LITIFILIMAB for Systemic lupus erythematosus

Inclusion criteria

• {"criterion_text":"- Participant must be diagnosed with systemic lupus erythematosus (SLE) at least 24 weeks prior to screening and must meet the 2019 European League Against Rheumatism (EULAR) / American College of Rheumatology (ACR) classification criteria for SLE at screening by a qualified physician.\n- Participant has a modified Systemic Lupus Erythematosus Disease Activity Index-2000 (SLEDAI-2K) score ≥ 6 (excluding alopecia, fever, lupus-related headache, and organic brain syndrome) at screening (adjudicated).\n- Participant has a modified clinical SLEDAI-2K score ≥ 4 (excluding anti-dsDNA, low complement component 3 (C3) and/or complement component 4 (C4), alopecia, fever, lupus-related headache, and organic brain syndrome) at Screening (adjudicated) and randomization.\n- Participant has BILAG-2004 grade A in ≥ 1 organ system or BILAG-2004 grade B in ≥ 2 organ systems at Screening (adjudicated) and randomization.\n- Participant must be treated with one of the following background nonbiologic lupus SOC therapies, initiated ≥ 12 weeks prior to Screening and at stable dose ≥ 4 weeks prior to randomization, a. Antimalarials as stand-alone treatment b. Antimalarial treatment in combination with OCS and/or a single immunosuppressant c. Treatment with OCS and/or a single immunosuppressant"}

Exclusion criteria

• {"criterion_text":"- History of or positive test result for human immunodeficiency virus (HIV).\n- Current hepatitis C infection (defined as positive hepatitis C virus (HCV) antibody and detectable HCV ribonucleic acid [RNA]).\n- Current hepatitis B infection (defined as positive for HBsAg and/or positive for total anti- HBc) with positive reflex HBV DNA).\n- Presence of uncontrolled or New York Heart Association class III or IV congestive heart failure. - Active severe lupus nephritis where, in the opinion of the Investigator, protocol-specified SOC is insufficient and use of a more aggressive therapeutic approach is indicated, such as adding IV cyclophosphamide and/or high-dose IV pulse corticosteroid therapy or other treatments not permitted in the protocol is indicated; or urine protein-creatinine ratio > 2.0 or severe chronic kidney disease (estimated glomerular filtration rate < 30 milliliters per minute per 1.73 meter square [mL/min/1.73 m^2]) calculated using the abbreviated modification of diet in renal disease equation.\n- Any active skin conditions other than cutaneous lupus erythematosus (CLE) that may interfere with the study assessment of CLE such as but not limited to psoriasis, dermatomyositis, systemic sclerosis, non-LE skin lupus manifestation or drug-induced lupus.\n- History or current diagnosis of a clinically significant non-SLE-related vasculitis syndrome.\n- Active neuropsychiatric SLE\n- Use of oral prednisone (or equivalent) above 20 mg/day.\n- Other protocol defined Exclusion criteria may apply."}

Primary endpoints

• {"endpoint_text":"- Percentage of Participants Who Achieved a Systemic Lupus Erythematosus Responder Index of 4 (SRI-4) Response at Week 52.","definition_or_measurement_approach":"SRI-4 response assessed at Week 52 (percentage of participants achieving SRI-4 at Week 52)."}

