NCT01233440已完成1 期
An Open-label, Multicenter, Dose-Escalation Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 25
- 试验地点
- 20
- 主要终点
- Occurrence of inhibitor against FIX
研究概览
简要总结
The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. The secondary objective of the study is to evaluate the pharmacokinetics parameters, following a single intravenous dose of rIX-FP.
详细描述
This study is comprised of both a rIX-FP dose-escalation safety segment (25, 50 and 75 IU/kg of rIX-FP), and PK evaluation of rIX-FP after a single dose of 50 IU/kg, as well as PK evaluation after a single dose of 50 IU/kg of the previously given Factor IX (FIX) product (recombinant FIX [rFIX] or plasma derived FIX [pdFIX]) which is used as the reference product.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male, 12 - 65 years, with body weight ≥ 30 kg and ≤ 120 kg
- •Documented severe Hemophilia B (FIX activity of ≤ 2%) or tested by the central laboratory at screening
- •Subjects who have received FIX products for > 150 exposure days (EDs) (estimated)
- •No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined as two consecutive positive tests - a confirmatory test on a second, separately drawn sample shortly after the previous positive test) and confirmed no detectable FIX inhibitors (negative FIX inhibitor defined as < 0.6 Bethesda Units [BU] by the central laboratory at screening
- •Subjects can be treated on-demand or under prophylactic therapy
- •Signed Informed Consent/Assent
排除标准
- •Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein
- •Any known congenital or acquired coagulation disorder other than congenital FIX deficiency
- •Platelet count < 100,000/µL
- •Immunocompromised (CD4 count < 200/mm3), (HIV positive subjects may participate in the study and protease inhibitors and antiviral therapy are permitted, at the discretion of the Investigator)
- •Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment
- •Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration > 5 times (x) the upper limit of normal (ULN)
- •Serum creatinine > 2 x ULN
- •Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months prior to enrollment
- •Use of an Investigational Medicinal Product (IMP) within 30 days prior to the first rIX-FP administration
- •Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to study entry
- •Subject currently on a dose and/or regimen of FIX that would preclude participation in the study due to possible increased risk of bleeding because of the requirement to withhold treatment during the PK sampling period
- •Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance
结局指标
主要结局
Occurrence of inhibitor against FIX
时间窗: up to 28 days after drug administration
Occurrence of antibodies against rIX-FP
时间窗: up to 28 days after drug administration
Frequency of adverse events (AEs)
时间窗: up to 14 days after drug administration
Frequency of serious adverse events (SAEs)
时间窗: up to 28 days after drug administration
次要结局
- AUC to the last sample with quantifiable drug concentration (AUC0-t)(From time of dosing up to 7 days after the dose)
- AUC extrapolated to infinity (AUCt-∞)(From time of dosing up to 7 days after the dose)
- Half-life (t1/2)(From time of dosing up to 7 days after the dose)
- Incremental recovery (IU/mL/IU/kg)(From time of dosing up to 7 days after the dose)
- Clearance(From time of dosing up to 7 days after the dose)
研究者
研究点 (20)
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