Tests trametinib for thickened heart muscle in children with RASopathies
Tests trametinib in children with thickened heart muscle and RAS/MAPK gene changes.
Plain-language title generated from the registry record
Registry title: MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Key facts
- Where
- Warsaw, PolandSee the site
- Phase
- Phase 2
Where in Poland
- Department of Paediatrics, The Medical University of Warsaw, PolandWarsaw, PolandTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Medical University of Warsaw
- Trial code
- NCT06555237
- Record updated
- 15 August 2024
- Original description
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.Read full original descriptionShow less
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies (MEKinRAS) - Randomized Controlled Trial
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.
Inclusion: patient with diagnosed RASopathy; patient with diagnosed hypertrophic cardiomyopathy; signed innform consent.
Exclusion: contraindications to treatment with propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia) disopyramide (drug hypersensitivity, WPW syndrome, atrioventricular block, QT prolongation) trametinib (drug hypersensitivity); lack of consent of the child's guardians to participate in the study.