NCT02048111撤回3 期
Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients With Severe Hemophilia B
Cangene Corporation2 个研究点 分布在 2 个国家开始时间: 2014年3月1日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 试验地点
- 2
- 主要终点
- Number of study subjects with adverse events
研究概览
简要总结
To evaluate the safety (acute adverse effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding and control of hemorrhaging during prophylaxis of IB1001 in subjects with hemophilia B.
详细描述
Primary Objectives:
- to evaluate safety of IB1001 within the first 50 exposure days,
- to determine IB1001 pharmacokinetics (PK), and
- to assess efficacy of IB1001 prophylaxis with respect to breakthrough bleeding and with respect to control of hemorrhaging in subjects with severe hemophilia B within the first 50 exposure days
Secondary Objectives:
- to evaluate long-term safety of IB1001; and
- to evaluate long term efficacy of IB1001.
Exploratory Objectives:
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of at least 12 years
- •Body Mass Index of ≤ 29, with a minimum body weight of 40 kg
- •Written Institutional Review Board (IRB)/ Ethics Committee (EC)-approved informed consent form (ICF)
- •Willingness to make the required study visits, and follow instructions while enrolled in the study (up to 12 months)
- •Severe (factor IX activity ≤2 U/dL) hemophilia B patients with a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months or in the event the subject is on prophylaxis, a minimum of 3 bleeding episodes over the preceding 6 months or 6 bleeding episodes over the preceding 12 months prior to being placed on prophylaxis
- •Subjects must be on prophylaxis or switch to a prophylaxis regimen for the duration of the PK and Treatment/Continuation Phase of the study
- •Previously treated patients with a minimum of 150 exposure days to a factor IX preparation
- •Willingness to adhere to the 5-day washout of any factor IX replacement therapy prior to PK evaluations
- •Immunocompetent (CD4 count >400/mm3) and not receiving immune modulating or chemotherapeutic agents
- •Platelet count at least 150,000/mm3
- •Liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times the upper limit of the normal range
- •Total bilirubin ≤1.5 times the upper limit of the normal range
- •Renal function: serum creatinine ≤1.25 times the upper limit of the normal range
- •Hemoglobin ≥7 g/dL at the time of the blood draw
排除标准
- •History of factor IX inhibitor ≥0.6 BU (Bethesda units)
- •Existence of another coagulation disorder
- •Evidence of thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
- •Use of an investigational drug within 30 days prior to study entry
- •Previous use of IB1001
- •Use of medications that could impact hemostasis, such as aspirin
- •Hypersensitivity to the active substance or to any of the excipients in the investigational products
- •Known allergic reaction to hamster proteins
- •History of poor compliance, a serious medical or social condition, or any other circumstance that, in the opinion of the investigator, would interfere with participation or compliance with the study protocol
- •History of adverse reaction to either plasma-derived factor IX or recombinant factor IX that interfered with the subject's ability to treat bleeding episodes with a factor IX product
研究组 & 干预措施
IB1001
Experimental
干预措施: IB1001 (Biological)
结局指标
主要结局
Number of study subjects with adverse events
时间窗: Within 6 months
Information on adverse events is collected after each infusion of study drug by a study subject. Assessment of adverse events is then performed by an investigator after 5 infusions of study drug, 1 month, 2 months, 3 months and 6 months of study drug treatment.
次要结局
- Number of bleeding episodes divided by number of months of observation(Within 12 months)
研究者
研究点 (2)
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