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

Phase 2 Recruiting 40 enrolled Medical University of Warsaw

RandomizedParallel-groupOpen-labelTreatment

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.

Timeline

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

Publications

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Trametinib	Small molecule	0.025 mg/kg	Oral
Comparator	Disopiramide	Small molecule	—	Oral

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

Summary

Timeline

Publications

Drugs

Indications