Status:
RECRUITING
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Lead Sponsor:
Medical University of Warsaw
Conditions:
Cardiomegaly
Noonan Syndrome
Eligibility:
All Genders
1-18 years
Phase:
PHASE2
Brief Summary
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.
Detailed Description
Introduction RASopathies are a group of genetic diseases caused by mutations in the mitogen-activated kinase (RAS-MAPK) pathway. These mutations affect many processes and are the cause of numerous gen...
Eligibility Criteria
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
Key Trial Info
Start Date :
August 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06555237
Start Date
August 1 2024
End Date
December 1 2026
Last Update
August 15 2024
Active Locations (1)
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1
Department of Paediatrics, The Medical University of Warsaw, Poland
Warsaw, Poland, 02-091