Martin S Maron, Masliza Mahmod, Azlan Helmy Abd Samat, Lubna Choudhury, Daniele Massera, Dermot M J Phelan, Sharon Cresci, Matthew W Martinez, Ahmad Masri, Theodore P Abraham, Eric Adler, Omar Wever-Pinzon, Sherif F Nagueh, Gregory D Lewis, Paul Chamberlin, Jai Patel, Arash Yavari, Hakim-Moulay Dehbi, Rizwan Sarwar, Betty Raman, Ladislav Valkovič, Stefan Neubauer, James E Udelson, Hugh Watkins
BACKGROUND: In nonobstructive hypertrophic cardiomyopathy (nHCM), there are no approved medical therapies. Impaired myocardial energetics is a potential cause of symptoms and exercise limitation. Ninerafaxstat, a novel cardiac mitotrope, enhances cardiac energetics. OBJECTIVES: To evaluate the safety and efficacy of ninerafaxstat in nHCM. METHODS: Patients with HCM and left ventricular (LV) outflow gradient <30 mmHg, ejection fraction ≥50% and peak VO2 <80% predicted, were randomized to ninerafaxstat 200 mg BID or placebo (1:1) for 12 weeks...
April 1, 2024: Journal of the American College of Cardiology