MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
MEKinRAS
1 other identifier
interventional
40
1 country
1
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Aug 2024
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2024
CompletedFirst Submitted
Initial submission to the registry
August 12, 2024
CompletedFirst Posted
Study publicly available on registry
August 15, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2026
August 15, 2024
August 1, 2024
2 years
August 12, 2024
August 12, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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.
1 year
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.
1 year
Secondary Outcomes (2)
MEK kinase activity
6 months
Effectivness of the trametynib treatment assessed in the cardia magnetic resonance
1 year
Study Arms (2)
Trametynib
EXPERIMENTALTrametynib in 0,025mg/kg dose orally once daily
Standard therapy
ACTIVE COMPARATORDisopiramide and Beta-blocker orally
Interventions
Eligibility Criteria
You may qualify if:
- patient with diagnosed RASopathy
- patient with diagnosed hypertrophic cardiomyopathy
- signed innform consent
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Department of Paediatrics, The Medical University of Warsaw, Poland
Warsaw, 02-091, Poland
Related Publications (1)
De Brouchoven I, Lorand J, Bofferding L, Sorlin A, Van Damme A, Danhaive O. Trametinib as a targeted treatment in cardiac and lymphatic presentations of Noonan syndrome. Front Pediatr. 2025 Feb 18;13:1475143. doi: 10.3389/fped.2025.1475143. eCollection 2025.
PMID: 40041314DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
MAciej Kołodziej, MD
Medical University of Warsaw
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 12, 2024
First Posted
August 15, 2024
Study Start
August 1, 2024
Primary Completion (Estimated)
August 1, 2026
Study Completion (Estimated)
December 1, 2026
Last Updated
August 15, 2024
Record last verified: 2024-08