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A Study of Ustekinumab in Participants With Active Systemic Lupus Erythematosus

Phase 3
Terminated
Conditions
Lupus Erythematosus, Systemic
Interventions
Registration Number
NCT03517722
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

The purpose of this study is to evaluate the efficacy of ustekinumab in participants with active systemic lupus erythematosus (SLE) who have not adequately responded to one or more standard of care treatments.

Detailed Description

This study evaluates the efficacy, safety, and tolerability of ustekinumab in participants with active SLE according to Systemic Lupus International Collaborating Clinics (SLICC) criteria Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score greater than (\>=) 6, despite receiving one or more standard-of-care treatments (example, immunomodulators, antimalarial drugs, and/or glucocorticoids). The total duration of the study is up to 182 weeks, consisting of 3 study periods: a screening period (approximately 6 weeks), a double blind period (52 weeks), and an extension period (124 weeks). Other study evaluations will include pharmacokinetics, immunogenicity, biomarkers and pharmacogenomic evaluations. The safety of the participants enrolled in the study will be monitored on an ongoing basis throughout the study.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
516
Inclusion Criteria
  • Be male or female

  • Has a documented medical history (that is, met at least 1 of the two criteria below) that participant met the Systemic Lupus International Collaborating Clinics (SLICC) classification criteria for systemic lupus erythematosus (SLE) at least 3 months prior to first dose of study agent:

    1. Met a total of at least 4 SLICC criteria, including at least 1 clinical and at least 1 immunologic;
    2. Has a diagnosis of lupus nephritis, confirmed by renal biopsy and at least 1 of the following autoantibodies: antinuclear antibodies (ANA) or anti-double-stranded deoxyribonucleic acid (anti-dsDNA)
  • Has a positive test in the medical history and confirmed at screening for at least 1 of the following autoantibodies: antinuclear antibodies, anti-double-stranded deoxyribonucleic acid, and/or anti-Smith

  • Has greater than or equal to (>=) 1 British Isles Lupus Assessment Group (BILAG) A and/or >= 2 BILAG B scores observed during screening

  • Has a Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) activity score >=4 (excluding diffuse non-inflammatory alopecia) or >= 4 joints with pain and signs of inflammation at screening, Week 0, or both

  • Has a Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score >=6 at screening. Must also have SLEDAI-2K >= 4 for clinical features (excluding headache and laboratory abnormalities) at Week 0

  • Cannot be pregnant, nursing, intending to become pregnant, or unwilling to follow contraception or egg/sperm donation guidelines

  • Must be receiving stable doses of >=1 protocol-permitted standard of care SLE treatment: oral glucocorticoids, anti-malarials, immunomodulators (methotrexate, azathioprine, 6-mercaptopurine, mycophenolate mofetil, mycophenolic acid)

Exclusion Criteria
  • Has any unstable or progressive SLE manifestation (example: central nervous system lupus, systemic vasculitis, end-stage renal disease, severe or rapidly progressive glomerulonephritis, pulmonary hemorrhage, myocarditis) that may warrant escalation in therapy beyond permitted background medications. Participants requiring renal hemodialysis or peritoneal dialysis are also excluded
  • Has other co-existent inflammatory diseases (example: rheumatoid arthritis, psoriasis, psoriatic arthritis, Crohn's disease)
  • Has a urinary protein to creatinine ratio of greater than (>)4 gram per gram (g/g) per day
  • Has an acute or chronic infectious illness (example: human immunodeficiency virus, hepatitis B or C virus, tuberculosis, opportunistic infections)
  • Has a history of cancer or lymphoproliferative disease within the last 5 years except for treated and non-recurrent cutaneous basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma
  • Has any condition requiring multiple courses of systemic glucocorticoids (example: uncontrolled asthma, chronic obstructive pulmonary disease)
  • Has a history of major surgery within the last month
  • Has received live virus or bacterial vaccines within 16 weeks prior to first dose of study agent or Bacille Calmette-Guerin (BCG) vaccination within 12 months of screening
  • Has previously received ustekinumab
  • Has received cyclophosphamide orally within 90 days or intravenously within 180 days of screening
  • Has received a single B-cell targeted therapy (e.g. belimumab) within 3 months, >1 previous B-cell targeted therapy within 6 months, or B-cell depleting therapy (example: rituximab) within 12 months of first dose of study agent
  • Has received protocol-prohibited oral or biologic immunomodulatory therapy in the last 3 months or less than (<)5 half-lives (whichever is longer) prior to first dose of study agent
  • Has received adrenocorticotropic hormone (ACTH) within 1 month prior to first dose of study agent
  • Has received epidural, intravenous, intramuscular, intraarticular, intrabursal, intralesional glucocorticoids within 6 weeks of first dose of study agent
  • Locally-delivered therapies except for ophthalmic use of cyclosporine A or topical use of nonsteroidal anti inflammatory drugs (NSAIDs), analgesics, or high-potency glucocorticoids (World Health Organization criteria) are prohibited

