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

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).

NCT Number	NCT05983159
Lead Sponsor	Murdoch Childrens Research Institute Collaborators: Peter MacCallum Cancer Centre, Australia, Royal Children's Hospital
Conditions	Slow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous Malformation, Lymphatic Malformation, Low Flow, Lymphatic Malformation, Lymphangioma, Arteriovenous Malformations +10 more
Enrollment	50 participants
Start Date	2024-09-13
Primary Completion	2027-01 (estimated)
Study Completion	2027-05 (estimated)
Updated on ClinicalTrials.gov	2026-05-05