Secondary endpoints

• {"endpoint_text":"- Percentage of Participants Who Achieved a BICLA Response at Week 52","definition_or_measurement_approach":"BICLA response assessed at Week 52."}
• {"endpoint_text":"- Percentage of Participants With at Least 4 Joints (Both Swollen and Tender) at Baseline Who Achieved a Joint-50 Response at Week 52","definition_or_measurement_approach":"Joint-50 response (50% improvement in joint counts) assessed at Week 52 in participants with ≥4 affected joints at baseline."}
• {"endpoint_text":"- Percentage of Participants with OCS ≥10 milligrams per day (mg/day) at Baseline with OCS Reduction to ≤7.5 mg/day at Week 40, Which Is Sustained Through Week 52 with No Disease Worsening from Week 40 to Week 52","definition_or_measurement_approach":"Oral corticosteroid (OCS) dose reduction to ≤7.5 mg/day by Week 40 sustained through Week 52 without disease worsening."}
• {"endpoint_text":"- Percentage of Participants with a CLASI-A score ≥10 at Baseline Who Achieved a 50% Improvement From Baseline in Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity Score (CLASI50) Response at Week 24","definition_or_measurement_approach":"CLASI50 response at Week 24 in participants with baseline CLASI-A ≥10."}
• {"endpoint_text":"- Annualized Flare Rate Through Week 52","definition_or_measurement_approach":"Annualized flare rate measured through Week 52."}
• {"endpoint_text":"- Change from Baseline in Physician's Global Assessment (PGA) Visual Analog Scale (VAS) Score by Visit","definition_or_measurement_approach":"Change from baseline in PGA-VAS measured at each visit."}
• {"endpoint_text":"- Percentage of Participants Who Achieved a British Isles Lupus Activity Group (BILAG)-Based Combined Lupus Assessment (BICLA) Response by Visit","definition_or_measurement_approach":"BICLA response assessed at each visit."}
• {"endpoint_text":"- Time to Onset of SRI-4 Response Sustained Through Week 52","definition_or_measurement_approach":"Time to first SRI-4 response that is sustained through Week 52."}
• {"endpoint_text":"- Percentage of Participants Who Achieved SRI-4, -5, or -6 Response by Visit","definition_or_measurement_approach":"Proportion achieving SRI-4/5/6 responses assessed by visit."}
• {"endpoint_text":"- Percentage of Participants with Joint-50 Response by Visit","definition_or_measurement_approach":"Joint-50 response assessed at each visit."}
• {"endpoint_text":"- Percentage of Participants with Baseline CLASI-A Score ≥10 Who Achieved a CLASI-20, -50, -70, or -90 Response by Visit","definition_or_measurement_approach":"CLASI percentage-improvement responses (20/50/70/90) assessed by visit in participants with baseline CLASI-A ≥10."}
• {"endpoint_text":"- Percentage of Participants with Baseline CLASI-A Score ≥10 Who Achieved a CLASI-A Score of ≤ 1 by Visit","definition_or_measurement_approach":"Proportion achieving CLASI-A ≤1 assessed by visit in participants with baseline CLASI-A ≥10."}
• {"endpoint_text":"- Time to First British Isles Lupus Activity Group-2004 (BILAG-2004) Severe Flare by Visit","definition_or_measurement_approach":"Time to first BILAG-2004-defined severe flare assessed by visit."}
• {"endpoint_text":"- Time to First Severe Flare as defined by the Safety of Estrogens in Systemic Lupus Erythematosus National Assessment - Systemic Lupus Erythematosus Disease Activity Index Flare Index (SFI)","definition_or_measurement_approach":"Time to first severe flare as defined by the SELENA-SFI."}
• {"endpoint_text":"- Percentage of time spent in LLDAS.","definition_or_measurement_approach":"Percentage of study time spent in Lupus Low Disease Activity State (LLDAS)."}
• {"endpoint_text":"- Proportion of participants with sustained LLDAS as defined by the number of participants with ≥ 3, ≥ 5, and ≥ 7 consecutive visits in LLDAS up to and including Week 52.","definition_or_measurement_approach":"Proportion achieving sustained LLDAS defined by ≥3, ≥5, and ≥7 consecutive LLDAS visits through Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved LLDAS at Week 52.","definition_or_measurement_approach":"Proportion in LLDAS at Week 52."}
• {"endpoint_text":"- Percentage of Participants With Baseline OCS ≥10 mg/day Who Achieved ≤7.5 mg/day at Week 52","definition_or_measurement_approach":"OCS dose reduction to ≤7.5 mg/day at Week 52 in those with baseline OCS ≥10 mg/day."}
• {"endpoint_text":"- Change from Baseline in Lupus-Specific Health-Related Quality-ofLife Questionnaire (LupusQoL) Score","definition_or_measurement_approach":"Change from baseline in LupusQoL score."}
• {"endpoint_text":"- Change From Baseline in Short Form Health Survey-36 (SF-36) Score","definition_or_measurement_approach":"Change from baseline in SF-36 score."}
• {"endpoint_text":"- Change From Baseline in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score","definition_or_measurement_approach":"Change from baseline in FACIT-Fatigue score."}
• {"endpoint_text":"- Change From Baseline in Patient Health Questionnaire-9 (PHQ-9) Score","definition_or_measurement_approach":"Change from baseline in PHQ-9 score."}
• {"endpoint_text":"- Change From Baseline in Work Productivity and Activity Impairment (WPAI): Lupus Score","definition_or_measurement_approach":"Change from baseline in WPAI:Lupus score."}
• {"endpoint_text":"- Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)","definition_or_measurement_approach":"Count of participants experiencing TEAEs and SAEs."}
• {"endpoint_text":"- Number of Participants with Antibodies to litifilimab","definition_or_measurement_approach":"Number of participants with anti-drug antibodies to litifilimab."}
• {"endpoint_text":"- Percentage of Participants with severe flares through Week 52","definition_or_measurement_approach":"Proportion of participants with severe flares through Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved SLEDAI-2K improvement at Week 52.","definition_or_measurement_approach":"Proportion achieving improvement in SLEDAI-2K at Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved no worsening of BILAG at Week 52.","definition_or_measurement_approach":"Proportion with no BILAG worsening at Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved no worsening of PGA-VAS at Week 52.","definition_or_measurement_approach":"Proportion with no PGA-VAS worsening at Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved no worsening of SLEDAI-2K at Week 52.","definition_or_measurement_approach":"Proportion with no SLEDAI-2K worsening at Week 52."}
• {"endpoint_text":"- Proportion of participants who achieved BILAG improvement at Week 52.","definition_or_measurement_approach":"Proportion with BILAG improvement at Week 52."}
• {"endpoint_text":"- Proportion of participants with OCS ≥ 10 mg/day at Baseline with OCS reduction to ≤ 5 mg/day at Week 40, which is sustained through Week 52 with no disease worsening5 from Week 40 to Week 52.","definition_or_measurement_approach":"OCS reduction to ≤5 mg/day at Week 40 maintained through Week 52 without worsening."}
• {"endpoint_text":"- Proportion of participants with OCS ≥ 7.5 mg/day at Baseline with OCS reduction to ≤ 7.5 mg/day at Week 52.","definition_or_measurement_approach":"OCS reduction to ≤7.5 mg/day at Week 52 in those with baseline OCS ≥7.5 mg/day."}

