Actively Recruiting

Phase 2
Age: 2Years +
All Genders
ID05983159

Trial of Targeted Oral Therapies Alpelisib and Mirdametinib for Slow-Flow or Fast-Flow Vascular Malformations Evaluating 48-Week Treatment and Follow-Up

Led by Murdoch Childrens Research Institute · Updated on 2026-05-05

50

Participants Needed

2

Research Sites

137 weeks

Total Duration

On this page

Sponsors

M

Murdoch Childrens Research Institute

Lead Sponsor

P

Peter MacCallum Cancer Centre, Australia

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating targeted therapies for patients with vascular malformations, focusing on those whose conditions are resistant to standard treatments or when standard therapies are unsuitable. The study is a modular, open-label, phase II trial that classifies vascular malformations into slow-flow or fast-flow types based on clinical criteria. Genetic testing is used to identify specific gene mutations affecting either the PI3K or MAPK signaling pathways, which drive the growth of these malformations. The trial has two treatment modules over a 48-week period followed by 24 weeks of follow-up. Module 1 involves oral alpelisib therapy for patients with slow-flow vascular malformations linked to PI3K pathway mutations. Module 2 involves oral mirdametinib therapy for patients with fast-flow vascular malformations related to MAPK pathway mutations. Both treatments are taken as monotherapy and aim to assess the clinical effectiveness based on genetic mutation status. Participants will undergo genetic testing, clinical evaluations, and regular monitoring throughout the treatment and follow-up periods. Researchers will measure improvements in symptoms using the Vascular Malformation Patient Specific Outcome Measure (VM-PSOM) at 48 weeks to determine treatment response. Safety and adherence to therapy will be closely observed, ensuring participants comply with study protocols for the entire duration, which can last up to 72 weeks including follow-up.

CONDITIONS

Official Title

A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 2 years or older
  • Clinical diagnosis of slow-flow or fast-flow vascular malformation
  • Received standard therapy or standard therapy is not appropriate
  • Documented genetic mutation in the PI3K pathway for slow-flow or MAPK pathway for fast-flow malformations
  • Adequate performance status (ECOG 0-2 for adults or Lansky > 50 for children under 16)
  • Life expectancy of at least 12 weeks
  • Ability to swallow and retain oral medication
  • Adequate blood counts and organ function as specified
  • Agreement to use effective contraception if of childbearing potential
  • Signed informed consent and willingness to comply with study procedures
Not Eligible

You will not qualify if you...

  • Allergy to study drugs or their components
  • Severe infection requiring IV antibiotics within 4 weeks before enrollment
  • Major surgery within 4 weeks before enrollment
  • Prior use of the study drug class (PI3K inhibitor for alpelisib module, MEK inhibitor for mirdametinib module)
  • Pregnancy or breastfeeding
  • Diabetes requiring medication or HbA1c above 6.4% (alpelisib module)
  • Use of medications that interact with study drugs and cannot be stopped
  • History of certain lung diseases like pneumonitis
  • Significant heart disease or recent cardiac events
  • Uncontrolled high blood pressure
  • Known HIV infection
  • Certain severe skin reactions history
  • Impaired gastrointestinal function affecting drug absorption
  • Recent or current malignancies (fast-flow module)
  • Retinal or eye conditions posing risk (fast-flow module)
  • Other severe uncontrolled medical conditions contraindicating study drugs
  • Inability to understand or comply with treatment instructions

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 2 locations

1

Peter MacCallum Cancer Centre

Parkville, Victoria, Australia, 3052

Actively Recruiting

2

The Royal Children's Hospital

Parkville, Victoria, Australia, 3052

Actively Recruiting

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Research Team

M

Michelle de Silva, PhD

T

Tony Penington, MBBS, FRACS

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

Frequently Asked Questions

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Published Research Related To This Trial

The Public Health Burden of Lymphatic Malformations in Children: National Estimates in the United States, 2000-2009.

Jeffrey Cheng, Beiyu Liu, Alfredo E Farjat...

https://pubmed.ncbi.nlm.nih.gov/28759318

Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations: a monocentric prospective phase II study.

Jennifer Hammer, Emmanuel Seront, Steven Duez...

https://pubmed.ncbi.nlm.nih.gov/30373605
Trial of Targeted Oral Therapies Alpelisib and Mirdametinib for Slow-Flow or Fast-Flow Vascular Malformations Evaluating 48-Week Treatment and Follow-Up | DecenTrialz