RecruitingPhase 2NCT05983159

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

A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)

Sponsor

Murdoch Childrens Research Institute

Enrollment

50 participants

Start Date

Sep 13, 2024

Study Type

INTERVENTIONAL

Conditions

PI3K Gene Mutation KRAS G12D Vascular Anomalies KRAS G12C Arteriovenous Malformations PIK3CA Related Overgrowth Spectrum Vascular Anomaly Vascular Malformations Lymphatic Malformation Lymphangioma Venous Malformation Slow-Flow Vascular Malformation Fast-Flow Vascular Malformation Lymphatic Malformation, Low Flow Venous Malformation, Low Flow Cystic Hygroma MAP2K1 Gene Mutation Arteriovenous Malformation (AVM)

Summary

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).

Eligibility

Min Age: 2 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Alpelisib and a drug called Mirdametinib for people with arteriovenous malformation (avm), arteriovenous malformations, and other related conditions. The study is currently recruiting participants at 2 locations. People eligible for this study include aged 2 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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