RecruitingPhase 2

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

Poland40 participantsStarted 2024-08-01

Plain-language 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.

Who can participate

Age range1 Day – 18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Effectiveness of the trametynib treatment on cardiac hyperthropy

Timeframe: 1 year

Effectiveness of the trametynib treatment on laboratory enzymes levels

Timeframe: 1 year

Trial details

NCT IDNCT06555237

SponsorMedical University of Warsaw

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-08-01

Contact for this trial

Maciej Kołodziej, MD

22-317-95-37 maciej.kolodziej@wum.edu.pl

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