A Phase 2, Randomized, Placebo-controlled, Parallel-group, Double-blinded, proof-of Concept Study to Evaluate the Safety and Efficacy of Intravenous Efgartigimod in Adult Participants With Primary Sjögren's Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 34
- 试验地点
- 17
- 主要终点
- Percentage of Participants Meeting Overall CRESS Response of at Least 3 of 5 Items at Week 24
研究概览
简要总结
The purpose of this study is to assess the efficacy and safety of human FcRn blocking therapy with efgartigimod compared to placebo, in participants with pSS.
详细描述
Primary Sjogren Syndrome (pSS) is an autoimmune disease with still unmet treatment needs. Efgartigimod, a human FcRn antagonist, has the potential to successfully treat pSS and improve disease manifestations by the reduction of IgG autoantibodies and immune complexes in pSS. The study design is randomized, double-blinded, and placebo-controlled to evaluate the effect of efgartigimod administered as an IV infusion compared to placebo. The study consists of a treatment period when all participants will receive infusions of IP/placebo for 24 weeks. At the end of the randomized treatment period, eligible participants may roll over to an OLE study or remain in this study through the end of the 56-day follow-up period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Participants will be randomized to receive efgartigimod 10 mg/kg or placebo in a 2:1 ratio, respectively. All participants will receive efgartigimod IV 10 mg/kg or placebo once weekly for 24 weeks during the treatment period
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is at least the legal age of consent for clinical trials when signing the informed consent form
- •Is capable of providing signed informed consent and complying with protocol requirements
- •Agrees to use contraceptive measures consistent with local regulations and measures described in the protocol
- •Meets the following criteria at screening: ACR/EULAR 2016 pSS who met criteria ≤7 years before screening; ESSDAI ≥5; Anti-Ro/SS-A positive; Residual salivary flow (UWSF rate >0 and/or SWSF rate >0.10)
排除标准
- •Known autoimmune disease or any medical condition that, in the investigator's judgment,would interfere with an accurate assessment of clinical symptoms of pSS or puts the participant at undue risk
- •History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP.
- •Adequately treated participants with the following cancers may be included at any time: Basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological finding of prostate cancer (TNM stage T1a or T1b) Clinically significant uncontrolled active acute or chronic bacterial, viral, or fungal infection
- •Positive serum test at screening for an active infection with any of the following: HBV that is indicative of an acute or chronic infection, unless associated with a negative HBsAg or negative HBV DNA test; HCV based on HCV antibody assay unless a negative RNA test is available; HIV based on test results of a CD4 count of <200 cells/mm3 that are associated with an AIDS-defining condition, HIV based on test results of a CD4 count of >200 cells/mm3 not adequately treated with antiviral therapy
- •Clinically significant disease, recent major surgery (within 3 months of screening), or intention to have surgery during the study; or any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk
- •Immunoglobulin G (IgG) levels cannot be below a certain threshold ( 4g/L)
- •Positive covid test at study start
- •Some of the medications such as vaccines with live components or medicines that may be prescribed cannot be taken either shortly before or during this study
- •Current participation in another interventional clinical study or previously participation in an efgartigimod clinical study and treatment with ≥1 dose of IMP
- •Known hypersensitivity to IMP or 1 of its excipients
- •History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator
- •Pregnant or lactating state or intention to become pregnant during the study
- •Secondary Sjögren's syndrome overlap syndromes where another confirmed autoimmune rheumatic or systemic inflammatory condition is the primary diagnosis
- •Chinese traditional medicine with known immunomodulatory action
研究组 & 干预措施
Efgartigimod IV arm
patients receiving infusions of Efgartigimod IV
干预措施: Efgartigimod (Biological)
Placebo arm
patients receiving infusions of placebo IV
干预措施: Placebo (Biological)
结局指标
主要结局
Percentage of Participants Meeting Overall CRESS Response of at Least 3 of 5 Items at Week 24
时间窗: Week 24
A Composite of Relevant Endpoints for Sjögren's Syndrome (CRESS) responder is defined as improvements in at least 3 of the 5 items of CRESS (systemic disease activity, patient-reported symptoms, tear gland function, salivary gland function and serology. The score ranges from 0 to 9 (higher score = worse symptoms).
次要结局
- Number of Participants With TEAEs, AESI, and SAEs(Up to 32 weeks)
- Percentage of Participants With MCII in ESSDAI at Week 24(Week 24)
- Percentage of Participants With Low Disease Activity in ESSDAI at Week 24(Week 24)
- Percentage of Participants With MCII in clinESSDAI at Week 24(Week 24)
- Percentage of Participants With Low Disease Activity in clinESSDAI at Week 24(Week 24)
- Percentage of Participants With MCII in ESSPRI at Week 24(Week 24)
- Change From Baseline in ESSDAI Score at Week 24(Baseline (Day 1) and Week 24)
- Change From Baseline in clinESSDAI Score at Week 24(Baseline (Day 1) and Week 24)
- Change From Baseline in ESSPRI Score at Week 24(Baseline (Day 1) and Week 24)
- Percentage of Participants With STAR Score of at Least 5 at Week 24(Week 24)
- Plasma Concentration of Efgartigimod(Pre-dose at Day 1, Weeks 1, 2, 4, 8, 12, 16, and 20; 30 minutes post-dose at Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24)
- Number of Participants With ADA Against Efgartigimod in Serum(Up to Week 24)
- Percentage Change From Baseline in Total IgG Levels in Serum at Week 24(Baseline (Day 1) and Week 24)