A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 63
- 试验地点
- 1
- 主要终点
- Adverse Events (AEs)(Serious and Non-serious)
研究概览
简要总结
This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For subjects who participated in F13CD-1725:
- •Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
- •For all other subjects:
- •Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
- •Body weight at least 20 kg
排除标准
- •Known neutralizing antibodies (inhibitors) towards FXIII
- •Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- •Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
- •Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods
研究组 & 干预措施
A
干预措施: catridecacog (Drug)
结局指标
主要结局
Adverse Events (AEs)(Serious and Non-serious)
时间窗: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
次要结局
- Antibody and Inhibitor Development(From week 0 to week 52)