A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- Clearance
研究概览
简要总结
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
- •Documented congenital FXIII deficiency that requires prophylactic treatment with a FXIII containing product.
- •Males and females of any age with congenital FXIII deficiency.
- •Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive
排除标准
- •Diagnosis of acquired FXIII deficiency
- •Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
- •Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- •Known or suspected to have antibodies towards FXIII
- •Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
- •Positive result at screening for human immunodeficiency virus (HIV)
- •Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
- •Fibrinogen < lower limit of normal
- •Active bleeding
- •Pregnant or breast-feeding
- •Intention to become pregnant during the course of the study
- •Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
- •Surgical procedure anticipated during the study period
- •Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance
结局指标
主要结局
Clearance
时间窗: 12 weeks
Trough FXIII Concentration at Steady State
时间窗: 12 weeks
Time to Peak Concentration
时间窗: 12 weeks
Volume of Distribution at Steady State
时间窗: 12 weeks
Terminal Half-life
时间窗: 12 weeks
Peak FXIII Concentration at Steady State
时间窗: 12 weeks
Incremental Recovery
时间窗: 12 weeks
Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.
Area Under the Curve at Steady State
时间窗: 12 weeks
Mean Residence Time
时间窗: 12 weeks
次要结局
- Laboratory Safety Parameters(16 weeks)
- Adverse Events(16 weeks)
- Vital Signs(16 weeks)