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New hope for rare vascular malformations: targeted drugs enter trial

NCT ID NCT05983159

Recruiting now Disease control Sponsor: Murdoch Childrens Research Institute Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial is testing two targeted drugs—alpelisib and mirdametinib—in 50 people with slow-flow or fast-flow vascular malformations that haven't responded to standard treatments. The goal is to see if these drugs can improve each person's most bothersome symptom over 48 weeks. The study uses a personalized approach, measuring success based on individual symptom improvement.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Alpelisib (for slow-flow malformations) and mirdametinib (for fast-flow malformations)
What this could lead to
If successful, this could point toward a new treatment option for people with vascular malformations that don't respond to standard care.
What could go wrong
This is a small, early-phase trial (50 people) designed to look for signals of effectiveness, not to prove a cure. The drugs may not work for all types of malformations, and side effects are possible.

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Conditions

The condition(s) this trial relates to.

arteriovenous hemangioma/malformation arteriovenous malformations of the brain cystic hygroma lymphangioma Lymphatic Abnormalities lymphatic malformation PIK3CA-related overgrowth spectrum skin vascular disease vascular malformation venous hemangioma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ARTERIOVENOUS MALFORMATION (AVM) ARTERIOVENOUS MALFORMATIONS CYSTIC HYGROMA FAST-FLOW VASCULAR MALFORMATION KRAS G12C KRAS G12D LYMPHANGIOMA LYMPHATIC MALFORMATION LYMPHATIC MALFORMATION, LOW FLOW MAP2K1 GENE MUTATION PI3K GENE MUTATION PIK3CA RELATED OVERGROWTH SPECTRUM SLOW-FLOW VASCULAR MALFORMATION VASCULAR ANOMALIES VASCULAR ANOMALY VASCULAR MALFORMATIONS VENOUS MALFORMATION VENOUS MALFORMATION, LOW FLOW

Contacts and locations

Locations

  • Peter MacCallum Cancer Centre

    RECRUITING

    Parkville, Victoria, 3052, Australia

  • The Royal Children's Hospital

    RECRUITING

    Parkville, Victoria, 3052, Australia

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