RitUximab in Guillain-Barré sYndrome (RUGBY) trial: a single center, double-blind, randomized phase 3 trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 33
- 试验地点
- 1
- 主要终点
- Proportion of patients who are able to ambulate independently (Hughes score≤ 2) at 4- and 24-weeks post symptom onset.
研究概览
简要总结
Guillain Barre syndrome (GBS) is an immune mediated polyradiculoneuropathy that causes acute flaccid paralysis. Intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX) are equally effective in treatment and form the standard of care. However, approximately 25% patients have residual disability despite treatment, and require support for ambulation/ ventilation. Recent studies have shown that B cell and complement activation play a central role in GBS-associated neuronal degeneration. Eculizumab is a monoclonal antibody which inhibits C5 complement, and has proven beneficial in two randomized controlled trials on GBS (ICA-GBS and JET-GBS). Rituximab is a chimeric monoclonal antibody which binds to CD20 antigens on B cells inducing their cytolysis, thereby decreasing downstream B-cell and complement mediated axonal damage. Since its mechanism of action includes that of Eculizumab, it is likely to be as effective and the cost of Rituximab in India is 1/50th that of Eculizumab.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Participant, Investigator and Outcome Assessor Blinded
入排标准
- 年龄范围
- 18.00 Year(s) 至 90.00 Year(s)（—）
- 性别
- All
入选标准
- •1.Fulfilling the Brighton Collaboration Diagnostic Criteria for GBS [11]: a.
- •Bilateral and flaccid paralysis of limbs AND b.
- •Decreased or absent tendon reflexes in weak limbs AND c.
- •Monophasic illness pattern and interval between onset and nadir of weakness between 12 hours and 28 days with subsequent clinical plateau AND d.
- •Absence of an identified alternative diagnosis for weakness 2.Presentation within 2 weeks on symptom onset 3.Patient unable to walk unassisted for ≥ 5 metres (Hughes score 3-5) 4.Undergoing treatment with IVIg 5.First dose of Rituximab administered within 2 weeks of symptom onset 6.Signed informed consent for participation in the study.
排除标准
- •1.Pregnant/ lactating women 2.GBS patients treated with plasma exchange 3.Patients who have received other immunosuppressants (Azathioprine, Mycophenolate, Methotrexate) within 4 weeks or Rituximab within 24 weeks prior to informed consent 4.Severe comorbid diseases like chronic liver disease, chronic kidney disease, malignancy, tuberculosis or chronic obstructive pulmonary disease 5.Presence of an active, inadequately treated, infection 6.Known immunocompromised state (hereditary or acquired) 7.Participation in any other clinical trial.
结局指标
主要结局
Proportion of patients who are able to ambulate independently (Hughes score≤ 2) at 4- and 24-weeks post symptom onset.
时间窗: 4 weeks and 24 weeks
次要结局
- Proportion of patients with improvement of Hughes score by ≥1 at each visit, duration required for improvement in Hughes score by 1, occurrence of relapse, overall survival and improvement in nerve conduction studies at 24 weeks.(Each follow up visit)
研究者
Ayush Agarwal
AIIMS, New Delhi