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. 2017 May 10;5(2):14. doi: 10.3390/diseases5020014

Table 5.

Experimental trials investigating the role of elamipretide in heart failure treatment.

Study Year Study Design No. of Patients Results
Daubert et al. [79] 2016 Phase I randomised, placebo-controlled trial 36 patients with stable heart failure (EF < 45% and NYHA Class II–III) Heart failure treated with elamipretide compared to placebo resulted in:
Reduced left ventricular end-systolic volume (between-group difference −13.7; 95% CI −22.7–−4.8; p = 0.005) and end-diastolic volume (between-group difference −17.9 mL; 95% CI −30.6–−5.2; p = 0.009)
Elamipretide was well tolerated with no influence on blood and pressure and heart rate
Sabbah et al. [71] 2016 Canine experiment 14 dogs Treatment of dogs with heart failure with elamipretide compared to intravenous saline resulted in:
Improved EF (6% increase compared to pretreatment, p < 0.05) with no change in control
Reduced end-systolic LV volume (3 mL, p < 0.05) compared to increased volume in the control group
Increased maximum rate of ATP synthesis and increased ATP/ADP ratio (p < 0.05)
No effect on heart rate, mean aortic pressure, systemic vascular resistance or LV end-diastolic volume
Gupta et al. [78] 2016 Canine experiment 14 dogs Treatment of dogs with heart failure with elamipretide resulted in restoration of near normal levels of cMyBPC-S282 in the left ventricle (p < 0.05)
Shi et al. [72] 2015 Murine experiment 24 rats Treatment of post-MI rats with elamipretide showed:
Restored gene expression of mitochondrial energy metabolism
Promotion of mitochondrial biogenesis
Regulation of glucose and fatty acid oxidation related gene expression
Preserved SERCA2a expression
Reduction in cardiac fibrosis
Dai et al. [77] 2014 Murine experiment 56 rats Rats treated with elamipretide compared to water after acute MI showed improved LV function and prevention of adverse left ventricle remodelling.
Sabbah et al. [80] 2014 Canine experiment 12 dogs Dogs with heart failure treated with elamipretide compared to normal saline resulted in normalised expression of cardiolipin-remodelling genes and proteins (p < 0.05)

EF = ejection fraction; LV = left ventricle; cMyBPC-S282 = cardiac myosin binding protein-C at serine 282; SERCA2a = sarco/endoplasmic reticulum; MI = myocardial infarct.