A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 19
- 试验地点
- 12
- 主要终点
- Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
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
- •Completion of a qualifying Ra Pharmaceuticals sponsored zilucoplan (RA101495) PNH study
- •Evidence of ongoing clinical benefit in the opinion of the Investigator
- •Exclusion criteria:
- •History of meningococcal disease
- •Current systemic infection or suspicion of active bacterial infection
排除标准
- 未提供
研究组 & 干预措施
Zilucoplan (RA101495)
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study
干预措施: Zilucoplan (RA101495) (Drug)
结局指标
主要结局
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
时间窗: From Day 1 until the Final Study Visit (up to Month 49)
TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start.
Percentage of Participants With Serious TEAEs
时间窗: From Day 1 until the Final Study Visit (up to Month 49)
Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe), • requires hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect.
次要结局
- Change From Baseline in Complement Component 5 (C5) Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49))
- Number of Participants With Anti-drug Antibodies (ADA)(At Day 1, Month 1, 2, 3, 6, 9, and 12)
- Change From Baseline in Total Hemoglobin Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Total Bilirubin Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Free Hemoglobin Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Haptoglobin Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Reticulocytes at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49))
- Change From Baseline in Hemoglobinuria Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49))
- Plasma Concentrations of RA101495 and Its Major Metabolite(s)(Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49))
- Maximum Plasma Concentration (Cmax) of RA101495(At Day 1, Month 1, 2, 3, 6, 9, and 12)
- Time Corresponding to Cmax (Tmax) of RA101495(At Day 1, Month 1, 2, 3, 6, 9, and 12)
- Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495(At Day 1, Month 1, 2, 3, 6, 9, and 12)
- Total Complement (CH50) Levels(At Day 1, Month 1, 2, 3, 6, 9, and 12)
- Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49))
- Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point(Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49))