A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations
Study Purpose
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).
Recruitment Criteria
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms |
No |
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies. |
Interventional |
Eligible Ages | 2 Years and Over |
Gender | All |
Trial Details
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries. |
NCT05983159 |
Phase
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use. |
Phase 2 |
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data. |
Murdoch Childrens Research Institute |
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study. |
Tony Penington, MBBS, FRACS.Stephen Luen, MBChB, FRACP.Lydia Pathmanathan, MBBS, FRACP. |
Principal Investigator Affiliation | Study Principal InvestigatorPrincipal InvestigatorPrincipal Investigator |
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial. |
Other |
Overall Status | Not yet recruiting |
Countries | Australia |
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied. |
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 |
Contact a Trial Team
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