Systemic Lupus Erythematosus (SLE)
Conditions
Keywords
SLE, Lupus
Brief 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 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
DRUGActhar
Acthar is given by subcutaneous (SC) injection (shot under the skin), at a dose of 40 units daily or 80 units every other day
DRUGPlacebo
Placebo contains the same inactive ingredients as Acthar, and is given by SC injection
DRUGSteroid Drug
The patient's steroid regimen 7.5 to 30 mg/day of prednisone or equivalent, chronic/stable within the 4 weeks prior to screening.
Sponsors
Mallinckrodt
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Meet the Definition of a Responder Within 4 Weeks | within 4 weeks | 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
| Measure | Time frame | Description |
|---|---|---|
| Score on the SELENA-SLEDAI Within 8 Weeks | within 8 weeks | 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 |
| BILAG Total Score Within 8 Weeks | within 8 weeks | 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 |
| Physician's Global Assessment (PGA) of Disease Severity at Baseline | at Baseline | 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. |
| Physician's Global Assessment (PGA) of Disease Severity at Week 4 | at Week 4 | 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. |
| Physician's Global Assessment (PGA) of Disease Severity at Week 8 | at Week 8 | 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. |
| Number of Tender or Swollen Joints Within 8 Weeks | at Baseline, Week 4, and Week 8 (within 8 weeks) | The doctor counted the number of tender or swollen joints at Baseline, at Week 4, and at Week 8 |
| Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Baseline, Week 4 and Week 8 (within 8 weeks) | 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 |
| Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Baseline, Week 4 and Week 8 (within 8 weeks) | 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 |
| Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline, Week 4 and Week 8 (within 8 weeks) | 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 |
| Number of Participants Who Meet the Definition of a Responder Within 8 Weeks | within 8 weeks | 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 |
| Number of Participants Who Meet the Definition of a Responder at Week 52 | at Week 52 | 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 |
| Score on the SELENA-SLEDAI at Week 52 | at Week 52 | 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. |
| Physician's Global Assessment (PGA) of Disease Severity at Week 52 | at Week 52 | 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. |
| Number of Tender or Swollen Joints at Week 52 | at Week 52 | The doctor counted the number of tender or swollen joints at Week 52. |
| Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) at Week 52 | at Week 52 | 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. |
| Krupp Fatigue Severity Score (FSS) at Week 52 | at Week 52 | 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). |
| Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | at Week 52 | 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. |
| Mean Score on the Mental Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | at Week 52 | 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. |
| Number of Participants With a Relapse Within 52 Weeks | within 52 weeks | — |
| Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline, Week 4 and Week 8 (within 8 weeks) | 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. |
Countries
United States
Participant flow
Recruitment details
38 enrolled participants were randomized 2:1:2:1 into four treatment groups, to receive Acthar 0.5 mL daily: Placebo 0.5 mL daily: Acthar 1 mL every other day: Placebo 1 mL every other day, during the double-blind period, along with their stable dose of steroids.
Pre-assignment details
All participants were to receive their stable steroid regimen in addition to the study drug throughout the double-blind period (Period 1). If they completed Period 1, they were invited to participate in an open label period (Period 2). Those who received Placebo went into Placebo/Acthar, and those who received Acthar went into Acthar/Acthar.
