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

Sponsor
Medical University of Warsaw
Study ID
NCT06555237
Phase
PHASE2
Status
Recruiting
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Conditions

  • Cardiomegaly
  • Noonan Syndrome

Eligibility Criteria

Sex
ALL
Age
1 Day - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Trametinib tablet — DRUG
    Trametynib orally once daily in 0,025mg/kg dose
  • Standard therapy — DRUG
    Disopiramide and Beta blockers orally

Study Details

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 Dates

Start date
Aug 1, 2024
Status verified
Aug 2024
Primary completion
Aug 1, 2026
Completion
Dec 1, 2026

Study Design

Enrollment
40 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Trametynib
    Trametynib in 0,025mg/kg dose orally once daily
  • Active Comparator: Standard therapy
    Disopiramide and Beta-blocker orally

Primary Outcome Measure

Effectiveness of the trametynib treatment on cardiac hyperthropy [ Time Frame: 1 year ]

Central Contacts

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