NCT06050226已完成2 期
A Multi-center, Randomized, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of MY008211A Tablets in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis.
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
- 34
- 试验地点
- 1
- 主要终点
- Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
研究概览
简要总结
The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH , showing signs of active hemolysis, in China.
详细描述
The purpose of this study is to determine whether MY008211A is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy, including anti-C5 antibody.
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, BMI≥18 kg/m2，with a diagnosis of PNH confirmed by laboratory tests, according to the PNH diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 edition) , and flow cytometry with clone size ≥ 10%.
- •Mean hemoglobin level <100 g/L.
- •LDH > 1.5 x Upper Limit of Normal (ULN)
- •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.
- •Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.
- •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
Arm1：low MY008211A dose
Experimental
Participants will receive low MY008211A dose orally b.i.d
干预措施: MY008211A tablets (Drug)
Arm2：high MY008211A dose
Experimental
Participants will receive high MY008211A dose orally b.i.d
干预措施: MY008211A tablets (Drug)
结局指标
主要结局
Proportion of participants achieving a sustained increase in hemoglobin levels of ≥ 20 g/L in the absence of red blood cell transfusion.
时间窗: up to 84 days
Proportion of participants achieving a sustained increase from baseline in hemoglobin levels of ≥ 20 g/L assessed , in the absence of red blood cell transfusions
次要结局
- Change from baseline in hemoglobin concentration.(up to 84 days)
- Change from baseline in serum LDH levels.(up to 84 days)
- Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions.(up to 84 days)
- Change from baseline in Reticulocyte count.(up to 84 days)
- Changes from baseline in transfusion volume.(up to 84 days)
- Change in the level of PNH red cell clones.(up to 84 days)
- Occurrences of AEs occurring between Day 1 and Day 84.(up to 84 days)
研究者
研究点 (1)
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