Participants by arm
| Arm | Count |
|---|---|
| Placebo Participants who were randomized to receive Placebo in Period 1 | 11 |
| Acthar Participants who were randomized to receive Acthar in Period 1 | 25 |
| Total | 36 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Double-blind Period (8 Weeks) | Adverse Event | 0 | 2 | 0 | 0 |
| Double-blind Period (8 Weeks) | Protocol Violation | 0 | 1 | 0 | 0 |
| Double-blind Period (8 Weeks) | Withdrawal by Subject | 1 | 1 | 0 | 0 |
| Open Label Period (44 Weeks) | Adverse Event | 0 | 0 | 3 | 4 |
| Open Label Period (44 Weeks) | Lack of Efficacy | 0 | 0 | 1 | 1 |
| Open Label Period (44 Weeks) | Reason not provided | 0 | 0 | 0 | 2 |
| Open Label Period (44 Weeks) | Withdrawal by Subject | 0 | 0 | 0 | 2 |
Baseline characteristics
| Characteristic | Placebo | Acthar | Total |
|---|---|---|---|
| Age, Continuous | 39 years | 43 years | 41.5 years |
| Race/Ethnicity, Customized Black or African American | 6 Participants | 18 Participants | 24 Participants |
| Race/Ethnicity, Customized Hispanic or Latino Ethnicity | 0 Participants | 6 Participants | 6 Participants |
| Race/Ethnicity, Customized Other | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized White or Caucasian | 5 Participants | 6 Participants | 11 Participants |
| Region of Enrollment United States | 11 participants | 25 participants | 36 participants |
| Sex: Female, Male Female | 10 Participants | 24 Participants | 34 Participants |
| Sex: Female, Male Male | 1 Participants | 1 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 9 / 11 | 19 / 25 | 8 / 11 | 19 / 22 |
| serious Total, serious adverse events | 0 / 11 | 3 / 25 | 4 / 11 | 2 / 22 |
Outcome results
Primary
Number of Participants Who Meet the Definition of a Responder Within 4 Weeks
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.
Time frame: within 4 weeks
Population: mITT
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Number of Participants Who Meet the Definition of a Responder Within 4 Weeks | 3 Participants |
| Acthar | Number of Participants Who Meet the Definition of a Responder Within 4 Weeks | 4 Participants |
Secondary
BILAG Total Score Within 8 Weeks
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
Time frame: within 8 weeks
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | BILAG Total Score Within 8 Weeks | at Baseline | 15.4 score on a scale | Standard Deviation 9.55 |
| Placebo | BILAG Total Score Within 8 Weeks | at Week 4 | 10.3 score on a scale | Standard Deviation 7.8 |
| Placebo | BILAG Total Score Within 8 Weeks | at Week 8 | 13.5 score on a scale | Standard Deviation 8.82 |
| Acthar | BILAG Total Score Within 8 Weeks | at Baseline | 15.7 score on a scale | Standard Deviation 5.93 |
| Acthar | BILAG Total Score Within 8 Weeks | at Week 4 | 9.2 score on a scale | Standard Deviation 5.36 |
| Acthar | BILAG Total Score Within 8 Weeks | at Week 8 | 6.8 score on a scale | Standard Deviation 4.31 |
Secondary
Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) at Week 52
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.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) at Week 52 | 0.4 score on a scale | Standard Deviation 0.79 |
| Acthar | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) at Week 52 | 1.3 score on a scale | Standard Deviation 1.55 |
Secondary
Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks
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
Time frame: at Baseline, Week 4 and Week 8 (within 8 weeks)
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Baseline | 6.1 score on a scale | Standard Deviation 6.63 |
| Placebo | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Week 4 | 6.3 score on a scale | Standard Deviation 4.6 |
| Placebo | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Week 8 | 5.7 score on a scale | Standard Deviation 6.87 |
| Acthar | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Baseline | 6.4 score on a scale | Standard Deviation 6.33 |
| Acthar | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Week 4 | 4.8 score on a scale | Standard Deviation 4.08 |
| Acthar | Cutaneous Lupus Activity as Measured by the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Within 8 Weeks | at Week 8 | 3.7 score on a scale | Standard Deviation 4.24 |
Secondary
Krupp Fatigue Severity Score (FSS) at Week 52
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).
