New hope for rare blood vessel disorders: targeted drugs enter trial
NCT ID NCT05983159
First seen Feb 22, 2026 · Last updated Jun 23, 2026 · Updated 17 times
Summary
This phase 2 trial is testing two targeted drugs—alpelisib and mirdametinib—in people with vascular malformations (abnormal blood vessels). The study enrolls 50 participants aged 2 and older, split into two groups based on whether their malformations are slow-flow or fast-flow. Each participant's main symptom is tracked over 48 weeks to see if the drug leads to meaningful improvement.
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This is a summary of
the original study
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Peter MacCallum Cancer Centre
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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The Royal Children's Hospital
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
alpelisib and mirdametinib
What this could lead to
If successful, this could point toward a new treatment option for people with vascular malformations that are not helped by standard therapies.
What could go wrong
This is a small, early-phase trial with only 50 participants, so results may not apply to everyone. The drugs also have known side effects from cancer use, and the study is 'signal seeking'—meaning it's designed to find hints of effectiveness, not prove a cure.
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.