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  • Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study

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Clinical trials and drug discovery
Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study
  1. Richard Furie1,
  2. Margaret Mitrane2,
  3. Enxu Zhao3,
  4. Maya Das3,
  5. Daner Li3 and
  6. Patrice M Becker3
  1. 1Hofstra Northwell School of Medicine, Northwell Health, Great Neck, New York, USA
  2. 2Manhattan BioPharm Consultants LLC, New York, New York, USA
  3. 3Research & Development, Mallinckrodt Pharmaceuticals Inc., Ellicott City, Maryland, USA
  1. Correspondence to Dr Richard Furie rfurie{at}northwell.edu

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.

Trial registration number NCT01753401; results.

  • Autoimmune Diseases
  • Corticosteroids
  • Systemic Lupus Erythematosus
  • Disease Activity

This is an Open Access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/

https://doi.org/10.1136/lupus-2016-000180

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    Footnotes

    • Contributors RF was involved in the design of the study, interpretation and analysis of data, and drafting of the manuscript or revising it for critically important intellectual content. MM was involved in the interpretation and analysis of data, and drafting of the manuscript or revising it for critically important intellectual content. EZ was involved in the interpretation and analysis of data, and drafting of the manuscript or revising it for critically important intellectual content. MD was involved in the design of the study, acquisition of data, and interpretation and analysis of data. DL was involved in the acquisition of data, and interpretation and analysis of data. PMB was involved in the design of the study, interpretation and analysis of data, and drafting of the manuscript or revising it for critically important intellectual content. All authors provided final approval of the submitted manuscript and vouch for the completeness and accuracy of the data and data analyses, and for the fidelity of the study to the protocol.

    • Funding Mallinckrodt Pharmaceuticals sponsored this study and provided the study drug, designed the study, conducted the study, collected the data, monitored the conduct of the study and performed statistical analyses in collaboration with the contract research organisation (INC Research, Raleigh, North Carolina, USA) and the external principal and site investigators. The sponsor also provided funding for professional medical writing and editorial assistance.

    • Competing interests RF has been a paid consultant to Mallinckrodt Pharmaceuticals as well as the study and a site principal investigator. MM is a paid consultant to Mallinckrodt Pharmaceuticals. EZ is an employee of Mallinckrodt Pharmaceuticals. MD and DL were employees of Mallinckrodt Pharmaceuticals at the time of the study and hold stock or stock options in the company. PMB is an employee of Mallinckrodt Pharmaceuticals and holds stock or stock options in the company.

    • Patient consent Obtained.

    • Ethics approval The protocol was approved by the Institutional Review Boards at all participating centres, and the study was conducted according to the principles of the Declaration of Helsinki and Good Clinical Practice.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Data sharing statement No additional data are available.