RecruitingPhase 2NCT06555237

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

MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies (MEKinRAS) - Randomized Controlled Trial

Sponsor

Medical University of Warsaw

Enrollment

40 participants

Start Date

Aug 1, 2024

Study Type

INTERVENTIONAL

Conditions

Noonan Syndrome Cardiomegaly

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.

Eligibility

Min Age: 1 DayMax Age: 18 Years

Inclusion Criteria3

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

Exclusion Criteria2

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

Print for Your Doctor

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGTrametinib tablet

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

DRUGStandard therapy

Disopiramide and Beta blockers orally

Locations(1)

Department of Paediatrics, The Medical University of Warsaw, Poland

Warsaw, Poland

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06555237

Related Trials

Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome

NCT052022101 location

National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype

NCT0746482114 locations

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies

NCT048889362 locations

A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

NCT0666880524 locations

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

NCT053618111 location