An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 25
- 试验地点
- 6
- 主要终点
- To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
研究概览
简要总结
The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
详细描述
This is a multicenter, open-label, single arm study. The primary objective is to assess the long-term safety and tolerability of OMS906 in patients with PNH. Secondary objectives of this study include assessment of the long-term efficacy of OMS906 in patients with PNH. Patients will receive OMS906 5 mg/kg administered as intravenous (IV) injections at 8-week intervals.
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 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Have completed the last dosing visit of the prior OMS906 PNH study.
- •Female patients of child bearing potential must have a negative result from a highly sensitive urine pregnancy test prior to each dose of OMS
- •Females must use highly effective birth control to prevent pregnancy during the clinical trial and for 20 weeks following their last dose of study drug.
- •Males must use highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 20 weeks after last dose of study drug.
- •Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Hemophilus influenza and agree to maintain vaccination throughout the study.
- •Have provided informed consent
排除标准
- •Platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at the start of the Evaluation Period.
- •Elevation of liver function tests, defined as total bilirubin > 2 x ULN, direct bilirubin > 1.5 x ULN, and elevated transaminases (alanine or aspartate aminotransferase), > 2 X ULN unless due to PNH-related hemolysis.
- •History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
- •Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.
- •Pregnant, planning to become pregnant, or nursing female patients.
- •History of any significant medical, neurologic, or psychiatric disorder that in the opinion of the investigator would make the patient unsuitable for participation in the long-term extension.
- •Unable or unwilling to comply with the requirements of the study.
研究组 & 干预措施
OMS906 study drug
OMS906 study drug repeat-dose 5 mg/kg IV administration at 8-week intervals.
干预措施: OMS906 study drug (Drug)
结局指标
主要结局
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
时间窗: 104 weeks
Treatment-emergent adverse events, including clinically significant clinical laboratory tests, 12-lead electrocardiograms, vital signs, and physical examinations recorded as an adverse event.
次要结局
- To assess efficacy measured by hemoglobin (Hgb).(6 month intervals)
- To assess efficacy by transfusion requirements.(Weeks 48 and 96)
- To assess efficacy by measurement of lactate dehydrogenase (LDH).(Weeks 48 and 96)
- To assess efficacy by measurement of reticulocyte count.(Weeks 48 and 96)
- To assess efficacy by measurement of clinical breakthrough hemolysis.(Weeks 48 and 96)
- To assess population PK Cmax of OMS906.(Weeks 48 and 96)
- To assess population PK AUC of OMS906.(Weeks 48 and 96)
- To assess population PK terminal half life of OMS906.(Weeks 48 and 96)
- To assess PD of OMS906(Weeks 48 and 96)
- OMS906 anti-drug antibodies (ADA).(Weeks 24, 48, 72, and 96)
- Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.(Weeks 24, 48, 72, and 96)