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Krupp Fatigue Severity Score (FSS) at Week 52 | 4.523 score on a scale | Standard Deviation 1.5491 |
| Acthar | Krupp Fatigue Severity Score (FSS) at Week 52 | 4.743 score on a scale | Standard Deviation 2.0428 |
Secondary
Krupp Fatigue Severity Score (FSS) Within 8 Weeks
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
Time frame: at Baseline, Week 4 and Week 8 (within 8 weeks)
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Baseline | 5.374 score on a scale | Standard Deviation 0.984 |
| Placebo | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Week 4 | 5.379 score on a scale | Standard Deviation 1.0697 |
| Placebo | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Week 8 | 5.404 score on a scale | Standard Deviation 1.1699 |
| Acthar | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Baseline | 5.648 score on a scale | Standard Deviation 1.3091 |
| Acthar | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Week 4 | 5.298 score on a scale | Standard Deviation 1.5401 |
| Acthar | Krupp Fatigue Severity Score (FSS) Within 8 Weeks | at Week 8 | 5.152 score on a scale | Standard Deviation 1.7858 |
Secondary
Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks
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.
Time frame: at Baseline, Week 4 and Week 8 (within 8 weeks)
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline | 41.304 score on a scale | Standard Deviation 11.844 |
| Placebo | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 4 | 38.744 score on a scale | Standard Deviation 16.8434 |
| Placebo | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 8 | 39.256 score on a scale | Standard Deviation 18.4576 |
| Acthar | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline | 38.406 score on a scale | Standard Deviation 14.6618 |
| Acthar | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 4 | 40.280 score on a scale | Standard Deviation 13.0274 |
| Acthar | Mean Score on the Mental Component Scale (MCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 8 | 40.408 score on a scale | Standard Deviation 15.8738 |
Secondary
Mean Score on the Mental Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52
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.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Mean Score on the Mental Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | 45.272 score on a scale | Standard Deviation 12.6597 |
| Acthar | Mean Score on the Mental Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | 39.700 score on a scale | Standard Deviation 16.3027 |
Secondary
Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52
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.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | 43.618 score on a scale | Standard Deviation 11.9596 |
| Acthar | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) at Week 52 | 39.710 score on a scale | Standard Deviation 10.8066 |
Secondary
Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks
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
Time frame: at Baseline, Week 4 and Week 8 (within 8 weeks)
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline | 32.927 score on a scale | Standard Deviation 12.3561 |
| Placebo | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 4 | 32.831 score on a scale | Standard Deviation 13.539 |
| Placebo | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 8 | 33.310 score on a scale | Standard Deviation 12.976 |
| Acthar | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Baseline | 31.526 score on a scale | Standard Deviation 11.7065 |
| Acthar | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 4 | 35.318 score on a scale | Standard Deviation 12.5159 |
| Acthar | Mean Score on the Physical Component Scale (PCS) of the Short Form 36 Health Status Questionnaire (SF-36) Within 8 Weeks | at Week 8 | 35.701 score on a scale | Standard Deviation 11.8032 |
Secondary
Number of Participants Who Meet the Definition of a Responder at Week 52
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
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Number of Participants Who Meet the Definition of a Responder at Week 52 | 4 Participants |
| Acthar | Number of Participants Who Meet the Definition of a Responder at Week 52 | 3 Participants |
Secondary
Number of Participants Who Meet the Definition of a Responder Within 8 Weeks
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
Time frame: within 8 weeks
Population: mITT
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Number of Participants Who Meet the Definition of a Responder Within 8 Weeks | 3 Participants |
| Acthar | Number of Participants Who Meet the Definition of a Responder Within 8 Weeks | 11 Participants |
Secondary
Number of Participants With a Relapse Within 52 Weeks
Time frame: within 52 weeks
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Number of Participants With a Relapse Within 52 Weeks | 1 Participants |
| Acthar | Number of Participants With a Relapse Within 52 Weeks | 6 Participants |
Secondary
Number of Tender or Swollen Joints at Week 52
The doctor counted the number of tender or swollen joints at Week 52.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Number of Tender or Swollen Joints at Week 52 | 1.1 Tender or Swollen Joints | Standard Deviation 2.27 |
| Acthar | Number of Tender or Swollen Joints at Week 52 | 0.7 Tender or Swollen Joints | Standard Deviation 2.21 |
Secondary
Number of Tender or Swollen Joints Within 8 Weeks
The doctor counted the number of tender or swollen joints at Baseline, at Week 4, and at Week 8
Time frame: at Baseline, Week 4, and Week 8 (within 8 weeks)
Population: mITT
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Number of Tender or Swollen Joints Within 8 Weeks | at Week 4 | 3.8 Tender or Swollen Joints | Standard Deviation 4.34 |
| Placebo | Number of Tender or Swollen Joints Within 8 Weeks | at Week 8 | 4.0 Tender or Swollen Joints | Standard Deviation 6.18 |
| Placebo | Number of Tender or Swollen Joints Within 8 Weeks | at Baseline | 6.2 Tender or Swollen Joints | Standard Deviation 5.4 |
| Acthar | Number of Tender or Swollen Joints Within 8 Weeks | at Baseline | 9.6 Tender or Swollen Joints | Standard Deviation 6.9 |
| Acthar | Number of Tender or Swollen Joints Within 8 Weeks | at Week 4 | 4.5 Tender or Swollen Joints | Standard Deviation 4.94 |
| Acthar | Number of Tender or Swollen Joints Within 8 Weeks | at Week 8 | 3.5 Tender or Swollen Joints | Standard Deviation 5.89 |
Secondary
Physician's Global Assessment (PGA) of Disease Severity at Baseline
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.
