A Phase 2, Randomized, Double-Blinded, Placebo-Controlled Study to Assess the Efficacy and Safety of Empasiprubart as Add-On Therapy to IVIg in Adult Participants With Guillain-Barré Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- argenx
- 入组人数
- 63
- 主要终点
- Proportion of Participants With a Guillain-Barré Syndrome Disability Scale (GBS-DS) Score ≤2 at Week 4
研究概览
简要总结
The main purpose of this study is to learn whether empasiprubart, when given in addition to intravenous immunoglobulin (IVIg), helps adults with Guillain-Barré syndrome (GBS) recover and whether it is safe. About 63 participants will be assigned by chance to receive either empasiprubart or placebo by intravenous infusion; all participants will also receive IVIg. The study includes screening for up to 72 hours, study treatment through Day 12, and follow-up through Week 67 (15 months after the last dose of study treatment). Participation lasts up to 67 weeks.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •At least 18 years old
- •Clinically diagnosed with Guillain-Barré syndrome (GBS) according to NINDS (National Institute of Neurological Disorders and Stroke) diagnostic criteria
- •GBS-related weakness beginning within 7 days before the first study treatment
- •GBS Disability Scale (GBS-DS) score of 3, 4, or 5 at screening and baseline
- •Was able to walk approximately 10 m or more without assistance before the onset of GBS symptoms
- •Agrees to receive antibiotic prophylaxis against N. meningitidis and S. pneumoniae for the first 90 days of the study or until a completed vaccination schedule against these bacterial pathogens per local guidelines has been shown.
排除标准
- •History of a previous GBS episode or diagnosis of an atypical GBS variant
- •Clinical diagnosis of systemic lupus erythematosus (SLE) or known complement deficiency
- •Autoimmune or neuromuscular diseases or medical conditions (eg, clinically significant renal, hepatic, cardiac, pulmonary, hematologic, or neurological conditions or clinically significant laboratory abnormalities) that would interfere with an accurate assessment of clinical symptoms of GBS, confound the study results, or put the participant at undue risk
- •IVIg therapy contraindications or another medical condition that, in the opinion of the investigator, would make IVIg administration unsafe
- •Recent treatment with IVIg or plasma exchange (PLEX), or recent use of certain immune-modulating or immunosuppressive therapies
研究组 & 干预措施
Placebo + IVIg
Participants receive placebo by intravenous and IVIg by intravenous infusion.
干预措施: Intravenous immunoglobulin (IVIg) (Biological)
Empasiprubart + IVIg
Participants receive empasiprubart by intravenous infusion and IVIg by intravenous infusion.
干预措施: Intravenous immunoglobulin (IVIg) (Biological)
Empasiprubart + IVIg
Participants receive empasiprubart by intravenous infusion and IVIg by intravenous infusion.
干预措施: Empasiprubart IV (Biological)
Placebo + IVIg
Participants receive placebo by intravenous and IVIg by intravenous infusion.
干预措施: Empasiprubart Placebo IV (Other)
结局指标
主要结局
Proportion of Participants With a Guillain-Barré Syndrome Disability Scale (GBS-DS) Score ≤2 at Week 4
时间窗: at week 4
The Guillain-Barré Syndrome Disability Scale (GBS-DS) is a clinician-reported measure of global functional disability. It has 7 categories ranging from 0 (healthy) to 6 (death), with higher scores indicating greater disability. A GBS-DS score ≤2 indicates the participant is able to walk 10 meters or more without assistance.
次要结局
- Number of calendar days in the intensive care unit (ICU) within the first 4 weeks of investigational medicinal product (IMP) administration(Up to 4 weeks)
- Number of calendar days receiving invasive mechanical ventilation within the first 4 weeks of investigational medicinal product (IMP) administration(Up to 4 weeks)
- Time From First Investigational Medicinal Product (IMP) Administration to Achieving a Guillain-Barré Syndrome Disability Scale (GBS-DS) Score ≤2(Up to 52 weeks)
- Actual values in the Inflammatory Rasch-Built Overall Disability Scale (I-RODS) Scores at weeks 13 and 26(Up to 26 weeks)
- Actual values in the Overall Neuropathy Limitations Scale (ONLS) Score at weeks 13 and 26(Up to 26 weeks)
- Change from baseline in the Medical Research Council Sum Score (MRC-SS) over time(Up to 52 weeks)
- Change from baseline in the Medical Research Council Sum Score (MRC-SS) neck flexion assessment over time(Up to 52 weeks)
- Change from baseline in the Guillain-Barré Syndrome Disability Scale (GBS-DS) score over time(Up to 52 weeks)
- Proportion of participants in the Intensive Care Unit (ICU) over time and at any time through week 52(Up to 52 weeks)
- Actual Inflammatory Rasch-Built Overall Disability Scale (I-RODS) Scores Over Time(Up to 52 weeks)
- Actual Overall Neuropathy Limitations Scale (ONLS) Score Over Time(Up to 52 weeks)
- Number of calendar days receiving invasive mechanical ventilation through week 52(Up to 52 weeks)
- Proportion of participants who require invasive mechanical ventilation over time and at any time through the week 52(Up to 52 weeks)
- Number of calendar days in the Intensive Care Unit (ICU) through week 52(Up to 52 weeks)
- Number of calendar days from the start of investigational medicinal product (IMP) until discharge from the acute care facility/department(Up to 52 weeks)
- Number of calendar days from the start of investigational medicinal product (IMP) until discharge from the last rehabilitation unit(Up to 52 weeks)
- All-cause mortality through week 52(Up to 52 weeks)
- Incidence of Adverse events (AEs) and Adverse Events of Special Interest (AESIs)(Up to 67 weeks)
- Incidence of Serious Adverse Events (SAEs)(Up to 67 weeks)
- Serum concentrations of empasiprubart over time(Up to 52 weeks)
- Percentage Change From Baseline in Free Complement Component 2 (C2) Over Time(Up to 52 weeks)
- Percentage Change From Baseline in Total Complement Component 2 (C2) Over Time(Up to 52 weeks)