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

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).

Conditions

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

Interventions

DRUG

Alpelisib

Oral alpha-specific PI3-kinase inhibitor

DRUG

Mirdametinib

An investigational oral MEK inhibitor

Sponsors & Collaborators

• Peter MacCallum Cancer Centre, Australia

  collaborator OTHER

• Royal Children's Hospital

  collaborator OTHER

• Murdoch Childrens Research Institute

  lead OTHER

Principal Investigators

• Tony Penington, MBBS, FRACS. · Study Principal Investigator

• Stephen Luen, MBChB, FRACP. · Principal Investigator

• Lydia Pathmanathan, MBBS, FRACP. · Principal Investigator

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

2 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2024-09-13

Primary Completion

2027-01-31

Completion

2027-05-31

FDA Drug

Yes

Countries

• Australia

Study Locations