The purpose of this study is to evaluate the effects and safety of an experimental drug called ninerafaxstat in people who are experiencing symptoms from non-obstructive hypertrophic cardiomyopathy (nHCM, also called “symptomatic nHCM”). In nHCM, the heart muscle is thicker than it should be, which can affect how well the heart pumps blood. Common symptoms include shortness of breath, discomfort or tightness in the chest, feeling unusually tired, faint, or dizzy, and palpitations (racing or irregular heartbeat). Ninerafaxstat is designed to help the heart make energy more efficiently. By boosting the energy levels and efficiency of the heart, ninerafaxstat can make the heart work with less strain, which may help improve symptoms of nHCM. Study procedures include physical exams, vitals, medical history, electrocardiograms, echocardiograms, exercise stress test, imaging, and blood draws. Ninerafaxstat will be compared to placebo (inactive substance) when taken alongside the patient’s regular treatment. Participants will be randomly assigned to receive either ninerafaxstat or placebo.
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A Global Phase 2b, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients with Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy