An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Percentage of Participants Who Achieved Lactate Dehydrogenase (LDH) Less Than or Equal to (≤) 1.5* ULN From Baseline to Week 26
研究概览
简要总结
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
The secondary objectives of the study are:
- To evaluate the long-term effect of REGN3918 on intravascular hemolysis
- To assess the concentrations of total REGN3918 in serum
- To evaluate the occurrence of the immunogenicity of REGN3918
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 with PNH who have completed, without discontinuation, study treatment in one of the parent studies in which they participated (either R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853)
排除标准
- •Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (for example, repetitive non-compliance with dosing by the patient)
- •Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- •NOTE: Other protocol defined exclusion criteria apply
研究组 & 干预措施
REGN3918
Participants who have completed 1 of the 2 parent studies (R3918-PNH-1852 [NCT03946748] or R3918-PNH-1853)
干预措施: REGN3918 (Drug)
结局指标
主要结局
Percentage of Participants Who Achieved Lactate Dehydrogenase (LDH) Less Than or Equal to (≤) 1.5* ULN From Baseline to Week 26
时间窗: Baseline up to Week 26
Percentage of participants who achieved LDH ≤1.5\* Upper limit of normal (ULN) over Week 26, defined as LDH ≤1.5\*ULN from baseline up to Week 26 were reported. A participant was considered to have met the criteria for adequate control of intravascular hemolysis if all of their LDH readings from the baseline through Week 26 inclusive or through the analysis end date, whichever is earlier, had values ≤ 1.5\*ULN.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
时间窗: Baseline up to Week 104
An adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs was defined as AEs that developed or worsened during the on-treatment period. SAE was defined as any untoward medical occurrence that resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. TEAEs included both Serious TEAEs and non-serious TEAEs.
次要结局
- Percentage of Participants Who Had Breakthrough Hemolysis Through Week 26 and 78(At Week 26 and 78)
- Overall Rate of Transfusion With Red Blood Cell (RBCs) Through Week 26(Baseline up to Week 26)
- Percentage of Participants Who Are Transfusion-free (With RBCs) Through Week 26 and 78(At Week 26 and 78)
- Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis Through Week 78(Baseline up to Week 78)
- Percentage of Participants Who Achieved Normalization of Intravascular Hemolysis Through Week 26 and Week 78(Baseline, Week 26 and 78)
- Changes From Baseline in LDH Levels at Week 26, 78, and 104(Baseline, Week 26, 78, and 104)
- Percent Change From Baseline in LDH Levels at Week 26, 78, and 104(Baseline, Week 26, 78, and 104)
- Change From Baseline in Red Blood Cell (RBC) Hemoglobin Levels at Week 26, 78, and 104(Baseline, Week 26, 78, and 104)
- Change From Baseline in Free Hemoglobin Levels at Week 26, 78 and 104(Baseline, Week 26, 78 and 104)
- Serum Concentrations of Total REGN3918(Pre-dose (Day 1), End of infusion at Week 13, 26, 39, 52, 65, 78, 91 and 104)
- Number of Participants With Treatment-emergent Anti-Drug Antibodies (ADA) to REGN3918(Baseline up to Week 104)