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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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Peter MacCallum Cancer Centre

    RECRUITING

    Parkville, Victoria, 3052, Australia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • The Royal Children's Hospital

    RECRUITING

    Parkville, Victoria, 3052, Australia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.