MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies (MEKinRAS) - Randomized Controlled Trial
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 1 day
Maximum Age: 18
Healthy Volunteers: f
View:
• patient with diagnosed RASopathy
• patient with diagnosed hypertrophic cardiomyopathy
• signed innform consent
Locations
Other Locations
Poland
Department of Paediatrics, The Medical University of Warsaw, Poland
RECRUITING
Warsaw
Contact Information
Primary
Maciej Kołodziej, MD
maciej.kolodziej@wum.edu.pl
22-317-95-37
Backup
Halszka Kamińska, MD
halszka.kaminska@wum.edu.pl
22-317-9444
Time Frame
Start Date: 2024-08-01
Estimated Completion Date: 2026-12-01
Participants
Target number of participants: 40
Treatments
Experimental: Trametynib
Trametynib in 0,025mg/kg dose orally once daily
Active_comparator: Standard therapy
Disopiramide and Beta-blocker orally
Related Therapeutic Areas
Sponsors
Leads: Medical University of Warsaw