MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorMedical University of Warsaw
About this 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.
Eligibility criteria
Qualifiers
patient with diagnosed RASopathy
patient with diagnosed hypertrophic cardiomyopathy
signed innform consent
Disqualifiers
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
Trial design
Treatments tested in this trial
- Trametinib tablet
- Standard therapy
Treatment groups
40 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov