Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy
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 (pVO<sub>2</sub>) 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<sub>2</sub> 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).
Authors
- Omar Wever‐Pinzon (ORCID: https://orcid.org/0000-0002-2828-707X)
- Theodore P. Abraham (ORCID: https://orcid.org/0000-0001-8966-6906)
- Yue Zhong (ORCID: https://orcid.org/0000-0002-9839-0160)
- Jonathan Myers (ORCID: https://orcid.org/0000-0003-2592-136X)
- Thomas A. Rano
- Iacopo Olivotto (ORCID: https://orcid.org/0000-0003-1751-9266)
- Deborah Davey
- Milind Y. Desai (ORCID: https://orcid.org/0000-0003-0010-9797)
- Lisa Mitchell
- ANJALI TIKU OWENS (ORCID: https://orcid.org/0000-0002-9669-8495)
- Ron Aronson
- Victoria Florea (ORCID: https://orcid.org/0000-0001-5656-8381)
- Nicolas Verheyen (ORCID: https://orcid.org/0000-0001-7414-4056)
- Pablo García‐Pavía (ORCID: https://orcid.org/0000-0002-5470-2257)
- Vandana Bhatia (ORCID: https://orcid.org/0000-0002-1614-7192)
- Wael A. Jaber (ORCID: https://orcid.org/0000-0001-5642-2112)
- Kathy Wolski
- S.E. Nissen
- Suzanne Carter-Bonanza
Institutions
- Cleveland Clinic (US)
- Duke University (US)
- Palo Alto University (US)
- University of California, San Francisco (US)
- Medical University of Graz (AT)
- VA Palo Alto Health Care System (US)
- University of Utah (US)
- Bristol-Myers Squibb (United States) (US)
- Centro de Investigación en Red en Enfermedades Cardiovasculares (ES)
- Hospital Universitario Puerta de Hierro Majadahonda (ES)
- Meyer Children's Hospital (IT)
- University of San Francisco (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- JACC Heart Failure
- Published
- 2025-02-01
- DOI
- https://doi.org/10.1016/j.jchf.2024.11.013
- Citations
- 30
- Primary Topic
- Cardiomyopathy and Myosin Studies
- Type
- article
- Field-Weighted Citation Impact
- 41.65
Funders
- Celgene