Results from a multicenter, randomized, double-blind, placebo-controlled study of repository corticotropin injection for multiple sclerosis relapse that did not adequately respond to corticosteroids.

Abstract

IntroductionAbout 20%–35% of multiple sclerosis (MS) patients fail to respond to high‐dose corticosteroids during a relapse. Repository corticotropin injection (RCI, Acthar® Gel) is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and pituitary peptides that has anti‐inflammatory and immunomodulatory effects.AimsThe study objective was to determine the efficacy and safety of RCI in patients with MS relapse that inadequately responded to corticosteroids. This was a multicenter, double‐blind, placebo‐controlled study. Nonresponders to high‐dose corticosteroids were randomized to receive RCI (80 U) or placebo daily for 14 days. Assessments included improvements on the Expanded Disability Status Scale (EDSS), Multiple Sclerosis Impact Scale (MSIS‐29), Clinical Global Impression of Improvement (CGI‐I), and adverse events (AEs).ResultsEighteen patients received RCI, and 17 received placebo. A greater proportion of EDSS responders was observed in the RCI group at Day 7, 21, and 42 compared with the placebo group. Qualitative CGI‐I showed that more patients receiving RCI were much improved or very much improved than with placebo. No meaningful differences were observed between treatment groups for MSIS‐29. No serious AEs or deaths were reported.ConclusionRCI is safe and effective for MS relapse patients who do not respond to high‐dose corticosteroids.