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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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.