Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 Trial.


Journal

Journal of cardiac failure
ISSN: 1532-8414
Titre abrégé: J Card Fail
Pays: United States
ID NLM: 9442138

Informations de publication

Date de publication:
May 2020
Historique:
received: 01 10 2019
revised: 05 02 2020
accepted: 10 02 2020
pubmed: 19 2 2020
medline: 19 8 2021
entrez: 19 2 2020
Statut: ppublish

Résumé

Elamipretide, a novel mitochondrial modulating agent, improves myocardial energetics; however, it is unknown whether this mechanistic benefit translates into improved cardiac structure and function in heart failure (HF) with reduced ejection fraction (HFrEF). The objective of this study was to evaluate the effects of multiple subcutaneous doses of elamipretide on left ventricular end systolic volume (LVESV) as assessed by cardiac magnetic resonance imaging. We randomized 71 patients with HFrEF (LVEF ≤ 40%) in a double-blind, placebo-controlled trial in a 1:1:1 ratio to receive placebo, 4 mg or 40 mg elamipretide once daily for 28 consecutive days. The mean age (standard deviation) of the study population was 65 ± 10 years, 24% were females, and the mean EF was 31% ± 7%. The change in LVESV from baseline to week 4 was not significantly different between elamipretide 4 mg (89.4 mL to 85 mL; difference, -4.4 mL) or 40 mg (77.9 mL to 76.6 mL; difference, -1.2 mL) compared with placebo (77.7 mL to 74.6 mL; difference, -3.8 mL) (4 mg vs placebo: difference of means, -0.3; 95% CI, -4.6 to 4.0; P  =  0.90; and 40 mg vs placebo: difference of means, 2.3; 95% CI, -1.9 to 6.5; P  =  0.28). Also, no significant differences in change in LVESV and LVEF were observed between placebo and either of the elamipretide groups. Rates of any study drug-related adverse events were similar in the 3 groups. Elamipretide was well tolerated but did not improve LVESV at 4 weeks in patients with stable HFrEF compared with placebo.

Sections du résumé

BACKGROUND BACKGROUND
Elamipretide, a novel mitochondrial modulating agent, improves myocardial energetics; however, it is unknown whether this mechanistic benefit translates into improved cardiac structure and function in heart failure (HF) with reduced ejection fraction (HFrEF). The objective of this study was to evaluate the effects of multiple subcutaneous doses of elamipretide on left ventricular end systolic volume (LVESV) as assessed by cardiac magnetic resonance imaging.
METHODS METHODS
We randomized 71 patients with HFrEF (LVEF ≤ 40%) in a double-blind, placebo-controlled trial in a 1:1:1 ratio to receive placebo, 4 mg or 40 mg elamipretide once daily for 28 consecutive days.
RESULTS RESULTS
The mean age (standard deviation) of the study population was 65 ± 10 years, 24% were females, and the mean EF was 31% ± 7%. The change in LVESV from baseline to week 4 was not significantly different between elamipretide 4 mg (89.4 mL to 85 mL; difference, -4.4 mL) or 40 mg (77.9 mL to 76.6 mL; difference, -1.2 mL) compared with placebo (77.7 mL to 74.6 mL; difference, -3.8 mL) (4 mg vs placebo: difference of means, -0.3; 95% CI, -4.6 to 4.0; P  =  0.90; and 40 mg vs placebo: difference of means, 2.3; 95% CI, -1.9 to 6.5; P  =  0.28). Also, no significant differences in change in LVESV and LVEF were observed between placebo and either of the elamipretide groups. Rates of any study drug-related adverse events were similar in the 3 groups.
CONCLUSIONS CONCLUSIONS
Elamipretide was well tolerated but did not improve LVESV at 4 weeks in patients with stable HFrEF compared with placebo.

Identifiants

pubmed: 32068002
pii: S1071-9164(19)31583-0
doi: 10.1016/j.cardfail.2020.02.001
pii:
doi:

Substances chimiques

Oligopeptides 0
arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide 0

Types de publication

Clinical Trial, Phase II Journal Article Randomized Controlled Trial

Langues

eng

Sous-ensembles de citation

IM

Pagination

429-437

Subventions

Organisme : NIGMS NIH HHS
ID : U54 GM115428
Pays : United States

Informations de copyright

Copyright © 2020. Published by Elsevier Inc.

Auteurs

Javed Butler (J)

Department of Medicine, University of Mississippi Medical Center, Jackson, Missisippi, USA.

Muhammad Shahzeb Khan (MS)

Department of Medicine, Cook County Hospital, Chicago, Illinois, USA.

Stefan D Anker (SD)

Department of Cardiology (CVK), Berlin Institute of Health Center for Regenerative Therapies (BCRT), German Centre for Cardiovascular Research (DZHK) partner site Berlin, Charité Universitätsmedizin Berlin, Germany.

Gregg C Fonarow (GC)

Division of Cardiology, Department of Medicine, University of California, Los Angeles, Los Angeles, California, USA.

Raymond J Kim (RJ)

Duke Cardiovascular Magnetic Resonance Center, Department of Medicine, Duke University School of Medicine, Durham, North Carolina, USA.

Savina Nodari (S)

Cardiology Section, Department of Clinical and Surgical Specialties, Radiological Sciences and Public Health, University of Brescia, Brescia, Italy.

Christopher M O'Connor (CM)

Inova Heart and Vascular Institute, Falls Church, Virginia, USA.

Burkert Pieske (B)

Department of Internal Medicine and Cardiology, Charité Universitätsmedizin Berlin, Campus Virchow Klinikum, Berlin, Germany; Department of Internal Medicine and Cardiology, German Heart Center Berlin, and German Centre for Cardiovascular Research (DZHK), Partner site Berlin, and Berlin Institute of Health (BIH), Berlin, Germany.

Elisabeth Pieske-Kraigher (E)

Department of Internal Medicine and Cardiology, Charité Universitätsmedizin Berlin, Campus Virchow Klinikum, Berlin, Germany; Department of Internal Medicine and Cardiology, German Heart Center Berlin, and German Centre for Cardiovascular Research (DZHK), Partner site Berlin, and Berlin Institute of Health (BIH), Berlin, Germany.

Hani N Sabbah (HN)

Division of Cardiovascular Medicine, Henry Ford Hospital, Detroit, Michigan, USA.

Michele Senni (M)

Cardiovascular Department, ASST Papa Giovanni XXIII, Bergamo, Italy.

Adriaan A Voors (AA)

Department of Cardiology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands.

James E Udelson (JE)

Division of Cardiology and the Cardiovascular Center, Tufts Medical Center, Boston, Massachusetts, USA.

Jim Carr (J)

Stealth BioTherapeutics, Newton, Massachusetts, USA.

Mihai Gheorghiade (M)

Department of Medicine, Bluhm Cardiovascular Institute, Northwestern University Feingerg School of Medicine, Chicago, Illinois, USA.

Gerasimos Filippatos (G)

National and Kapodistrian University of Athens, Attikon University Hospital, Athens, Greece. Electronic address: geros@otenet.gr.

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