Cancer drug shows promise for rare blood vessel disorders
NCT ID NCT07549646
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.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States