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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Alpelisib's Long-Term safety tracked in PIK3CA patients
Disease control Not yet recruitingThis study provides continued access to alpelisib for people who are already benefiting from it in a previous Novartis trial. It focuses on tracking long-term safety, including any side effects, in participants with PIK3CA-related overgrowth spectrum or certain breast cancers. Pa…
Phase: PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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New pill could shrink abnormal vessels in rare childhood disorders
Disease control Not yet recruitingThis Phase 3 trial tests an oral drug called KP-001 in 150 people aged 2 and older with venous malformations, lymphatic malformations, or KTS/CLOVES syndrome. Participants receive either KP-001 or a placebo for 24 weeks, then everyone gets the drug for another 28 weeks. The goal …
Phase: PHASE3 • Sponsor: Kaken Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC