A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 9
- 试验地点
- 6
- 主要终点
- The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months
研究概览
简要总结
The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months.
The secondary objectives of the study are:
- To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
- To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks).
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
- — 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children with hereditary severe or moderate FX deficiency (FX:C <5 IU/dL), based on their lowest reliable FX:C recorded.
- •Children under 12 years old, whose parent/guardian has given informed consent.
- •Children with a history of severe bleeding e.g.: intracranial haemorrhage, before starting prophylactic therapy, OR a mutation in the F10 gene causing a documented severe bleeding phenotype.
排除标准
- •Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia
- •Children must have no history or suspicion of inhibitors to factor X.
- •Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
- •Children with a history of unreliability or non-cooperation.
- •Children who are participating or have taken part in another trial within the last 30 days.
- •Children planning more than 4 weeks' continuous absence from the locality of the investigational site, between the Screening Visit and the End of Study Visit at approximately 6 months (26 weeks) post-Baseline.
研究组 & 干预措施
FACTOR X
At the Baseline Visit, eligible children will receive a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children will be treated with FACTOR X prophylactically for a period of 6 months (26 weeks).
A dosing regimen of 40-50 IU/kg twice a week is recommended, but is not mandatory. Each dose of FACTOR X must not exceed 60 IU/kg.
干预措施: FACTOR X (Biological)
结局指标
主要结局
The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months
时间窗: 6 months
The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months. The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.
次要结局
- Safety of FACTOR X: Number of Participants Experiencing Adverse Events(6 months)
- Pharmacokinetics: FX:C Incremental Recovery(Baseline Visit and End of Study Visit, 30 minutes post-dose)