Cancer drug shows promise for rare blood vessel disorders

NCT ID NCT07549646

Ongoing Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This 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 life, and provides clinical benefit. Trametinib is already approved for melanoma but is experimental for this use.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Trametinib (a targeted cancer drug taken daily as a pill)
What this could lead to: If it works, this could offer a new treatment option for people with certain vascular anomalies that are driven by the Ras/MAPK pathway.
What could go wrong: This is a small, early-phase trial (45 people) and trametinib is not yet approved for this condition. Side effects from the drug are possible, and not everyone may respond.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

RASopathy vascular malformation

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

RAS/MAPK PATHWAY VASCULAR ANOMALIES VASCULAR ANOMALIES VASCULAR ANOMALY

Contacts and locations

Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

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