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Trial · Interventional

CompletedPhase 4Results postedRegistry data

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

NCTNCT01753401 (opens ClinicalTrials.gov in a new tab)Other idQSC01-SLE-01
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Overview

Status
Completed
Phase
Phase 4
Enrollment
38 participants (actual)
Sponsor
Mallinckrodt · Industry
Design
Randomized · Double masking
Countries
1 country

United States

Start
Jan 2013
Primary completion
Oct 2015
Completion
Oct 2015
First posted
20 Dec 2012 (estimated)
Last update posted
27 Feb 2020
Conditions
Systemic Lupus Erythematosus (SLE)

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2013201420152016
  1. Ph 4

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

    NCT01753401

    Jan 2013 to Oct 2015

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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 periods: an 8-week double-blind period, to provide placebo-controlled safety, efficacy, and pharmacodynamic data, and an optional open-label period, to examine the prolonged effects of Acthar maintenance.

Interventions

  • ActharDrug
  • PlaceboDrug
  • Steroid DrugDrug

Arms4

  • Experimental

    Period 2: Placebo/Acthar

    Participants receive Placebo in Part 1, but after completion of Week 8 in the double-blind phase, patients who received Placebo 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 during Part 2 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.

  • Experimental

    Period 2: Acthar/Acthar

    After completion of Week 8 in the double-blind phase, patients who received Acthar 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.

  • Placebo comparator

    Period 1: Placebo

    Participants receive matching placebo (in 0.5 mL daily or in 1 mL every other day) for 4 weeks. In Weeks 5-8, they will taper the study medication. Participants will continue on their stable steroid regimen during this phase of the study. After completion of Week 8 in the double-blind phase, they may choose to participate in the optional open-label phase. Participants will continue on their stable steroid regimen during this phase of the study.

  • Experimental

    Period 1: Acthar

    Participants receive Acthar (40 units in 0.5 mL daily or 80 units in 1 mL every other day) for 4 weeks. In Weeks 5-8, they will taper the study medication. Participants will continue on their stable steroid regimen during this phase of the study. After completion of Week 8 in the double-blind phase, they may choose to participate in the optional open-label phase. Participants will continue on their stable steroid regimen during this phase of the study.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Number of Participants Who Meet the Definition of a Responder Within 4 Weeks

    Time frame · within 4 weeks

    Registry description

    Participants are counted as responders based on two SLE indices: the Systemic Lupus Erythematosus Disease Activity Index amended by the SELENA group (SELENA-SLEDAI) and the British Isles Lupus Assessment Group (BILAG) Index. * decrease in SELENA-SLEDAI score from 4 to 0 for the arthritis descriptor (highest possible score is 4) and no worsening in other organ systems based on BILAG OR * decrease in SELENA-SLEDAI score from 2 to 0 for rash (highest possible score is 2) and no worsening in other organ systems based on BILAG The BILAG is a transitional index that captures changing severity of clinical manifestations. It has an ordinal scale scoring system by design that produces an overview of disease activity across eight systems. The individual system scores were not intended to be summated into a global score.

Secondary outcomes(20)

  1. Number of Participants Who Meet the Definition of a Responder Within 8 Weeks

    Time frame · within 8 weeks

    Registry description

    Participants are counted as responders based on: * 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

  2. Score on the SELENA-SLEDAI Within 8 Weeks

    Time frame · within 8 weeks

    Registry description

    SLEDAI was modeled on the basis of clinician global judgment. A participant's SELENA-SLEDAI total score is the sum of all marked SLE-related descriptors on a checklist developed by the SELENA Group (also referred to as hybrid SLEDAI). The scores of the descriptors range from 0 to 8. A total score can fall between 0 and 105, with a higher score representing a more significant degree of disease activity. Rows: Week 2, Week 4, Week 6, Week 8

  3. BILAG Total Score Within 8 Weeks

    Time frame · within 8 weeks

    Registry description

    The BILAG is a transitional index that captures changing severity of clinical manifestations that produces an overview of disease activity across eight systems. The 8 systems are scored on a scale from 0=not present to 4=worse, for the 4 week period before the assessment. The lowest possible score is 0, and the highest possible score is 32. A higher score means the symptoms are worse. Rows: Baseline, Week 4, Week 8

  4. Physician's Global Assessment (PGA) of Disease Severity at Baseline

    Time frame · at Baseline

    Registry description

    PGA of disease severity on a 100 mm visual analogue scale are categorized to the following: 0 point (none) = 0 mm; 1 point (mild) = \>0 - 33.33 mm; 2 points (moderate) = \>33.33 - 66.67 mm; and 3 points (severe) = \>66.67 - 100 mm. The count of participants in each category is reported.

