An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.
研究概览
简要总结
This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.
详细描述
The purpose of this open-label, single arm, multicenter study is to evaluate the long-term safety, tolerability and efficacy of MY008211A tablets in patients with PNH and to provide access to patients who have completed (without tapering down) Phase 2 and Phase 3 trials and derived benefit from MY008211A treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.
- •Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.
排除标准
- •History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.
- •Known or suspected hereditary complement deficiency.
- •Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.
研究组 & 干预措施
MY008211A tablets
MY008211A tablets 400mg BID
干预措施: MY008211A tablets (Drug)
结局指标
主要结局
Proportion of participants with adverse events, safety laboratory parameters, vital signs, ECG.
时间窗: About 100 weeks
Safety evaluations including but not limited to adverse events, laboratory parameters, vital signs, ECG through End of Study visit every 12 weeks.
次要结局
- Proportion of participants achieving sustained hemoglobin levels ≥ 120 g/L in the absence of red blood cell transfusions(About 100 weeks)
- Change From Baseline in Hemoglobin(About 100 weeks)
- The proportion of patients without RBC transfusion.(About 100 weeks)
- The Clinical BTH Rate(About 100 weeks)
- The Major Adverse Vascular Events Rate(About 100 weeks)