Cancer drug shows promise for rare blood vessel disorders in kids

NCT ID NCT07549646

First seen Apr 25, 2026 · Last updated May 10, 2026 · Updated 1 time

Summary

This study tests the drug trametinib, already approved for melanoma, in people aged 2 months to 30 years with rare blood vessel growth problems caused by a specific gene pathway. The goal is to see if the drug can shrink abnormal vessels and improve quality of life. Participants take the drug for 2-3 years and attend up to 16 clinic visits.

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Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

Conditions

Explore the condition pages connected to this study.