1. Randomized controlled trial of perhexiline on regression of left ventricular hypertrophy in patients with symptomatic hypertrophic cardiomyopathy (RESOLVE-HCM trial).
- Author
-
Ananthakrishna R, Lee SL, Foote J, Sallustio BC, Binda G, Mangoni AA, Woodman R, Semsarian C, Horowitz JD, and Selvanayagam JB
- Subjects
- Adult, Female, Humans, Male, Double-Blind Method, Echocardiography, Magnetic Resonance Imaging, Prospective Studies, Randomized Controlled Trials as Topic, Multicenter Studies as Topic, Cardiomyopathy, Hypertrophic diagnostic imaging, Cardiomyopathy, Hypertrophic drug therapy, Cardiomyopathy, Hypertrophic physiopathology, Cardiovascular Agents therapeutic use, Hypertrophy, Left Ventricular diagnostic imaging, Hypertrophy, Left Ventricular etiology, Hypertrophy, Left Ventricular prevention & control, Perhexiline therapeutic use
- Abstract
Background: The presence and extent of left ventricular hypertrophy (LVH) is a major determinant of symptoms in patients with hypertrophic cardiomyopathy (HCM). There is increasing evidence to suggest that myocardial energetic impairment represents a central mechanism leading to LVH in HCM. There is currently a significant unmet need for disease-modifying therapy that regresses LVH in HCM patients. Perhexiline, a potent carnitine palmitoyl transferase-1 (CPT-1) inhibitor, improves myocardial energetics in HCM, and has the potential to reduce LVH in HCM., Objective: The primary objective is to evaluate the effects of perhexiline treatment on the extent of LVH, in symptomatic HCM patients with at least moderate LVH., Methods/design: RESOLVE-HCM is a prospective, multicenter double-blind placebo-controlled randomized trial enrolling symptomatic HCM patients with at least moderate LVH. Sixty patients will be randomized to receive either perhexiline or matching placebo. The primary endpoint is change in LVH, assessed utilizing cardiovascular magnetic resonance (CMR) imaging, after 12-months treatment with perhexiline., Summary: RESOLVE-HCM will provide novel information on the utility of perhexiline in regression of LVH in symptomatic HCM patients. A positive result would lead to the design of a Phase 3 clinical trial addressing long-term effects of perhexiline on risk of heart failure and mortality in HCM patients., (Copyright © 2021 Elsevier Inc. All rights reserved.)
- Published
- 2021
- Full Text
- View/download PDF