  5. Physician's Global Assessment (PGA) of Disease Severity at Week 4

    Time frame · at Week 4

    Registry description

    PGA of disease severity on a 100 mm visual analogue scale are categorized to the following: 0 point (none) = 0 mm; 1 point (mild) = \>0 - 33.33 mm; 2 points (moderate) = \>33.33 - 66.67 mm; and 3 points (severe) = \>66.67 - 100 mm. The count of participants in each category is reported.

  6. Physician's Global Assessment (PGA) of Disease Severity at Week 8

    Time frame · at Week 8

    Registry description

    PGA of disease severity on a 100 mm visual analogue scale are categorized to the following: 0 point (none) = 0 mm; 1 point (mild) = \>0 - 33.33 mm; 2 points (moderate) = \>33.33 - 66.67 mm; and 3 points (severe) = \>66.67 - 100 mm. The count of participants in each category is reported.

  7. Number of Tender or Swollen Joints Within 8 Weeks

    Time frame · at Baseline, Week 4, and Week 8 (within 8 weeks)

    Registry description

    The doctor counted the number of tender or swollen joints at Baseline, at Week 4, and at Week 8

  8. Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks

    Time frame · at Baseline, Week 4 and Week 8 (within 8 weeks)

    Registry description

    The CLASI consists of two scores the first summarizes the activity of the disease while the second is a measure of the damage done by the disease. Activity is scored on the basis of erythema, scale/hyperkeratosis, mucous membrane involvement, acute hair loss and non-scarring alopecia. The CLASI score ranges from 0 to 70, with higher scores indicating more severe skin disease. Rows: at Baseline, at Week 4, at Week 8

  9. Krupp Fatigue Severity Score (FSS) Within 8 Weeks

    Time frame · at Baseline, Week 4 and Week 8 (within 8 weeks)

    Registry description

    The Krupp FSS is a scale to rate disability-related fatigue. Respondents use a scale ranging from 1 ("completely disagree") to 7 ("completely agree") to indicate their agreement with nine statements about fatigue. A visual analogue scale is also included with the scale; respondents are asked to denote the severity of their fatigue over the past 2 weeks by placing a mark on a line extending from "no fatigue" to "fatigue as bad as could be." Higher scores on the scale are indicative of more severe fatigue. This validated fatigue severity scale measures impact of fatigue with a 9-item questionnaire, with a 7-point Likert scale for each question. Total score ranges from 0 (best possible outcome) to 63 (worst possible fatigue). Rows: at Baseline, at Week 4, at Week 8

  10. Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks

    Time frame · at Baseline, Week 4 and Week 8 (within 8 weeks)

    Registry description

    The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the PCS score of the SF-36. Items 5-8 primarily contribute to the MCS score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best). Higher scores indicate improvement. Rows: at Baseline, at Week 4, at Week 8

  11. Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks

    Time frame · at Baseline, Week 4 and Week 8 (within 8 weeks)

    Registry description

    The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the PCS score of the SF-36. Items 5-8 primarily contribute to the MCS score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best). Higher scores indicate improvement.

  12. Number of Participants Who Meet the Definition of a Responder at Week 52

    Time frame · at Week 52

    Registry description

    Participants are counted as responders based on: * 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

  13. Score on the SELENA-SLEDAI at Week 52

    Time frame · at Week 52

    Registry description

    SLEDAI was modeled on the basis of clinician global judgment. A participant's SELENA-SLEDAI total score is the sum of all marked SLE-related descriptors on a checklist developed by the SELENA Group (also referred to as hybrid SLEDAI). The scores of the descriptors range from 0 to 8. A total score can fall between 0 and 105, with a higher score representing a more significant degree of disease activity.

  14. Physician's Global Assessment (PGA) of Disease Severity at Week 52

    Time frame · at Week 52

    Registry description

    PGA of disease severity on a 100 mm visual analogue scale are categorized to the following: 0 point (none) = 0 mm; 1 point (mild) = \>0 - 33.33 mm; 2 points (moderate) = \>33.33 - 66.67 mm; and 3 points (severe) = \>66.67 - 100 mm. The count of participants in each category is reported.

