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, 15 tagged with PIK3CA-related overgrowth spectrum itself.
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Sub-types of PIK3CA-related overgrowth spectrum
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CLOVES syndrome 5 trials
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Cowden syndrome 5 0 trials
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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 2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Aug 20, 2026 00:00 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 2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC