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

NCT06555237 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2024-08-15

No results posted yet for this study

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.

Conditions

  • Cardiomegaly
  • Noonan Syndrome

Interventions

DRUG

Trametinib tablet

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

DRUG

Standard therapy

Disopiramide and Beta blockers orally

Sponsors & Collaborators

  • Medical University of Warsaw

    lead OTHER

Principal Investigators

  • MAciej Kołodziej, MD · Medical University of Warsaw

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Day
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-01
Primary Completion
2026-08-01
Completion
2026-12-01

Countries

  • Poland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06555237 on ClinicalTrials.gov