Acthar for the Treatment of Systemic Lupus Erythematosus in Patients With a History of Persistently Active Disease

This study is currently recruiting participants.
Verified April 2013 by Questcor Pharmaceuticals, Inc.
Sponsor:
Information provided by (Responsible Party):
Questcor Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01753401
First received: December 17, 2012
Last updated: April 8, 2013
Last verified: April 2013
  Purpose

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.


Condition Intervention Phase
Systemic Lupus Erythematosus (SLE)
Drug: Acthar
Drug: Placebo for Acthar
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Questcor Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Proportion of patients that meet the definition of a responder [ Time Frame: Week 4 ] [ Designated as safety issue: No ]

    Proportion of patients meeting responder definition at Week 4:

    • decrease in SELENA-SLEDAI score from 4 to 0 for arthritis and no worsening in other organ systems based on BILAG OR
    • decrease in SELENA-SLEDAI score from 2 to 0 for rash and no worsening in other organ systems based on BILAG


Secondary Outcome Measures:
  • Proportion of patients meeting responder definition at Week 8 and Week 52 [ Time Frame: Week 8 and Week 52 ] [ Designated as safety issue: No ]

    Proportion of patients meeting responder definition at Week 8 and Week 52:

    • decrease in SELENA-SLEDAI score from 4 to 0 for arthritis and no worsening in other organ systems based on BILAG OR
    • decrease in SELENA-SLEDAI score from 2 to 0 for rash and no worsening in other organ systems based on BILAG

  • Change from baseline in SELENA-SLEDAI at Weeks 2, 4, 6, and 8 [ Time Frame: Weeks 2, 4, 6, and 8 ] [ Designated as safety issue: No ]
  • Change from baseline in BILAG at Weeks 4 and 8 [ Time Frame: Weeks 4 and 8 ] [ Designated as safety issue: No ]
  • Change from baseline in tender and swollen joint counts at Weeks 4 and 8 [ Time Frame: Weeks 4 and 8 ] [ Designated as safety issue: No ]
  • Change from baseline in cutaneous lupus activity as measured by the CLASI at Weeks 4 and 8 [ Time Frame: Weeks 4 and 8 ] [ Designated as safety issue: No ]
  • Change from baseline in SF-36 and Krupp-Fatigue Assessment in Weeks 4 and 8 [ Time Frame: Weeks 4 and 8 ] [ Designated as safety issue: No ]
  • Time and rate of flare based on SFI and BILAG [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
  • Proportion of patients with a relapse [ Time Frame: Week 52 ] [ Designated as safety issue: No ]

Estimated Enrollment: 36
Study Start Date: December 2012
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 0.5 mL Acthar
H.P. Acthar Gel 40 U (0.5 mL) daily
Drug: Acthar
Acthar given SC (40 U daily or 80 U every other day) for Weeks 1-4 and tapered for Weeks 5-8
Other Names:
  • H.P. Acthar Gel
  • Repository Corticotropin Injection
  • ACTH
Placebo Comparator: 0.5 mL Placebo
Placebo (0.5 mL) daily
Drug: 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: 1.0 mL Acthar
H.P. Acthar Gel 80 U (1.0 mL) every other day
Drug: Acthar
Acthar given SC (40 U daily or 80 U every other day) for Weeks 1-4 and tapered for Weeks 5-8
Other Names:
  • H.P. Acthar Gel
  • Repository Corticotropin Injection
  • ACTH
Placebo Comparator: 1.0 mL Placebo
Placebo (1.0 mL) every other day
Drug: 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.

Detailed 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01753401

Contacts
Contact: Shanique Smythe, MS 443-973-2012 shanique.smythe@questcor.com

Locations
United States, California
University of California San Diego Not yet recruiting
La Jolla, California, United States, 92037
Principal Investigator: Kenneth Kalunian, MD            
Valerius Medical Group and Research Center Recruiting
Long Beach, California, United States, 90806
Principal Investigator: Nathaniel Neal, MD            
United States, Florida
Clinical Research of West Florida, Inc Recruiting
Clearwater, Florida, United States, 33765
Principal Investigator: Robert Levin, MD            
Burnette & Silverfield MDs, PLC Recruiting
Tampa, Florida, United States, 33614
Principal Investigator: Michael Burnette, MD            
United States, Indiana
Memorial Health System Inc Recruiting
Granger, Indiana, United States, 46530
Principal Investigator: Nicholas Straniero, MD            
United States, Louisiana
Ochsner Clinic Foundation Not yet recruiting
Baton Rouge, Louisiana, United States, 70809
Principal Investigator: Stephen Lindsey, MD            
United States, Maryland
Johns Hopkins University Not yet recruiting
Baltimore, Maryland, United States, 21209
Principal Investigator: Michelle Petri, MD            
United States, Michigan
Clinical Trials Office - Henry Ford Health System Not yet recruiting
Detroit, Michigan, United States, 48202
Principal Investigator: Kathleen McKinnon, MD            
Fiechtner Research, Inc. Recruiting
Lansing, Michigan, United States, 48910
Principal Investigator: Justus Fiechtner, MD            
United States, New York
North Shore-Long Island Health System Recruiting
Lake Success, New York, United States, 11042
Principal Investigator: Richard Furie, MD            
United States, North Carolina
Arthritis and Rheumatology of the Carolinas, PLLC Recruiting
Charlotte, North Carolina, United States, 28210
Principal Investigator: Emily Jane Herron Box, MD            
United States, Pennsylvania
Penn State University Not yet recruiting
Hershey, Pennsylvania, United States, 17033
Principal Investigator: Nancy Olsen, MD            
United States, Texas
Rheumatic Disease Clinical Research Recruiting
Houston, Texas, United States, 77004
Principal Investigator: Samuel Pegram, MD            
Accurate Clinical Research Recruiting
Houston, Texas, United States, 77034
Principal Investigator: Philip Waller, MD            
Sponsors and Collaborators
Questcor Pharmaceuticals, Inc.
Investigators
Principal Investigator: Richard Furie, MD North Shore Long Island Health System
  More Information

No publications provided

Responsible Party: Questcor Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01753401     History of Changes
Other Study ID Numbers: QSC01-SLE-01
Study First Received: December 17, 2012
Last Updated: April 8, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Questcor Pharmaceuticals, Inc.:
SLE
Lupus

Additional relevant MeSH terms:
Lupus Erythematosus, Systemic
Connective Tissue Diseases
Autoimmune Diseases
Immune System Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013