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MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies

RECRUITINGPhase 2Sponsored by Medical University of Warsaw
Actively Recruiting
PhasePhase 2
SponsorMedical University of Warsaw
Started2024-08-01
Est. completion2026-08-01
Eligibility
Age1 Day – 18 Years
Healthy vol.Accepted
View on ClinicalTrials.gov →

NCT06555237

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.

Eligibility

Age: 1 Day – 18 YearsHealthy volunteers accepted
Inclusion Criteria:

* patient with diagnosed RASopathy
* patient with diagnosed hypertrophic cardiomyopathy
* signed innform consent

Exclusion Criteria:

* 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

Conditions3

CardiomegalyHeart DiseaseNoonan Syndrome

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