Journal Article

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

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

2020

Overview

The differential mechanism of action of vamorolone compared to traditional corticosteroid anti-inflammatory drugs is attributed to the loss of gene transcriptional activities associated with glucocorticoid response element binding and activation, potent antagonist activity for the mineralocorticoid receptor, superior membrane stabilization properties, and retention of the distinct NFκB inhibitory (anti-inflammatory) activities [3,5–7]). Safety endpoints (linear growth, body mass index) are also compared with data from a 12-month trial of daily prednisone (0.75 mg/kg group) in similar-aged boys with DMD [17]. Methods Ethics statement All studies had appropriate approvals by ethics committees or institutional review boards, as required by the 11 participating international academic clinical recruitment sites: (Duke University, Durham, NC, US; Alberta Children’s Hospital, Calgary, AB, Canada; Nemours Children’s Hospital, Orlando, FL, US; John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK; Queen Silvia Children’s Hospital, Gothenburg, Sweden; Schneider Children’s Medical Center, Tel Aviv University, Petah Tikvah, Israel; Royal Children’s Hospital and Murdoch Children’s Research Institute, Melbourne, VIC, Australia; The Children’s Hospital at Westmead, Sydney, NSW, Australia; University of Texas Southwestern Medical Center, Dallas, TX, US; Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, US; University of California Davis, Davis, CA, US). [...]the total duration of corticosteroid treatment was longer than 18 months for most participants.

• Publisher: Public Library of Science,Public Library of Science (PLoS)
• Subject: Anti-inflammatory agents / Biology and Life Sciences / Body mass index / Children / Corticosteroids / Drug dosages / Duchenne's muscular dystrophy / Engineering and Technology / Enrollments / Ethics / Glucocorticoids / Immunomodulators / Inflammation / Medicine and Health Sciences / Muscular dystrophy / NF-κB protein / People and Places / Prednisone / Research and Analysis Methods / Transcription