EXPLORER-HCM
Clinical Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Patient / Population Intervention / Exposition Comparaison Critère de jugement
In 251 patients with symptomatic obstructive HCM (NYHA II-III, LVOT gradient ≥50 mmHg), mavacamten improved the composite of peak VO2 and NYHA functional class compared with placebo.
N
251
Schéma
Double-blind placebo-controlled RCT, phase 3
Critère
Composite responder (peak VO2 rise plus NYHA-class improvement) at week 30
Pertinence
2
Résultat45/123 (37%) with mavacamten vs 22/128 (17%) with placebo met the primary endpoint (difference +19.4%, 95% CI 8.7-30.1; P=0.0005). LVOT gradient and symptoms also improved.
Olivotto I, et al. Lancet. 2020;396(10253):759-769. 10.1016/S0140-6736(20)31792-X
Discussion et critique
Surrogate/functional composite endpoint rather than hard outcomes, with a modest absolute responder difference. First pivotal trial of a cardiac myosin inhibitor, leading to FDA approval of mavacamten for obstructive HCM.