Time frame: at Baseline
Population: mITT
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Mild | 1 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Severe | 2 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Moderate | 8 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Missing | 0 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Baseline | None | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Missing | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Baseline | None | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Mild | 2 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Moderate | 19 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Baseline | Severe | 4 Participants |
Secondary
Physician's Global Assessment (PGA) of Disease Severity at Week 4
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.
Time frame: at Week 4
Population: mITT
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Mild | 5 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Severe | 2 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Moderate | 3 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Missing | 1 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | None | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Missing | 3 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | None | 1 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Mild | 9 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Moderate | 11 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 4 | Severe | 1 Participants |
Secondary
Physician's Global Assessment (PGA) of Disease Severity at Week 52
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.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Mild | 7 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Severe | 0 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Moderate | 0 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Missing | 4 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | None | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Missing | 9 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | None | 4 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Mild | 7 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Moderate | 2 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 52 | Severe | 0 Participants |
Secondary
Physician's Global Assessment (PGA) of Disease Severity at Week 8
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.
Time frame: at Week 8
Population: mITT
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Mild | 5 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Severe | 3 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Moderate | 3 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Missing | 0 Participants |
| Placebo | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | None | 0 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Missing | 3 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | None | 2 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Mild | 12 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Moderate | 7 Participants |
| Acthar | Physician's Global Assessment (PGA) of Disease Severity at Week 8 | Severe | 1 Participants |
Secondary
Score on the SELENA-SLEDAI at Week 52
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.
Time frame: at Week 52
Population: mITT with necessary data at Week 52
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Placebo | Score on the SELENA-SLEDAI at Week 52 | 3 score on a scale |
| Acthar | Score on the SELENA-SLEDAI at Week 52 | 4 score on a scale |
Secondary
Score on the SELENA-SLEDAI Within 8 Weeks
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
Time frame: within 8 weeks
Population: mITT
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Placebo | Score on the SELENA-SLEDAI Within 8 Weeks | Week 2 | 10.0 score on a scale |
| Placebo | Score on the SELENA-SLEDAI Within 8 Weeks | Week 4 | 9.0 score on a scale |
| Placebo | Score on the SELENA-SLEDAI Within 8 Weeks | Week 6 | 8.0 score on a scale |
| Placebo | Score on the SELENA-SLEDAI Within 8 Weeks | Week 8 | 9.0 score on a scale |
| Acthar | Score on the SELENA-SLEDAI Within 8 Weeks | Week 8 | 6.0 score on a scale |
| Acthar | Score on the SELENA-SLEDAI Within 8 Weeks | Week 2 | 8.0 score on a scale |
| Acthar | Score on the SELENA-SLEDAI Within 8 Weeks | Week 6 | 6.0 score on a scale |
| Acthar | Score on the SELENA-SLEDAI Within 8 Weeks | Week 4 | 8.0 score on a scale |