NCT00885742已完成3 期
A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 41
- 试验地点
- 25
- 主要终点
- The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
研究概览
简要总结
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.
In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and 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 which 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)
- •Known Positivity for human immunodeficiency virus (HIV) or a positive result for HIV at the Screening Visit of this study or the FXIII study 2002 (NCT00883090).
- •Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal at the Screening Visit of this study or at the Day 56 Visit of Factor XIII Study BI71023_2002 (NCT00883090)
- •Fibrinogen level less than 85% of the lower limit of normal at the Screening Visit of this study or the Factor XIII Study BI71023_2002 (NCT00883090)
- •Active bleeding ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 and/or ≥ moderate between the Screening and Baseline Visits
- •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
- •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
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
时间窗: Up to week 52
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
次要结局
- Trough FXIII Concentration at Steady State(At 12, 24, 36 and 48 weeks: immediately before infusion.)
- Adverse Events(12 months)
- Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels(12 months)
- Peak FXIII Concentration at Steady State(At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.)
- Time to Peak Concentration(At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.)
- Incremental Recovery(At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.)
- Achievement of Trough Factor XIII Levels of 5% or Higher.(At 12, 24, 36 and 48 weeks: immediately before infusion.)
研究者
研究点 (25)
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