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.
Disclaimer
Read more
Show less
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.
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States