JACC: Basic to Translational Science (Dec 2016)

A Phase I, Single Ascending Dose Study of Cimaglermin Alfa (Neuregulin 1β3) in Patients With Systolic Dysfunction and Heart Failure

  • Daniel J. Lenihan, MD,
  • Sarah A. Anderson, ACNP,
  • Carrie Geisberg Lenneman, MD,
  • Evan Brittain, MD, MSCI,
  • James A.S. Muldowney, III, MD,
  • Lisa Mendes, MD,
  • Ping Z. Zhao, PhD,
  • Jennifer Iaci, MS,
  • Stephen Frohwein, MD,
  • Ronald Zolty, MD, PhD,
  • Andrew Eisen, MD, PhD,
  • Douglas B. Sawyer, MD, PhD,
  • Anthony O. Caggiano, MD, PhD

DOI
https://doi.org/10.1016/j.jacbts.2016.09.005
Journal volume & issue
Vol. 1, no. 7
pp. 576 – 586

Abstract

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A first-in-human, phase 1, double blind, placebo-controlled, single ascending dose study examined the safety, tolerability, and exploratory efficacy of intravenous infusion of a recombinant growth factor, cimaglermin alfa, in patients with heart failure and left ventricular systolic dysfunction (LVSD). In these patients on optimal guideline-directed medical therapy, cimaglermin treatment was generally tolerated except for transient nausea and headache and a dose-limiting toxicity was noted at the highest planned dose. There was a dose-dependent improvement in left ventricular ejection fraction lasting 90 days following infusion. Thus, cimaglermin is a potential therapy to enhance cardiac function in LVSD and warrants further investigation.

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