A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of MY008211A Tablets in PNH Patients Who Are Naive to Complement Inhibitor Therapy
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 66
- 试验地点
- 2
- 主要终点
- The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
研究概览
简要总结
The main purpose of this study is to evaluate the efficacy of MY008211A compared to eculizumab in PNH patients.
详细描述
This is a multi-center, randomized, open-label, active comparator-controlled, parallel group study. The purpose of this study is to determine whether MY008211A compared to eculizumab is efficacious and safe for the treatment of PNH patients who were naive to complement inhibitor therapy.
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
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
- •Mean hemoglobin level <100 g/L at screening.
- •LDH > 1.5 x Upper Limit of Normal (ULN) at screening.
- •Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.
排除标准
- •Patients with reticulocytes <100x10^9/L; platelets <30x10^9/L; neutrophils <0.5x10^9/L.
- •History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
- •Known or suspected hereditary complement deficiency.
- •Previous bone marrow or hematopoietic stem cell transplantation.
- •Previous splenectomy.
- •A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.
研究组 & 干预措施
MY008211A tablets
MY008211A tablets 400mg BID
干预措施: MY008211A tablets (Drug)
Eculizumab
Eculizumab Injection
干预措施: Eculizumab Injection (Drug)
结局指标
主要结局
The proportion of Participants With Sustained Hemoglobin Levels of ≥ 120 g/L in the Absence of Red Blood Cell Transfusions
时间窗: between Day 126 and Day 168
The proportion of patients with sustained hemoglobin levels ≥ 120 g/L among those without RBC transfusion (defined as no red blood cell infusion after D14 to D168).
次要结局
- Change (Expressed as Percentages) in LDH level from baseline(between Day 126 and Day 168)
- Change in reticulocyte count from baseline(between Day 126 and Day 168)
- The proportion of patients without RBC transfusion(between Day 14 and Day 168)
- The Clinical BTH Rate(between Day 1 and Day 168)
- The Major Adverse Vascular Events Rate(between Day 1 and Day 168)
- The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion(between Day 126 and Day 168)
- The proportion of patients with LDH < 1.5 ULN among those without RBC transfusion.(between Day 126 and Day 168)
- Change in FACIT-F score from baseline(between Day 126 and Day 168)