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

ConditionsSlow-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

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age2+

SponsorMurdoch Childrens Research Institute

About this trial

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 criteria

Qualifiers

Adult or paediatric patient, 2 years of age or over

Patient has a clinical diagnosis of a slow-flow vascular malformation

Patient has received standard therapy for the vascular malformation or in which, in the opinion of the investigator, standard therapy is not appropriate

A documented genetic alteration in the PI3K signalling pathway identified by genetic sequencing prior to enrolment in this study

Disqualifiers

History of hypersensitivity to any drugs or metabolites of PI3K inhibitors or any of the excipients of alpelisib

Severe infection requiring intravenous antibiotics within 4 weeks prior to enrolment

Patient has had a major surgical procedure within 4 weeks prior to enrolment

Prior use of an alpha-specific PI3K inhibitor

Trial design

Treatments tested in this trial

Alpelisib
Mirdametinib

Treatment groups

50 Participants

are divided into 2 treatment groups

Sponsors and collaborators

Murdoch Childrens Research Institute

Lead sponsor

Peter MacCallum Cancer Centre, Australia

Collaborator

Royal Children's Hospital

Collaborator

Source ClinicalTrials.gov