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Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): A randomised, double-blind, placebo-controlled, phase 3 trial

The Lancet Sep 16, 2020

Olivotto I, Oreziak A, Barriales-Villa R, et al. - Researchers undertook this phase 3, randomised, double-blind, placebo-controlled trial (EXPLORER-HCM) to evaluate mavacamten's (a first-in-class cardiac myosin inhibitor) efficacy as well as safety in the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. This inquiry was performed in 68 clinical cardiovascular centres in 13 countries. Participants were patients suffering from hypertrophic cardiomyopathy with an left ventricular outflow tract (LVOT) gradient of 50 mm Hg or greater and New York Heart Association (NYHA) class II–III symptoms. These patients were allocated (1:1) to receive mavacamten (starting at 5 mg) or placebo for 30 weeks. According to findings, improvements in exercise capacity, LVOT obstruction, NYHA functional class, and health status, were brought about by treatment with mavacamten in patients with obstructive hypertrophic cardiomyopathy. The advantages of disease-specific treatment for this condition were emphasized by the findings of this pivotal trial.

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