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

ConditionsCardiomegalyNoonan Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorMedical University of Warsaw

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

patient with diagnosed RASopathy

patient with diagnosed hypertrophic cardiomyopathy

signed innform consent

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Trametinib tablet

    Drug

    Trametynib orally once daily in 0,025mg/kg dose

  • Standard therapy

    Drug

    Disopiramide and Beta blockers orally

Treatment groups

40 Participants
are divided into 2 treatment groups
Group A: TrametynibExperimental treatment 1 intervention
Group B: Standard therapyActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

Effectiveness of the trametynib treatment on cardiac hyperthropy

Reduction of cardiac hypertrophy and/or LVOTO at 6 and 12 months of treatment and at 3 months of follow-up after treatment discontinuation using echocardiographic examination.

Time frame
1 year
2

Effectiveness of the trametynib treatment on laboratory enzymes levels

Decrease in cardiac enzyme levels (NT-pro-BNP and high-sensitivity troponin I) determined in the 6th and 12th month of treatment and in the 3rd month of observation after its discontinuation.

Time frame
1 year

Secondary outcomes

1

MEK kinase activity

Assessment of MEK kinase activity before treatment and after 6 months of its duration, in the patients' blood samples

Time frame
6 months
2

Effectivness of the trametynib treatment assessed in the cardia magnetic resonance

reduction of cardiac hypertrophy in magnetic resonance imaging in the age group of 7-18 years not requiring general anesthesia (comparison of results before inclusion in the study, after 12 months of trametinib treatment and after the end of follow-up after its discontinuation)

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Medical University of Warsaw

Lead sponsor

Source ClinicalTrials.gov
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