A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 35
- 试验地点
- 9
- 主要终点
- Subjective assessment of Haemostatic efficacy
研究概览
简要总结
The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects between 0 and <12 years of age.
- •Diagnosed with severe haemophilia A (FVIII:C <1%), and pre-treated for a minimum of 20 to 50 exposure days.
- •Have evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunisation), as documented in the medical notes at enrolment.
- •The subject and/or legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
排除标准
- •For all subjects at Day 1: Are actively bleeding.
- •Have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma or desmopressin acetate in the 4 days prior to their dosing within the PK component.
- •Have a known history of, or who are suspected of having FVIII inhibitors.
- •Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of administration of the IMP.
- •Have an impaired liver function ie, bilirubin >1.5 x upper limit of normal (ULN) and/or aspartate/alanine aminotransferase (AST/ALT) >2.5 x ULN (referring to limits of the laboratory that performs the determination) at Screening.
- •Are human immunodeficiency virus [HIV]-1/-2 antibody positive with a viral load of >200/µL.
- •Suffer from an acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study.
- •Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin cofactor (VWF:RCo) level <50 IU/dL at Screening.
- •Have a known or suspected hypersensitivity or previous evidence of severe side effects to a plasma-derived FVIII product or to human albumin.
- •Have participated in a clinical study or used an investigational compound in another study (eg, a new chemical entity not registered for clinical use) in the 3 months preceding the first day of IMP administration, or are planning to enter such a study during the study period.
- •Unwillingness and/or inability to comply with the study requirements.
研究组 & 干预措施
Biostate
干预措施: Biostate (Biological)
结局指标
主要结局
Subjective assessment of Haemostatic efficacy
时间窗: Over minimum of 50 exposure days
Incremental recovery of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Half-life of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Area under the concentration curve (AUC) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Mean residence time (MRT) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution at steady state (Vss) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Maximum Plasma Concentration (Cmax) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Minimum Plasma Concentration (Cmin) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Time the maximum concentration occurs (tmax) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Total clearance of the drug from the body (CL=dose/AUC) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Number of infusions per bleeding event
时间窗: 1 day
Number of infusions per month
时间窗: 1 month
Number of infusions per year
时间窗: 1 year
Dose (IU/kg) per bleeding event
时间窗: 1 day
Dose (IU/kg) per month
时间窗: 1 month
Dose (IU/kg) per year
时间窗: 1 year
次要结局
- Frequency of adverse events (AEs)(6 months)
- Severity of AEs per subject(6 months)
- Severity of AEs per infusion(6 months)
- Relatedness of AEs per subject(6 months)
- Relatedness of AEs per infusion(6 months)
- Development of FVIII inhibitors(Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit)