VASCULAR ANOMALIES
Clinical trials for VASCULAR ANOMALIES explained in plain language.
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Cancer drug shows promise for rare blood vessel disorders
Disease control OngoingThis phase 2 trial tests the drug trametinib in 45 people aged 2 months to 30 years with vascular anomalies caused by changes in the Ras/MAPK pathway. Participants take trametinib daily for up to 3 years. The study measures whether the drug shrinks lesions, improves quality of li…
Matched conditions: VASCULAR ANOMALIES
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Cancer drug shows promise for rare blood vessel disorders in kids and young adults
Disease control OngoingThis study tests the drug alpelisib, already approved for some breast cancers, in people aged 2 months to 30 years with certain blood vessel growth disorders caused by PIK3CA or TIE2 gene changes. Participants take the drug for 2-3 years and attend up to 16 clinic visits to check…
Matched conditions: VASCULAR ANOMALIES
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC