrasound (HIFU) to treat vein problems like varicose veins, leg ulcers, and certain birth defects. About 30 adults with these conditions will receive the treatment, which does not involve c…
Phase: NA • Sponsor: Sonablate • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC