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Could a melanoma drug tame rare blood vessel disorders?

NCT ID NCT07549646

First seen Apr 25, 2026 · Last updated May 25, 2026 · Updated 3 times

Summary

This study tests whether trametinib, a drug already approved for melanoma, can help people with rare blood vessel growth problems caused by a specific genetic pathway. About 45 children and young adults (ages 2 months to 30 years) will take the drug for up to 3 years. The goal is to see if it shrinks abnormal vessels, improves symptoms, and enhances quality of life.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.