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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

Author

Listed:
  • Edward C Smith
  • Laurie S Conklin
  • Eric P Hoffman
  • Paula R Clemens
  • Jean K Mah
  • Richard S Finkel
  • Michela Guglieri
  • Mar Tulinius
  • Yoram Nevo
  • Monique M Ryan
  • Richard Webster
  • Diana Castro
  • Nancy L Kuntz
  • Laurie Kerchner
  • Lauren P Morgenroth
  • Adrienne Arrieta
  • Maya Shimony
  • Mark Jaros
  • Phil Shale
  • Heather Gordish-Dressman
  • Laura Hagerty
  • Utkarsh J Dang
  • Jesse M Damsker
  • Benjamin D Schwartz
  • Laurel J Mengle-Gaw
  • Craig M McDonald
  • the CINRG VBP15 and DNHS Investigators

Abstract

Background: Treatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti-inflammatory investigational drug that has shown evidence of efficacy in DMD after 24 weeks of treatment at 2.0 or 6.0 mg/kg/day. Here, open-label efficacy and safety experience of vamorolone was evaluated over a period of 18 months in trial participants with DMD. Methods and findings: A multicenter, open-label, 24-week trial (VBP15-003) with a 24-month long-term extension (VBP15-LTE) was conducted by the Cooperative International Neuromuscular Research Group (CINRG) and evaluated drug-related effects of vamorolone on motor outcomes and corticosteroid-associated safety concerns. The study was carried out in Canada, US, UK, Australia, Sweden, and Israel, from 2016 to 2019. This report covers the initial 24-week trial and the first 12 months of the VBP15-LTE trial (total treatment period 18 months). DMD trial participants (males, 4 to

Suggested Citation

  • Edward C Smith & Laurie S Conklin & Eric P Hoffman & Paula R Clemens & Jean K Mah & Richard S Finkel & Michela Guglieri & Mar Tulinius & Yoram Nevo & Monique M Ryan & Richard Webster & Diana Castro & , 2020. "Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study," PLOS Medicine, Public Library of Science, vol. 17(9), pages 1-18, September.
    • Handle: RePEc:plo:pmed00:1003222
    DOI: 10.1371/journal.pmed.1003222
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