Randomized, Multicenter, Double-blind, Placebo-controlled, Parallel-group Phase III Study to Investigate the Efficacy, Safety, and Tolerability of 2 Different Doses of IgPro20 (Subcutaneous Immunoglobulin) for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) - the PATH Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 208
- 试验地点
- 91
- 主要终点
- Percentage (%) of Subjects With CIDP Relapse or Are Withdrawn for Any Other Reason During the Subcutaneous (SC) Treatment Period
研究概览
简要总结
This is a prospective, multicenter, randomized, double-blind, placebo-controlled, parallel-group 3-arm study to investigate 2 different doses of subcutaneous (SC) IgPro20 compared with placebo for maintenance treatment of patients with CIDP.
Patients who received at lease 1 dose of intravenous immunoglobulin (IVIG) within 8 weeks before screening will be assessed during 4 separate study periods. Patients first undergo a Screening Period, followed by an IgG Dependency Test Period of up to 12 weeks to test for ongoing need of IgG. Those patients experiencing CIDP relapse during this test period will be administered a standardized IVIG regimen during an IVIG Re-stabilization Period. Patients with improved and maintained adjusted inflammatory neuropathy cause and treatment scale (INCAT) in the IVIG Re-stabilization Period will continue to the SC Treatment Period of the study. Patients entering the 24 week SC Treatment Period will be randomized to receive weekly infusions of 1 of 2 IgPro20 doses (0.2 or 0.4 g/kg body weight) or placebo.
The overall study duration is up to 52 weeks. Clinical outcomes will be assessed by the Inflammatory Neuropathy Cause and Treatment (INCAT) score, maximum grip strength, the Medical Research Council (MRC) sum score, the Rasch-built Overall Disability Scale (R-ODS), and electrophysiological evaluations.
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
- •Definite or probable CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria
- •An IVIG treatment during the last 8 weeks prior to enrollment.
- •Age ≥18 years.
- •Written informed consent for study participation obtained before undergoing any study-specific procedures.
排除标准
- •Any polyneuropathy of other causes
- •Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments
- •Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study
- •History of thrombotic episodes within the 2 years prior to enrolment
- •Known allergic or other severe reactions to blood products including intolerability to previous IVIG
研究组 & 干预措施
IgPro20 low dose
干预措施: IgPro20 (low dose) (Biological)
IgPro20 high dose
干预措施: IgPro20 (high dose) (Biological)
Placebo
干预措施: Placebo (Biological)
Placebo
干预措施: IgPro10 (Biological)
IgPro20 high dose
干预措施: IgPro10 (Biological)
IgPro20 low dose
干预措施: IgPro10 (Biological)
结局指标
主要结局
Percentage (%) of Subjects With CIDP Relapse or Are Withdrawn for Any Other Reason During the Subcutaneous (SC) Treatment Period
时间窗: Up to 25 weeks
Relapse is defined as an increase of at least 1 INCAT score point (except for the increase from 0 to 1 in the upper limb score). The INCAT score is a 10-point scale that covers the functionality of legs and arms, and has been successfully used to measure treatment effects in various CIDP studies. Scores for arm disability range from 0 ("No upper limb problems") to 5 ("Inability to use either arm for any purposeful movement"), and scores for leg disability range from 0 ("Walking not affected") to 5 ("Restricted to wheelchair, unable to stand and walk a few steps with help"). The INCAT (total) score is the sum of these 2 scores and ranges from 0 to 10. For the "adjusted" INCAT score, changes in the function of the upper limbs from 0 (normal) to 1 (minor symptoms) or from 1 to 0 were not recorded as deterioration or improvement because these changes are not considered clinically significant.
次要结局
- Number of Subjects With Adverse Events During the SC Treatment Period(Up to 28 weeks)
- Number of Adverse Events Per IgPro10 Infusion During Rescue Therapy(Up to 13 weeks)
- Percent of Subjects With Adverse Events During IgPro10 Rescue Therapy(Up to 13 weeks)
- Number of Subjects With Adverse Events During IgPro10 Re-stabilization Therapy(Up to 13 weeks)
- Number of Adverse Events Per IgPro20 Infusion During the SC Treatment Period(Up to 28 weeks)
- Percentage of Subjects With Adverse Events During the SC Treatment Period(Up to 28 weeks)
- Percent of Subjects With Adverse Events During IgPro10 Re-stabilization Therapy(Up to 13 weeks)
- Change in the Medical Research Council (MRC) Sum Scores During the SC Treatment Period(Baseline and up to 25 weeks)
- Change in Mean Grip Strength During IgPro10 Re-stabilization Therapy(Reference visit and up to 13 weeks)
- Change in Inflammatory Neuropathy Cause and Treatment (INCAT) Scores During the SC Treatment Period(Baseline and up to 25 weeks)
- Median Change From Baseline in the Mean Grip Strength Scores of the Dominant Hand During the SC Treatment Period(Baseline and up to 25 weeks)
- Change in Rasch-built Overall Disability Scale (R-ODS) Scores During the SC Treatment Period(Baseline and up to 25 weeks)
- Number of Adverse Events Per IgPro10 Infusion During Re-stabilization Therapy(Up to 13 weeks)
- Number of Subjects With Adverse Events During IgPro10 Rescue Therapy(Up to 13 weeks)
- Time to Improvement During IgPro10 Re-stabilization Therapy(Up to 13 weeks)
- Time to CIDP Relapse or Withdrawal Due to Any Other Reason During the SC Treatment Period(Up to 25 weeks)
- Change in INCAT During IgPro10 Re-stabilization Therapy(Reference visit and up to 13 weeks)
- Time to Improvement After CIDP Relapse During IgPro10 Rescue Therapy(Up to 13 weeks)
- Change in MRC Sum Score During IgPro10 Rescue Therapy(Before first rescue IgPro10 infusion and up to 13 weeks)
- Change in MRC Sum Score During IgPro10 Re-stabilization Therapy(Reference visit and up to 13 weeks)
- Change in R-ODS During IgPro10 Re-stabilization Therapy(Reference visit and up to 13 weeks)
- Change in Mean Grip Strength During IgPro10 Rescue Therapy(Before first rescue IgPro10 infusion and up to 13 weeks)
- Change in R-ODS During IgPro10 Rescue Therapy(Before first rescue IgPro10 infusion and up to 13 weeks)
- Change in INCAT During IgPro10 Rescue Therapy(Before first rescue IgPro10 infusion and up to 13 weeks)