Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
Year: 2025
Type: article
Study design: Study Protocol
Abstract: There 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 (pVO2) 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 pVO2 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)
Source: JACC Heart Failure
Authors: Milind Y. Desai, Steve E. Nissen, Theodore Abraham, Iacopo Olivotto, Pablo Garcia-Pavia, Renato D. Lopes, Nicolas Verheyen, Omar Wever-Pinzon, Kathy Wolski, Wael Jaber, Lisa Mitchell, Deborah Davey, Jonathan Myers, Thomas Rano, Vandana Bhatia, Yue Zhong, Suzanne Carter-Bonanza, Victoria Florea, Ron Aronson, Anjali T. Owens
Institutions: Cleveland Clinic, University of San Francisco, Meyer Children's Hospital, Centro de Investigación en Red en Enfermedades Cardiovasculares, Hospital Universitario Puerta de Hierro Majadahonda, Duke University, Medical University of Graz, University of Utah, Stanford University, Bristol-Myers Squibb (United States), University of Pennsylvania
Language: English
Subfield: Cardiology and Cardiovascular Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $5,000
Funder: Celgene
OpenAlex ID: W4407096475