New hope for rare blood vessel disorders: targeted drugs enter trial
NCT ID NCT05983159
First seen Feb 22, 2026 · Last updated Apr 28, 2026 · Updated 9 times
Summary
This study tests two targeted drugs for people with vascular malformations (abnormal blood or lymph vessels). It includes 50 participants aged 2 and older. One group with slow-flow malformations and a specific gene mutation receives alpelisib for 48 weeks. Another group with fast-flow malformations and a different gene mutation receives mirdametinib. The goal is to see if these drugs improve each person's most bothersome symptom.
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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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