Actively Recruiting
A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations
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
Recent studies have demonstrated that growth of vascular malformations can be driven by genetic variants in one of 2 signalling pathways. Targeted drugs specific to these pathways have been developed and shown to be effective in treating cancer. This study will describe the effectiveness of (i) 48 weeks of alpelisib therapy for participants with slow-flow vascular malformations and a gene mutation in one of these signalling pathways (module 1) and (ii) 48 weeks of mirdametinib therapy for participants with fast-flow vascular malformations and a gene mutations in the other signalling pathway (module 2).
CONDITIONS
Official Title
A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations
Who Can Participate
Eligibility Criteria
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
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
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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
Research Team
M
Michelle de Silva, PhD
CONTACT
T
Tony Penington, MBBS, FRACS
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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