Actively Recruiting

Phase 2
Age: 1Day - 18Years
All Genders
NCT06555237

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

Led by Medical University of Warsaw · Updated on 2024-08-15

40

Participants Needed

1

Research Sites

121 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 1Day - 18Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient with diagnosed RASopathy
  • Patient with diagnosed hypertrophic cardiomyopathy
  • Signed informed consent
Not Eligible

You will not qualify if you...

  • Contraindications to propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia)
  • Contraindications to disopyramide (drug hypersensitivity, WPW syndrome, atrioventricular block, QT prolongation)
  • Contraindications to trametinib (drug hypersensitivity)
  • Lack of consent from the child's guardians to participate in the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Department of Paediatrics, The Medical University of Warsaw, Poland

Warsaw, Poland, 02-091

Actively Recruiting

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Research Team

M

Maciej Kołodziej, MD

CONTACT

H

Halszka Kamińska, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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