  15. Number of Tender or Swollen Joints at Week 52

    Time frame · at Week 52

    Registry description

    The doctor counted the number of tender or swollen joints at Week 52.

  16. Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) at Week 52

    Time frame · at Week 52

    Registry description

    The CLASI consists of two scores the first summarizes the activity of the disease while the second is a measure of the damage done by the disease. Activity is scored on the basis of erythema, scale/hyperkeratosis, mucous membrane involvement, acute hair loss and non-scarring alopecia. The CLASI score ranges from 0 to 70, with higher scores indicating more severe skin disease.

  17. Krupp Fatigue Severity Score (FSS) at Week 52

    Time frame · at Week 52

    Registry description

    The Krupp FSS is a scale to rate disability-related fatigue. Respondents use a scale ranging from 1 ("completely disagree") to 7 ("completely agree") to indicate their agreement with nine statements about fatigue. A visual analogue scale is also included with the scale; respondents are asked to denote the severity of their fatigue over the past 2 weeks by placing a mark on a line extending from "no fatigue" to "fatigue as bad as could be." Higher scores on the scale are indicative of more severe fatigue. This validated fatigue severity scale measures impact of fatigue with a 9-item questionnaire, with a 7-point Likert scale for each question. Total score ranges from 0 (best possible outcome) to 63 (worst possible fatigue).

  18. Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52

    Time frame · at Week 52

    Registry description

    The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the PCS score of the SF-36. Items 5-8 primarily contribute to the MCS score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best). Higher scores indicate improvement.

  19. Mean Score on the Mental Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52

    Time frame · at Week 52

    Registry description

    The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the PCS score of the SF-36. Items 5-8 primarily contribute to the MCS score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best). Higher scores indicate improvement.

  20. Number of Participants With a Relapse Within 52 Weeks

    Time frame · within 52 weeks

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· within 4 weeks

Number of Participants Who Meet the Definition of a Responder Within 4 Weeks

GroupValue (Participants)
Placebo3
Acthar4
Secondary and other outcome results(7)
Secondary outcomeCount of participants· within 8 weeks

Number of Participants Who Meet the Definition of a Responder Within 8 Weeks

GroupValue (Participants)
Placebo3
Acthar11
Secondary outcomeMedian· within 8 weeks

Score on the SELENA-SLEDAI Within 8 Weeks

GroupValue (score on a scale)
Placebo10.0
Acthar8.0
Secondary outcomeMean· within 8 weeks

BILAG Total Score Within 8 Weeks

GroupValue (score on a scale)Spread
Placebo15.49.55
Acthar15.75.93
Secondary outcomeCount of participants· at Baseline

Physician's Global Assessment (PGA) of Disease Severity at Baseline

GroupValue (Participants)
Placebo0
Acthar0
Secondary outcomeCount of participants· at Week 4

Physician's Global Assessment (PGA) of Disease Severity at Week 4

GroupValue (Participants)
Placebo0
Acthar1
Secondary outcomeCount of participants· at Week 8

Physician's Global Assessment (PGA) of Disease Severity at Week 8

GroupValue (Participants)
Placebo0
Acthar2
Secondary outcomeMean· at Baseline, Week 4, and Week 8 (within 8 weeks)

Number of Tender or Swollen Joints Within 8 Weeks

GroupValue (Tender or Swollen Joints)Spread
Placebo6.25.40
Acthar9.66.90

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Period 1: PlaceboParticipants who receive Placebo in Period 1–0 / 119 / 11
Period 1: ActharParticipants who receive Acthar in Period 1–3 / 2519 / 25
Period 2: Placebo/ActharParticipants who receive Placebo in Period 1, but Acthar in Period 2–4 / 118 / 11
Period 2: Acthar/ActharParticipants who receive Acthar in both Periods 1 and 2–2 / 2219 / 22

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedHannon CW, McCourt C, Lima HC, Chen S, Bennett C. Interventions for cutaneous disease in systemic lupus erythematosus. Cochrane Database Syst Rev. 2021 Mar 9;3(3):CD007478. doi: 10.1002/14651858.CD007478.pub2. PMID 33687069

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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. intravenous immunoglobulin (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

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