Rationale and design of MUSIC-HFpEF: a phase 1b, pilot trial evaluating the safety and pharmacodynamic effects of AAV1.SERCA2A in heart failure with preserved ejection fraction.
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2026-01
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Heart failure with preserved ejection fraction (HFpEF) has limited therapeutic options. Abnormal calcium handling through impaired sarcoplasmic/endoplasmic reticulum Ca2+ ATPase 2a (SERCA2a) activity contributes to diastolic dysfunction. AAV1.SERCA2A, an Adeno-Associated Virus Serotype 1 (AAV1) vector encoding SERCA2a, may improve myocardial relaxation in HFpEF. Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction (MUSIC-HFpEF) is a Phase 1b, open-label, multicenter trial (NCT06061549) currently enrolling 10 patients with hemodynamically confirmed HFpEF. Patients receive a one-time intracoronary infusion of AAV1.SERCA2A. The primary objective is to assess safety and tolerability; secondary endpoints include effects on resting and exercise hemodynamics, echocardiographic measures of relaxation, N-terminal pro-B-type natriuretic peptide (NT-proBNP), and functional status. MUSIC-HFpEF is the first-in-human trial of SERCA2a gene therapy in HFpEF. Findings will inform the feasibility, safety, and potential clinical benefit of targeted gene therapy in this population. The study is enrolling participants and aims to clarify the safety profile and potential benefits of administering a single dose of AAV1.SERCA2A to individuals with HFpEF.
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Fudim, Marat, Satyam Sarma, James P MacNamara, Deborah D Ascheim, Veraprapas Kittipibul, Katheryn Doukellis, Andy Dhimitri, Robert Fitzhugh, et al. (2026). Rationale and design of MUSIC-HFpEF: a phase 1b, pilot trial evaluating the safety and pharmacodynamic effects of AAV1.SERCA2A in heart failure with preserved ejection fraction. Heart failure reviews, 31(1). p. 19. 10.1007/s10741-025-10589-w Retrieved from https://hdl.handle.net/10161/33994.
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Scholars@Duke
Marat Fudim
Manesh Raman Patel
Manesh Patel is the Chief of the Division of Cardiology and the Division of Clinical Pharmacology. His clinical interests include diagnostic and interventional coronary angiography, peripheral angiography and endovascular intervention. His is involved in several clinical trials involving patients with cardiovascular disease and in cardiac imaging. He is also the Chair of the American College of Cardiology Task Force for Appropriate Use Criteria for Cardiovascular Procedures and is Chair of the American Heart Association Diagnostic and Interventional Cath Committee.
Patel's interest in cardiac imaging, quality of care, cardiac devices is also evident in his research. His integration of these efforts into his roles at Duke was recognized in 2010 when he received the prestigious Duke Cardiology Fellowship Mentor Award. In 2011, Dr. Patel was named the endowed John Bush Simpson Assistant Professor of Cardiology. In 2013, Dr. Patel received the Robert M. Califf Faculty clinical research Award.
Currently, Dr. Patel is leading an effort to redesign the delivery of care to patients undergoing invasive catheterization procedures in the health system with a specific aim of measure and providing individualized, patient centered, innovative, and efficient care.
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