Overview

This trial is active, not recruiting.

Condition systemic lupus erythematosus (sle)
Treatments acthar, placebo for acthar
Phase phase 4
Sponsor Mallinckrodt
Start date December 2012
End date May 2017
Trial size 36 participants
Trial identifier NCT01753401, QSC01-SLE-01

Summary

This Phase 4 study is being performed to examine the effects of Acthar for the indicated use of treatment of SLE. This study will enroll patients with steroid-dependent, persistently active SLE with arthritic and/or cutaneous involvement. The study will involve two phases: a double-blind phase, to provide placebo-controlled safety, efficacy, and pharmacodynamic data, and an optional open-label phase, to examine the prolonged effects of Acthar maintenance.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Experimental)
H.P. Acthar Gel 40 U (0.5 mL) daily
acthar H.P. Acthar Gel
Acthar given SC (40 U daily or 80 U every other day) for Weeks 1-4 and tapered for Weeks 5-8
(Placebo Comparator)
Placebo (0.5 mL) daily
placebo for acthar
Placebo contains the same inactive ingredients as H.P. Acthar Gel with the API. Placebo is given SC for 6 months in equal volume as the Acthar comparator.
(Experimental)
H.P. Acthar Gel 80 U (1.0 mL) every other day
acthar H.P. Acthar Gel
Acthar given SC (40 U daily or 80 U every other day) for Weeks 1-4 and tapered for Weeks 5-8
(Placebo Comparator)
Placebo (1.0 mL) every other day
placebo for acthar
Placebo contains the same inactive ingredients as H.P. Acthar Gel with the API. Placebo is given SC for 6 months in equal volume as the Acthar comparator.

Primary Outcomes

Measure
Proportion of patients that meet the definition of a responder
time frame: Week 4

Secondary Outcomes

Measure
Proportion of patients meeting responder definition at Week 8 and Week 52
time frame: Week 8 and Week 52
Change from baseline in SELENA-SLEDAI at Weeks 2, 4, 6, and 8
time frame: Weeks 2, 4, 6, and 8
Change from baseline in BILAG at Weeks 4 and 8
time frame: Weeks 4 and 8
Change from baseline in tender and swollen joint counts at Weeks 4 and 8
time frame: Weeks 4 and 8
Change from baseline in cutaneous lupus activity as measured by the CLASI at Weeks 4 and 8
time frame: Weeks 4 and 8
Change from baseline in SF-36 and Krupp-Fatigue Assessment in Weeks 4 and 8
time frame: Weeks 4 and 8
Time and rate of flare based on SFI and BILAG
time frame: Week 52
Proportion of patients with a relapse
time frame: Week 52

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Male or female ≥ 18 years of age at screening who are able to provide informed consent - Diagnosis of SLE according to the American College of Rheumatology revised criteria (fulfilled ≥ 4 criteria) - Active SLE with arthritic and/or cutaneous involvement as demonstrated by a SELENA-SLEDAI score ≥ 2 (clinical manifestation must include rash and/or arthritis) - Moderate to severe rash and/or arthritis as demonstrated by BILAG score A or B in the mucocutaneous and/or musculoskeletal body systems - Documented history of autoantibodies to at least one of the following: anti-dsDNA, anti-Smith, or anti-cardiolipin - Documented history of positive antinuclear antibody (ANA) - Currently on a stable dose of prednisone (7.5 to 30 mg/day of prednisone or equivalent within the 4 weeks prior to screening). The prednisone regimen must remain stable through the double-blind phase and until the stable Acthar regimen is attained in the open-label phase. Exclusion Criteria: - Patients with a recent history (≤ 2 months prior to screening) of starting prednisone (or equivalent) use - Patients with active nephritis defined as serum creatinine > 2.5 mg/dL or protein creatinine ratio (PCR) > 1.5 g/g, or patients that required hemodialysis within 3 months prior to screening - Active central nervous system (CNS) lupus (including seizures, psychosis, organic brain syndrome, cerebrovascular accident, cerebritis, or CNS vasculitis), requiring therapeutic intervention within 3 months prior to screening - Type 1 or type 2 diabetes mellitus (history of gestational diabetes mellitus is not an exclusion), or patients currently taking hypoglycemic medication - History of using certain medications prior to screening: 1. oral prednisone (or equivalent) > 30 mg/day, any steroid injection, cyclosporine, or non-biologic investigational drug within 3 months prior to screening 2. IVIg or plasmapheresis within 4 months prior to screening 3. cyclophosphamide within 6 months prior to screening; and/or 4. B-cell targeted therapy, abatacept, or any biologic investigational agent within 12 months prior to screening - Contraindication per Acthar Prescribing Information: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction 1. For the purposes of this study, osteoporosis is defined as evidence of vertebral or long bone fracture or vertebral T-score > 2.0 2. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening 3. For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV

Additional Information

Official title A Two-part Study Exploring the Efficacy, Safety, and Pharmacodynamics of Acthar in Systemic Lupus Erythematosus Patients With a History of Persistently Active Disease
Description The first phase of the study is an 8-week randomized, double-blind, placebo-controlled, parallel-group add-on pilot study exploring the efficacy, safety, and pharmacodynamics of Acthar in SLE patients with a history of persistently active disease with arthritic and/or cutaneous involvement despite standard of care, which includes chronic/stable prednisone use for a minimum of 4 weeks prior to screening. Patients will be randomized to one of four possible treatment groups (Acthar [low dose (40 U [0.5 mL]) or high dose (80 U [1.0 mL])] or equivalent volumes of Placebo [low or high 'dose']) in a 2:1:2:1 ratio (Acthar:Placebo:Acthar:Placebo). In Weeks 1-4, patients will administer study medication via subcutaneous (SC) injection 0.5 mL daily or 1.0 mL every other day based on randomization. In Weeks 5-8, patients will taper the study medication. Patients will continue on their stable steroid regimen during this phase of the study. After completion of Week 8 in the double-blind phase, patients may choose to participate in the optional open-label phase where they will receive an Acthar maintenance regimen for 44 weeks. The initial Acthar regimen will be assigned based on the study medication regimen the patient received at the completion of the double-blind phase (Visit 6, Week 8). Acthar regimen may be adjusted based on the Investigator's judgment with the goal of achieving a stable Acthar regimen no later than Week 28. Once the stable Acthar regimen is achieved, the Investigator should consider tapering the steroid regimen to a low daily dose or completely discontinue.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Mallinckrodt.