title: Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study.
abstract: OBJECTIVE
  To evaluate the efficacy of a prolonged-release formulation of a porcine adrenocorticotropic hormone analogue (repository corticotropin injection (RCI)) added to standard of care in patients requiring moderate-dose corticosteroids for symptomatic SLE.
  
  
  METHODS
  This prospective, randomised, double-blind, phase 4, pilot study (NCT01753401) enrolled 38 patients with persistently active SLE involving skin and/or joints. Enrolled patients received RCI, 40 U daily or 80 U every other day, or volume-matched placebo gel, for 8 weeks, with dose tapering to twice weekly during weeks 5-8. Efficacy endpoints included proportion of responders at week 4 based on a novel composite measure that included resolution of rash or arthritis measured using the hybrid SLE Disease Activity Index (hSLEDAI) without worsening British Isles Lupus Assessment Group (BILAG) scores in other organ systems at week 4 (primary), as well as improvements in total hSLEDAI and BILAG scores and other measures of skin and joint disease activity over the 8-week treatment period.
  
  
  RESULTS
  Response, as defined for the primary endpoint, did not differ significantly between the combined placebo and RCI-treated groups at week 4. At week 8, the proportion of responders was higher in RCI-treated patients but did not statistically differ between groups (RCI 40 U (53.8%), RCI 80 U (33.3%), combined placebo (27.3%)). However, RCI treatment was associated with statistically significant improvements in several secondary endpoints, including total hSLEDAI, total BILAG and Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity scores within 8 weeks. Treatment was well tolerated.
  
  
  CONCLUSIONS
  Although the primary endpoint was not met in this pilot study, secondary and post hoc analyses suggested that RCI was associated with improvements in SLE disease activity in a select patient population with steroid-dependent persistent disease.
  
  
  BACKGROUND
  NCT01753401; results.
authors.affiliation: Hofstra Northwell School of Medicine, Northwell Health , Great Neck, New York , USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Furie
authors.affiliation: Manhattan BioPharm Consultants LLC , New York, New York , USA.
authors.forename: Margaret
authors.initials: M
authors.lastname: Mitrane
authors.affiliation: Research & Development, Mallinckrodt Pharmaceuticals Inc. , Ellicott City, Maryland , USA.
authors.forename: Enxu
authors.initials: E
authors.lastname: Zhao
authors.affiliation: Research & Development, Mallinckrodt Pharmaceuticals Inc. , Ellicott City, Maryland , USA.
authors.forename: Maya
authors.initials: M
authors.lastname: Das
authors.affiliation: Research & Development, Mallinckrodt Pharmaceuticals Inc. , Ellicott City, Maryland , USA.
authors.forename: Daner
authors.initials: D
authors.lastname: Li
authors.affiliation: Research & Development, Mallinckrodt Pharmaceuticals Inc. , Ellicott City, Maryland , USA.
authors.forename: Patrice M
authors.initials: PM
authors.lastname: Becker
keywords: Autoimmune Diseases; Corticosteroids; Disease Activity; Systemic Lupus Erythematosus