A single ascending-dose study of muscle regulator ace-031 in healthy volunteers
Article first published online: 21 NOV 2012
Copyright © 2012 Wiley Periodicals, Inc., a Wiley company
Muscle & Nerve
Volume 47, Issue 3, pages 416–423, March 2013
How to Cite
Attie, K. M., Borgstein, N. G., Yang, Y., Condon, C. H., Wilson, D. M., Pearsall, A. E., Kumar, R., Willins, D. A., Seehra, J. S. and Sherman, M. L. (2013), A single ascending-dose study of muscle regulator ace-031 in healthy volunteers. Muscle Nerve, 47: 416–423. doi: 10.1002/mus.23539
- Issue published online: 23 FEB 2013
- Article first published online: 21 NOV 2012
- Accepted manuscript online: 1 AUG 2012 03:16AM EST
- Manuscript Accepted: 28 JUL 2012
- neuromuscular disease;
- placebo-controlled clinical trial
ACE-031 is a soluble form of activin receptor type IIB (ActRIIB). ACE-031 promotes muscle growth by binding to myostatin and other negative regulators of muscle mass.
This double-blind, placebo-controlled study evaluated the safety, pharmacokinetics, and pharmacodynamics of ACE-031 in 48 healthy, postmenopausal women randomized to receive 1 dose of ACE-031 (0.02–3 mg/kg SC) or placebo (3:1).
ACE-031 was generally well-tolerated. Adverse events included injection site erythema. Mean ACE-031 AUC0–∞ and Cmax increased linearly with dose; mean T½ was 10–15 days. Statistically significant increases in mean total body lean mass (3.3%; P = 0.03, by DXA) and thigh muscle volume (5.1%; P = 0.03, by MRI) were observed at day 29 in the 3 mg/kg group. Statistically significant changes in serum biomarkers suggest ACE-031 also improved bone and fat metabolism.
Single-dose ACE-031 treatment was generally well-tolerated and resulted in increases in muscle mass in healthy postmenopausal women. Muscle Nerve [B]47[/B]: 416–423, 2013