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临床试验/NCT03315130
NCT03315130已完成2 期

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects With Generalized Myasthenia Gravis

Ra Pharmaceuticals30 个研究点 分布在 2 个国家目标入组 45 人开始时间: 2017年10月11日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
2 期
状态
已完成
入组人数
45
试验地点
30
主要终点
Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 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 85 Years（Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IVa] at Screening
  • •Positive serology for acetylcholine receptor (AChR) autoantibodies
  • •QMG score ≥ 12 at Screening and Randomization
  • •No change in corticosteroid dose for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period
  • •No change in immunosuppressive therapy, including dose, for at least 30 days prior to Randomization or anticipated to occur during the 12-week Treatment Period

排除标准

  • •Thymectomy within 6 months prior to Randomization or scheduled to occur during the 12 week Treatment Period
  • •History of meningococcal disease
  • •Current or recent systemic infection within 2 weeks prior to Randomization or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Randomization

研究组 & 干预措施

0.1 mg/kg zilucoplan (RA101495)

Experimental

干预措施: zilucoplan (RA101495) (Drug)

0.3 mg/kg zilucoplan (RA101495)

Experimental

干预措施: zilucoplan (RA101495) (Drug)

Placebo

Placebo Comparator

干预措施: Placebo (Drug)

结局指标

主要结局

Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score

时间窗: From Baseline to Week 12

The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for myasthenia gravis (MG). The scale consists of 13 individual assessments, each scored on a 0-3 point scale (i.e., 0=none, 1=mild, 2=moderate, and 3=severe). The total score is the sum of the individual scores with a range of 0-39. Higher scores are representative of more severe impairment.

次要结局

  • Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale(From Baseline to Week 12)
  • Main Portion: Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life 15r (MG-QOL15r) Survey(From Baseline to Week 12)
  • Main Portion: Change From Baseline to Week 12 in the MG Composite Scale Total Score(From Baseline to Week 12)
  • Main Portion: Percentage of Participants With >= 3-point Reduction in QMG Total Score at Week 12(Week 12)
  • Main Portion: Percentage of Participants Who Required Rescue Therapy Over the 12-week Treatment Period(Up to Week 12)
  • Main Portion: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(From Baseline to Week 12)
  • Main Portion: Change From Baseline in Sheep Red Blood Cell (sRBC) Lysis Assay at Week 12 (Pre-dose)(Baseline and Week 12 (Pre-dose))
  • Main Portion: Change From Baseline in C5 Levels at Week 12 (Pre-dose)(Baseline and Week 12 (Pre-dose))
  • Main Portion: Plasma Concentration of RA101495 and Its Major Metabolites(1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12)
  • Main Portion: Maximum Plasma Concentration (Cmax) on Day 1(Pre-dose, 1, 3 and 6 hours postdose on Day 1)
  • Main Portion: Time Corresponding to Cmax (Tmax) on Day 1(Pre-dose, 1, 3 and 6 hours postdose on Day 1)
  • Main Portion: Metabolites (RA102758 and RA103488) to Parent Ratio(Pre-dose, 1, 3 and 6 hours postdose on Day 1; Pre-dose on Week 1, 2, 4, 8 and 12)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (30)

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