A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (NCT05983159) | Clinical Trial Compass
RecruitingPhase 2
A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations
Australia50 participantsStarted 2024-09-13
Plain-language 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).
Who can participate
Age range2 Years
SexALL
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Inclusion criteria
✓. 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
✓. Adequate performance status (Eastern Cooperative Oncology Group Performance Status Scale (ECOG) 0-2 in patients ≥ 16 years of age; Lansky \> 50 in patients \< 16 years of age)
✓. Patient has a life expectancy ≥ 12 weeks
✓. Patient is able to swallow and retain oral medication
✓. Adequate haematologic and end-organ function:
Exclusion criteria
✕. History of hypersensitivity to any drugs or metabolites of PI3K inhibitors or any of the excipients of alpelisib
What they're measuring
1
The number of participants who achieve or exceed a predetermined threshold of improvement in their most significant symptom at 48 weeks based on the Vascular Malformation Patient Specific Outcome Measure (VM-PSOM).
✕. 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
✕. History of pneumonitis or interstitial lung disease
✕. Patient is pregnant or lactating at the time of study registration. A pregnancy test is mandated for women of child-bearing potential in the screening period
✕. Established diagnosis of type I diabetes mellitus, type II diabetes mellitus requiring anti-hyperglycaemic medication or any participant with HbA1c \> 6.4%
✕. Patient who is currently receiving medication with a known risk of prolonging the QT interval or inducing Torsades de Pointes