NCT06134414尚未招募2 期
A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 40
- 主要终点
- The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing
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.
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 naïve 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 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.
- •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
Arm 1 MY008211A low dose
Experimental
Participants will receive MY008211A at a dose of 400 mg orally b.i.d
干预措施: MY008211A tablets (Drug)
Arm 2 MY008211A high dose
Experimental
Participants will receive MY008211A at a dose of 600 mg orally b.i.d
干预措施: MY008211A tablets (Drug)
结局指标
主要结局
The proportion of subjects with an increase in hemoglobin concentration ≥ 20 g/L from baseline among subjects who do not receive RBC transfusion after 4 weeks of dosing
时间窗: Day 70
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 in hemoglobin concentration from baseline in patients without RBC transfusion(Day14, 21, 28, 42, 56 and 70)
- Change in the average weekly amount of RBC transfused during the efficacy observation period(Day70)
- Change in the amount of fragment Bb of CFB in plasma from baseline(Day14, 28, 56 and 70)
- Change in LDH level from baseline(Day7, 14, 21, 28, 42, 56 and 70)
- Change From Baseline in FACIT-Fatigue Questionnaire(Day7, 14, 21, 28, 42, 56 and 70)
- The proportion of patients with an increase in hemoglobin ≥ 20 g/L from baseline among those without RBC transfusion(Day14, 21, 28, 42, 56)
- The proportion of patients with hemoglobin ≥ 120 g/L among those without RBC transfusion(Day14, 21, 28, 42, 56 and 70)
- The proportion of patients with hemolysis controlled(Day7, 14, 21, 28, 42, 56 and 70)
- The proportion of patients without RBC transfusion(Day14, 21, 28, 42, 56 and 70)
- Changes from baseline in alternative complement pathway activity(Day14, 28, 56 and 70)
- Change in the level of PNH RBC clones from baseline in patients without RBC transfusion.(Day70)
- Change in reticulocyte count from baseline in patients without RBC transfusion(Day7, 14, 21, 28, 42, 56 and 70)
- Change in indirect bilirubin level from baseline(Day7, 14, 21, 28, 42, 56 and 70)
- Incidence of Adverse Events (AEs) between Day 1 and Day 70(Day 70)
研究者
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