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. 2016 Oct 21;3(1):e000180.
doi: 10.1136/lupus-2016-000180. eCollection 2016.

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

Richard Furie  1 , Margaret Mitrane  2 , Enxu Zhao  3 , Maya Das  3 , Daner Li  3 , Patrice M Becker  3
Affiliations

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

Richard Furie et al. Lupus Sci Med. .

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.

Keywords: Autoimmune Diseases; Corticosteroids; Disease Activity; Systemic Lupus Erythematosus.

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Conflict of interest statement

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.

Figures

Figure 1
Figure 1
CONSORT diagram of study participants. RCI, repository corticotropin injection.
Figure 2
Figure 2
Proportion of patients showing a response to treatment at weeks 4 and 8. QD, once daily; QOD, once every other day; RCI, repository corticotropin injection.
Figure 3
Figure 3
Response rate as defined by Systemic Lupus Erythematosus Responder Index at weeks 4 and 8. *p<0.05. QD, once daily; QOD, once every other day; RCI, repository corticotropin injection.
Figure 4
Figure 4
Least squares (LS) mean (SE) changes from baseline in (A) hybrid SLE Disease Activity Index scores at weeks 2, 4, 6 and 8, and (B) global British Isles Lupus Assessment Group scores at weeks 4 and 8. Values shown below error bars are LS means. *p<0.05. QD, once daily; QOD, once every other day; RCI, repository corticotropin injection.
Figure 5
Figure 5
Least squares (LS) mean (SE) changes from baseline in (A) Cutaneous Lupus Erythematosus Disease Area and Severity Index activity scores and (B) Tender and Swollen Joint Count at weeks 4 and 8. Values shown below error bars are LS means. *p<0.05. QD, once daily; QOD, once every other day; RCI, repository corticotropin injection.
Figure 6
Figure 6
Least squares (LS) mean (SE) changes from baseline in Physician's Global Assessment scores at weeks 4 and 8. Values shown below error bars are LS means. QD, once daily; QOD, once every other day; RCI, repository corticotropin injection.
See this image and copyright information in PMC

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