A Multicentre Study of Subcutaneous Immunoglobulin (SCIG) in Patients With Multifocal Motor Neuropathy (MMN)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 8
- 试验地点
- 3
- 主要终点
- Mean Overall MRC Score at Baseline and Week 24
研究概览
简要总结
The objective of this study is to assess efficacy, safety, and convenience of purified human antibodies administered under the skin in the treatment of MMN patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Patients with documented clinical diagnosis and electrophysiological evidence of MMN
- •Patients who have previously responded to intravenous immunoglobulin (IVIG) and have been on stable treatment with IVIG for at least 12 weeks prior to screening
- •Patients treated with the equivalent of ≥0.4g/kg body weight (bw) IVIG per month
- •Provision of informed consent by patient
排除标准
- •Aspartate aminotransferase (ASAT) or alanine aminotransferase (ALAT) concentration >2.5 times the upper normal limit (UNL)
- •Creatinine concentration >1.5 times the UNL
- •Known allergic reactions to blood products
- •Any skin disease interfering with the assessment of injection site reactions
- •Any other medical condition, which in the opinion of the investigator, might interfere with successful completion of the protocol
- •Any condition likely to interfere with the evaluation of the study drug or satisfactory conduct of the trial
- •Participation in a study with an investigational drug within three months prior to enrolment
- •Patients treated with the equivalent of >2.0g/kg bw IVIG per month
结局指标
主要结局
Mean Overall MRC Score at Baseline and Week 24
时间窗: Baseline and week 24
The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement.
Change From Baseline to Week 24 in Muscle Strength
时间窗: Baseline to week 24
The change in Medical Research Council (MRC) score was determined at week 24 compared to baseline using descriptive statistics and nonparametric, two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available. The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement. Positive values for change in MRC sum score indicate improvement, with a more positive value indicating greater muscle contraction/ limb movement compared with the value at baseline.
次要结局
- Treatment Satisfaction at Baseline and Week 25(At baseline and week 25)
- Change From Baseline to the Completion Visit in Motor Function(Baseline to the completion visit (up to week 25))
- Change From Baseline to Week 24 in Disability(Baseline to week 24)
- Mean Disability Score at Baseline and Week 24(Baseline and Week 24)
- Health-Related Quality of Life at Baseline and Week 25(At baseline and week 25)
- Mean Motor Function Score at Screening and Week 25(Screening and week 25)
- Overall Health Status at Baseline and Week 25(Baseline and week 25)
- Number of Patients With Adverse Events (AEs) by Severity and Relatedness(For the duration of the study, up to Week 25)
- Rate of AEs by Severity and Relatedness(For the duration of the study, up to Week 25)
- Number of Patients With Local/Injection Site Reactions(For the duration of the study, up to Week 25)
- Number of Patients With Clinically Relevant Changes in Vital Signs(Baseline to Week 25)
- Number of Patients With Clinically Relevant Changes in Laboratory Parameters(Baseline to Week 25)