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

Recruiting now · Phase 2

Conditions studied: Cardiomegaly, Noonan Syndrome

In brief

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.

Key facts

Study ID

NCT06555237

Run by

Medical University of Warsaw

People needed

40

Starts

2024-08-01

Expected to finish

2026-12-01

Last updated by the study team

2024-08-15

Who can join

Age: 0 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• 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

Where it is running

• Department of Paediatrics, The Medical University of Warsaw, Poland — Warsaw, Poland (enrolling)

Full record on ClinicalTrials.gov

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