A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 17
- 试验地点
- 7
- 主要终点
- Haemostatic efficacy
研究概览
简要总结
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
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
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects between 0 and <12 years of age
- •Diagnosed with VWD Type 1, 2A, or 3
- •Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- •von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- •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 immunization
- •Written informed consent given
排除标准
- •Active bleeding immediately prior to initial PK period
- •Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- •Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- •Known history or suspicion of having VWF or FVIII inhibitors
- •Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- •Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
- •Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
- •Unwillingness and/or inability to comply with the study requirements
结局指标
主要结局
Haemostatic efficacy
时间窗: From Day 1 until final study visit
Incremental Recovery of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Area under the concentration curve (AUC) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Time to maximum concentration (tmax) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Time to maximum concentration (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
Mean residence time (MRT) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Volume of distribution of steady state (Vss) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Volume of distribution of 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
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 VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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
Minimum plasma concentration (Cmin) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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
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
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
Maximum plasma concentration (Cmax) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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
Clearance (CL) of VWF
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
Clearance (CL) of FVIII
时间窗: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
次要结局
- Development of FVIII inhibitors(Sample taken at baseline, then every 3 months up to 12 months)
- Frequency of adverse events (AEs) per subject(13 months)
- Severity of AEs per infusion(13 months)
- Severity of AEs per subject(13 months)
- Frequency of adverse events (AEs) per infusion(13 months)
- Relatedness of AEs per infusion(13 months)
- Relatedness of AEs per subject(13 months)
- Development of VWF inhibitors(Sample taken at baseline, then every 3 months up to 12 months)