New hope for rare blood vessel disorders: targeted drugs enter trial
NCT ID NCT05983159
First seen Feb 22, 2026 · Last updated May 12, 2026 · Updated 14 times
Summary
This study tests two targeted drugs for people with abnormal blood vessels (vascular malformations). One drug, alpelisib, is for slow-flow types; the other, mirdametinib, is for fast-flow types. About 50 participants aged 2 and older will take the drug for 48 weeks to see if their main symptom improves.
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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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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: •••••@•••••
Conditions
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