Cancer drug shows promise for rare blood vessel disorder in tiny trial
NCT ID NCT05125471
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This small, completed study tested the cancer drug cobimetinib (Cotellic) in just 3 people with extracranial arteriovenous malformations (AVMs), which are abnormal tangles of blood vessels. The goal was to see if the drug could shrink the AVM by at least 20% and to check for safety. Because only 3 people took part, the results are very preliminary and cannot tell us if the drug works for most patients.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Cobimetinib (Cotellic), a cancer drug taken by mouth
- What this could lead to
- If it works, this could point toward a new drug treatment for arteriovenous malformations (AVMs) that are hard to treat with surgery or other methods.
- What could go wrong
- This is a very early, tiny study with only 3 participants, so results may not apply to most people. The drug also has side effects, and it is not yet clear if it shrinks AVMs enough to be helpful.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ARTERIOVENOUS MALFORMATIONS (EXTRACRANIAL) are added.
By submitting, you agree to our Terms of use
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
Locations
-
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States