Desai, Milind YNissen, Steve EAbraham, TheodoreOlivotto, IacopoGarcia-Pavia, PabloLopes, Renato DVerheyen, NicolasWever-Pinzon, OmarWolski, KathyJaber, WaelMitchell, LisaDavey, DeborahMyers, JonathanRano, ThomasBhatia, VandanaZhong, YueCarter-Bonanza, SuzanneFlorea, VictoriaAronson, RonOwens, Anjali T2025-07-092025-07-092025-02JACC Heart Fail. 2025 Feb;13(2):358-370.https://hdl.handle.net/20.500.12105/26807There are no approved therapies for patients with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The authors describe the baseline characteristics of ODYSSEY-HCM (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy), a phase 3, randomized, double-blind, placebo-controlled trial conducted worldwide at 201 sites evaluating mavacamten in symptomatic adult patients with nHCM. The 2 primary endpoints are the changes from baseline to week 48 in: 1) Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score; and 2) peak oxygen consumption (pVO) on cardiopulmonary exercise testing. Dose titrations are made on blinded core laboratory assessments. Of 1,088 patients screened, 580 are randomized (mean age 56 ± 15 years, 46% women, 43% with family histories). All patients are nonobstructive and symptomatic (70% in NYHA functional class II and 30% class III), with a mean Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score of 58 ± 20, and 77% are on beta-blockers. The mean left ventricular ejection fraction and pVO are 66% ± 4% and 18 ± 6 mL/kg/min, respectively. ODYSSEY-HCM will report if mavacamten improves patient-reported health status and exercise capacity in patients with symptomatic nHCM. (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy (ODYSSEY-HCM); NCT05582395).engVoRhttp://creativecommons.org/licenses/by-nc-nd/4.0/characteristicsdesignmavacamtennonobstructive HCMAttribution-NonCommercial-NoDerivatives 4.0 International39909647JACC. Heart Failureopen access