Methods

• Doctor-to-patient letters and HCP brochures (site-mediated recruitment) - materials present for multiple countries (e.g., 'Doctor-to-Patient Letter').
• Patient brochures, flyers and posters (printed materials distributed at sites) - country-specific patient brochure/flyer/poster materials included.
• Local social media posts and social media ad packets (digital advertising) - 'Local Social Media Post', 'Social Media Ad Packet', 'Local Social Media Templates' present.
• Online prescreener / Phone & Digital Self Screener / Landing page ads - 'Online Prescreener', 'Phone & Digital Self Screener', 'Landing Page Ad' documents present for recruitment.
• Website and digital content (study landing pages / website mockups) - 'TOPAZ Studies Website Share Mockups' and 'Website' materials.
• Patient photography / study materials and study visit schedules distributed digitally and in print.

France

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

03-04-2024

Processing Time Days

56

Number Of Sites

3

Number Of Participants

6

Sweden

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

02-04-2024

Processing Time Days

55

Number Of Sites

3

Number Of Participants

16

Greece

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

20-05-2024

Processing Time Days

40

Number Of Sites

2

Number Of Participants

24

Poland

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

04-04-2024

Processing Time Days

57

Number Of Sites

13

Number Of Participants

60

Spain

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

02-04-2024

Processing Time Days

55

Number Of Sites

7

Number Of Participants

16

Bulgaria

Earliest CTIS Part Ii Submission Date

07-02-2024

Latest Decision Or Authorization Date

08-04-2024

Processing Time Days

61

Number Of Sites

8

Number Of Participants

48

Primary sponsor

Full Name

Biogen Idec Research Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

United Kingdom

Contract research organisations

Name

Drugdev Inc.

Responsibilities

Site Start-up Management, Project & Vendor management, Medical Monitoring & 24hr Emergency, contact

Name

IQVIA RDS Hellas Single Member S.A.

Responsibilities

Clinical operations / central functions (sponsor duties codes: 1,12,2,5)

Name

IQVIA Limited

Responsibilities

Clinical operations / central functions (sponsor duties codes: 1,12,2,5)

Name

Pharmaceutical Product Development LLC

Responsibilities

Pharmacovigilance services

Name

Medidata Solutions Inc.

Responsibilities

Data management/technology (sponsor duties code: 7)

Name

Signant Health Global LLC

Responsibilities

IVRS – treatment randomization

Third parties

• {"country":"United States","full_name":"Drugdev Inc.","duties_or_roles":"Site Start-up Management, Project & Vendor management, Medical Monitoring & 24hr Emergency, contact","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Docs24 Limited","duties_or_roles":"Printing Vendor","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"Central Laboratory","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Greece","full_name":"IQVIA RDS Hellas Single Member S.A.","duties_or_roles":"Sponsor duties codes: 1, 12, 2, 5","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"Photography / Imaging","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Study equipment supply","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Cisys Inc.","duties_or_roles":"Adjudication of patient photos","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Pharmacovigilance services","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Sponsor duties codes: 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Travel and Reimbursement","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Crisalis LLC","duties_or_roles":"Lupus Specific Rater Training","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Sponsor duties codes: 1, 12, 2, 5","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"IVRS – treatment randomization","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"Photography / Imaging","organisation_type":"Pharmaceutical company"}