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

RecruitingPhase 2Ages 1 Day - 18 YearsAllMedical University of Warsaw
NCT06555237 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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
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What it tests

  • Trametinib tablet
  • Standard therapy
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Where

1 site
Department of Paediatrics, The Medical University of Warsaw, Poland
Warsaw, Poland
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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