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
UstekinumabUstekinumab (approximately 6 mg/kg)Participants will receive ustekinumab approximately 6 milligram per kilogram (mg/kg) intravenously (IV) based on body weight-range at Week 0 followed by 90 mg ustekinumab subcutaneously (SC) at Week 8 and every 8 weeks (q8w) thereafter through Week 48 during double-blind period. Eligible participants who will enter the extension period will continue to receive 90 mg ustekinumab SC q8w through Week 160.
UstekinumabUstekinumab 90 mgParticipants will receive ustekinumab approximately 6 milligram per kilogram (mg/kg) intravenously (IV) based on body weight-range at Week 0 followed by 90 mg ustekinumab subcutaneously (SC) at Week 8 and every 8 weeks (q8w) thereafter through Week 48 during double-blind period. Eligible participants who will enter the extension period will continue to receive 90 mg ustekinumab SC q8w through Week 160.
PlaceboPlaceboParticipants will receive matching placebo to ustekinumab IV at Week 0, followed by matching placebo to ustekinumab SC at Week 8 and q8w thereafter through Week 48 during double-blind period. Eligible participants who will enter the extension period will cross-over to receive 90 mg ustekinumab SC q8w through Week 160.
PlaceboUstekinumab 90 mgParticipants will receive matching placebo to ustekinumab IV at Week 0, followed by matching placebo to ustekinumab SC at Week 8 and q8w thereafter through Week 48 during double-blind period. Eligible participants who will enter the extension period will cross-over to receive 90 mg ustekinumab SC q8w through Week 160.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants Achieving a Systemic Lupus Erythematosus Responder Index-4 (SRI-4) Composite Response at Week 52Week 52

SRI-4 response:\>=4-point reduction in SLEDAI-2K total score, no British Isles Lupus Assessment Group (BILAG) A (severe disease) and no more than 1 new BILAG B (moderate disease) domain score and no worsening (\<10 % increase)from baseline in Physician's Global Assessment(PGA).SLEDAI measures disease activity in 9 organ systems,higher scores=more severe disease activity.Each organ system measured as either absent/present within last 30 days and weighted score across systems was utilized to calculate total SLEDAI score(range:0=no symptoms to 105=presence of all defined symptoms). Improvement is defined as reduction in SLEDAI score (BILAG) Index: assessing clinical signs, symptoms,or laboratory parameters related to SLE,divided into 9 domains. Each domain can range from A=new domain activity, B=worse domain activity, C=same domain activity, D=improving domain activity to E=absence of domain activity. PGA assesses disease activity on visual analogue scale from very well(0)-very poor(10).

Secondary Outcome Measures
NameTimeMethod
Time to First FlareUp to Week 52

Time to flare is defined as the time (in days) post baseline when the first flare occurs. It was calculated with flare defined as either 1 or more BILAG A (severe disease activity) or 2 or more new BILAG B (moderate disease activity) domain scores relative to baseline. BILAG was defined as a measure of alterations or intensification to therapy consisting of 97 questions in 9 domains. Each domain can range from A=new domain activity, B=worse domain activity, C=same domain activity, D=improving domain activity to E=absence of domain activity. BILAG A flare was defined as at least 1 new BILAG A scores. BILAG B flare was defined as at least 2 new BILAG B scores.

Percentage of Participants Receiving Glucocorticoid at Baseline Who Achieved Change in Glucocorticoid Dose by Week 40, Sustained That Change Through Week 52, and Achieved an SRI-4 Composite Response at Week 52Up to Week 52

Percentage of participants with reduction in glucocorticoid dose by Week 40, its sustenance through Week 52, and SRI 4 composite response at Week 52 were reported. Reduction of glucocorticoid dose was defined as reduction in average daily oral glucocorticoid dose by at least 50% (relative to baseline dose) or reduction of average daily oral glucocorticoid dose by at least 25% (relative to baseline dose) so that average daily dose is reduced to \<=7.5 mg (prednisone or equivalent). Sustained reduction of glucocorticoid dose was defined as achieving an average daily oral glucocorticoid dose reduction between Weeks 24 and 40, and sustaining that reduction through Week 52, in those participants who,at baseline,were receiving oral glucocorticoids. SRI-4 was defined as composite of at least 4-point improvement in SLEDAI-2K score of 0=no symptoms to 105=presence of all defined symptoms with higher scores representing increased disease activity),no worsening in BILAG and no worsening in PGA.

Percentage of Participants With an SRI-4 Composite Response at Week 24Week 24

SRI-4 response:\>=4-point reduction in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) total score, no BILAG A (severe disease) and no more than 1 new BILAG B (moderate disease) domain score and no worsening (\<10 % increase)from baseline in PGA.SLEDAI measures disease activity in 9 organ systems, higher scores=more severe disease activity. Each organ system measured as either absent/present within last 30 days and weighted score across systems was utilized to calculate total SLEDAI score(range:0=no symptoms to 105=presence of all defined symptoms). Improvement is defined as reduction in SLEDAI score (BILAG) Index: assessing clinical signs, symptoms,or laboratory parameters related to SLE,divided into 9 domains. Each domain can range from A=new domain activity, B=worse domain activity, C=same domain activity, D=improving domain activity to E=absence of domain activity. PGA assesses disease activity on visual analogue scale from very well(0)-very poor(10).

Percentage of Participants With 50 Percent (%) Improvement in Joints With Pain and Signs of Inflammation (Active Joints) at Week 52Week 52

The percentage of participants who achieved at least 50% improvement from baseline in number of joints with pain and signs of inflammation at Week 52 for participants with at least 4 joints with pain and signs of inflammation at baseline were reported.

Percentage of Participants Receiving Glucocorticoid at Baseline Who Achieved Change in Glucocorticoid Dose by Week 40 and Sustain That Change Through Week 52Up to Week 52

Reduction of glucocorticoid dose was defined as a reduction in average daily oral glucocorticoid dose by at least 50% (relative to the baseline dose) or reduction of average daily oral glucocorticoid dose by at least 25% (relative to the baseline dose) so that the average daily dose was reduced to less than or equal to (\<=) 7.5 milligram (mg) (prednisone or equivalent). Sustained reduction of glucocorticoid dose was defined as achieving an average daily oral glucocorticoid dose reduction between Weeks 24 and 40, and sustaining that reduction through Week 52, in those participants who, at baseline, were receiving oral glucocorticoids.

Percentage of Participants With at Least a 50% Improvement in the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score at Week 52Week 52

Percentage of participants achieving at least 50% improvement in CLASI activity score at Week 52 reported in participants with a CLASI activity score of 4 or greater at baseline. The CLASI is an instrument to assess the disease activity and damage caused to the skin for cutaneous lupus erythematosus participants with or without systemic involvement. The CLASI activity score ranges from 0-70 with lower score being improved. Activity is scored based on erythema, scale/hyperkeratosis, mucous membrane involvement, acute hair loss, and non-scarring alopecia.

Trial Locations

Locations (204)

Pinnacle Research Group, LLC

🇺🇸

Anniston, Alabama, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Achieve Clinical Research, LLC

🇺🇸

Vestavia Hills, Alabama, United States

Medvin Clinical Research

🇺🇸

Covina, California, United States

Lugene Eye Institute

🇺🇸

Glendale, California, United States

C.V. Mehta, MD Medical Corp.

🇺🇸

Hemet, California, United States

University of California at San Diego

🇺🇸

La Jolla, California, United States

Advanced Medical Research - Lakewood

🇺🇸

Lakewood, California, United States

Loma Linda University

🇺🇸

Loma Linda, California, United States

Loma Linda University Health Care

🇺🇸

Loma Linda, California, United States

Scroll for more (194 remaining)
Pinnacle Research Group, LLC
🇺🇸Anniston, Alabama, United States

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