MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP)
Clinical trials for MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP) explained in plain language.
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New drug targets root cause of rare overgrowth syndromes
Disease control Recruiting nowThis Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…
Matched conditions: MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP)
Phase: PHASE2 • Sponsor: Relay Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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Could a cancer drug help kids with rare brain disorder?
Disease control Recruiting nowThis phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, the…
Matched conditions: MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP)
Phase: PHASE2 • Sponsor: Centre Hospitalier Universitaire Dijon • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC