NCT02565576已完成2 期
A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Preliminarily Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of CFZ533 in Patients With Moderate to Severe Myasthenia Gravis
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
- 44
- 试验地点
- 1
- 主要终点
- Mean Change From Baseline in the Quantitative Myastenia Gravis (QMG) Score at Week 25. Posterior Median Was Used as Measure Type.
研究概览
简要总结
The purpose of this study is to evaluate safety, tolerability, pharmacokinetics/pharmacodynamics and efficacy of CFZ533 as an add-on therapy to standard of care in patients with moderate to severe myasthenia gravis (MG).
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
- Double (Participant, Investigator)
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 MG class IIa to IVa inclusive (Myasthenia Gravis Foundation of America Clinical Classification).
- •Quantitative Myasthenia Gravis (QMG) score of 10 or greater. If the QMG score is < 15 no more than 4 points may be derived from items 1 or 2 (ocular motility disturbance and ptosis).
- •Documented history of acetylcholine receptor (AChR) or Muscle Specific Kinase (MuSK) antibody positive.
- •Only one immunosuppressant or immunomodulatory drug at a stable dose is allowed during the study (i) azathioprine and mycophenolate mofetil must be stable for at least 4 months prior to randomization (ii) cyclosporine must be stable for at least 3 months prior to randomization.
- •If the patient is on oral corticosteroids, methotrexate or tacrolimus at screening, the dose must be stable for at least 1 month prior to randomization.
- •If the patient is on cholinesterase inhibitors at screening, the dose must be stable for at least 2 weeks prior to randomization.
- •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, may be included in the study if they are using highly effective methods of contraception during the study and for 12 weeks after study treatment.
排除标准
- •MGFA grade I, IVb, or V disease.
- •Documented presence of unresected thymoma.
- •Patients having undergone thymectomy or thymo thymectomy (resection of thymoma) within 6 months of screening.
- •Patients having received any of the following treatments prior to randomization:
- •IVIg or plasma exchange within 8 weeks;
- •oral or IV cyclosphosphamide treatment within 3 months;
- •IV corticosteroid bolus (dose higher than 1 mg/kg) within 3 months;
- •belimumab within 6 months. For patients who received belimumab earlier, B cell count should be within normal range;
- •rituximab within 12 months. For patients who received rituximab earlier, B cell count should be within normal range;
- •any other biologic or an investigational drug within 1 month or five times thehalf-life, whichever is longer.
- •Live vaccines within 4 weeks of study drug infusion.
- •Patients who are at significant risk for TE as judged by the investigator or have any one of the following:
- •History of either thrombosis or 3 or more spontaneous abortions with or without the presence of anti-cardiolipin autoantibodies;
- •Presence of prolonged partial thromboplastin time (PTT).
研究组 & 干预措施
CFZ533
Active Comparator
CFZ533
干预措施: Placebo (Drug)
CFZ533
Active Comparator
CFZ533
干预措施: CFZ533 (Drug)
Placebo
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Mean Change From Baseline in the Quantitative Myastenia Gravis (QMG) Score at Week 25. Posterior Median Was Used as Measure Type.
时间窗: week 25
QMG score is an established validated measure of disease severity used in MG trials (Jaretzki et al 2000). The scoring system is based on quantitative testing of sentinel muscle groups by means of a 4 point scale ranging from 0 (no symptoms) to 3 (severe symptoms). The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and the total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) (Sharshar et al 2000, Bedlack et al 2005).
次要结局
- Number of Patients Who Discontinued Due to Inefficacy or Worsening(week 49)
- Mean Changes From Baseline in the Myasthenia Gravis Composite (MGC) Score. Posterior Median Was Used as Measure Type.(From baseline to week 49)
- Proportion of Patients With Improvement or Worsening by ≥ 3 Points in the QMG Score(at week 49)
- Proportion of Patients Intolerant to Steroid Taper(week 49)
- Mean Change From Baseline in the Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL)(week 25)
- Mean Changes From Baseline in the QMG Score at Week 49(week 49)
- Mean Change From Baseline in the Myasthenia Gravis Quality of Life (MG QOL-15)(week 25)
- Free CD40 on B Cells(week 1, week 25)
- Total Soluble CD40 (sCD40) in Plasma(week1, week 25)
- Plasma CFZ533 Concentration at Steady State Conditions (Week 17)(week 17)
研究者
研究点 (1)
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