NCT07806916尚未招募2 期
Efficacy and Safety of Efgartigimod in the Treatment of Guillain-Barré Syndrome: A Prospective, Multicenter, Randomized Controlled Clinical Trial
Beijing Tiantan Hospital0 个研究点目标入组 110 人开始时间: 2026年9月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 110
- 主要终点
- Guillain-Barre Syndrome Disability Scale (GBS-DS)
研究概览
简要总结
The goal of this clinical trial is to evaluate the safety and effectiveness of Efgartigimod in patients with Guillain-Barre syndrome (GBS). The main questions it aims to answer are:
Is Efgartigimod a safe treatment option for GBS patients? Does treatment with Efgartigimod improve patient outcomes?
Participants will:
Take drug Efgartigimod or Intravenous Immunoglobulin during hospitalization Undergo six blood draws in six follow-up study visits to evaluate the concentration of IgG Keep a diary of their symptoms and drugs using at the same time
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18-70 years
- •Diagnosis of sensorimotor or pure motor Guillain-Barré syndrome (GBS) based on the 2023 EAN/PNS guidelines (corresponding to classical GBS in the Chinese Guidelines for the Diagnosis and Treatment of Guillain-Barré Syndrome 2024).
- •Onset of GBS within 14 days
- •GBS Disability Scale (GBS-DS) score of 3 to 5
排除标准
- •Patients with other diagnoses cannot be excluded
- •Patients with recurrent GBS
- •Patients with more than 14 days after onset of symptoms
- •GBS DS of 2 or less
- •Patients unable to cooperate with scale assessments due to psychiatric disorders or impaired consciousness
- •Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening
- •Patients with any known severe bacterial, viral or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy in the last 8 weeks prior to Screening
- •Total IgG level ≤ 6 g/L at screening
- •Concomitant diseases that can cause peripheral neuropathy, such as metabolic neuropathies (e.g., diabetes, alcoholism), connective tissue disease-associated neuropathies (e.g., rheumatoid arthritis, Sjögren's syndrome, systemic lupus erythematosus), or trauma, which may confound the trial results
- •Subjects with serious underlying diseases, such as cardiac dysfunction, coagulopathy dysfunction
- •Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing
- •Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 3 years before Screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time
- •Use of investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening
- •Use of immunosuppressants, monoclonal antibodies, or other immunomodulatory drugs within 6 months prior to enrollment; use of corticosteroids at a dose exceeding 20 mg within 3 months prior to enrollment; or use of IVIG or plasma exchange (PE) within 1 month prior to enrollmen
研究组 & 干预措施
Intravenous Immunoglobulin (IVIg)
Active Comparator
0.4g/kg of IVIg daily for 5 days
Interventions:
Drug: Intravenous Immunoglobulin (IVIg)
干预措施: Intravenous immunoglobulin (IVIG) (Drug)
Efgartigimod Alfa-Fcab
Experimental
20mg/kg of Intravenous efgartigimod on days 1 and 5
Interventions:
Drug: Efgartigimod Alfa-Fcab
干预措施: Efgartigimod Alfa-Fcab (Drug)
结局指标
主要结局
Guillain-Barre Syndrome Disability Scale (GBS-DS)
时间窗: week 4
Scoring system that assesses the functional status of GBS subjects. Scores range from 0 to 6, with higher scores indicating a worse outcome.
Number and seriousness of adverse events
时间窗: Through study completion, an average of 2 years
safety monitoring
次要结局
- Number of days to GBS-DS ≤2 within 24 weeks(24 weeks)
- Guillain-Barre Syndrome Disability Scale (GBS-DS)(week 1，2，3，8，12，24)
- Proportion of patients with GBS-DS ≤2(week 1, 2, 3, 4, 8, 12, and 24)
- Neuropathy Impairment Scale (NIS)(week 1,4,8,12,24)
- Inflammatory Rasch-Built Overall Disability Scale (I-RODS)(week 1,4,8,12,24)
- MRC Sum Score(week 1,4,8,12,24)
- Incidence of assisted mechanical ventilation(24 weeks)
- Duration of mechanical ventilation (hours)(24 weeks)
- Number of days in an intensive care unit(24 weeks)
- Number of days to hospital discharge(24 weeks)
- Total hospitalization cost (in RMB)(24 weeks)
- Forced Vital Capacity (FVC)(week 4,12,24)
- Maximal Inspiratory Pressure (MIP)(week 4,12,24)
- Maximal Expiratory Pressure (MEP)(week 4,12,24)
- Change from baseline in Guillain-Barre Syndrome Disability Scale (GBS-DS)(week 1,2,3,4,8,12,24)
- Inflammatory Neuropathy Cause and Treatment (INCAT) Scale(week 1,4,8,12,24)
- Modified Rankin Scale (mRS)(week 1,4,8,12,24)
- Medical Research Council Sum Score（MRC-SS）(week 1,4,8,12,24)
- Hospitalization cost (inpatient cost)(24 weeks)
研究者
Zaiqiang Zhang
Chief Physician,Professor
Beijing Tiantan Hospital
相似试验
招募中
1 期
A study to assess the effects of efgartigimod on emasiprubart in healthy adult participants2024-517511-70-00Argenx81
招募中
1 期
A Study to Assess the Bioequivalence of Efgartigimod PH20 SC Between Autoinjector and Prefilled Syringe in Healthy Adult ParticipantsHealthy VolunteerHealthy Adult ParticipantsNCT07782775argenx32
已完成
3 期
A phase 2/3 study of efgartigimod PH20 SC in adult participants with bullous pemphigoid2023-508645-40-00Argenx90
招募中
不适用
Real-World Efgartigimod Effectiveness in CIDP: A Prospective StudyCIDPNCT07264426argenx200
尚未招募
1 期
Role of Marichadi Gutika and Mandookparni vati in the management of Irritable Bowel Syndrome in ChildrenCTRI/2026/01/100562State Ayurvedic college and hospital Lucknow60