An Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy and Safety of Biostate® in Subjects With Von Willebrand Disease.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 22
- 试验地点
- 6
- 主要终点
- Number of spontaneous or traumatic NSB events
研究概览
简要总结
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
Pharmacokinetic Component:
PK parameters will be determined from a subgroup of subjects. Subjects who complete the PK component will subsequently continue in the efficacy component of the study, either continuing on a previously established prophylaxis regimen or continuing to receive on-demand treatment with the occurrence of non-surgical bleeding (NSB) events.
Efficacy Component:
Three treatment arms are defined for the efficacy component of the study. (1) Subjects who are currently being treated on a set prophylaxis regimen with a VWF product at the time of study entry will be enrolled in the "Prophylaxis" arm. (2) Subjects not being treated on a set prophylaxis regimen at the time of study entry who require a VWF product for the treatment of NSB events will be enrolled in the "On-demand" arm and commence using Biostate in the treatment of NSB events. (3) Subjects enrolled in the "On-demand" arm have the possibility to enter the "Cross-over to Prophylaxis" arm to receive an additional 12 months of prophylactic treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with VWD
- •Desmopressin acetate (DDAVP) treatment is ineffective or contraindicated or not available
- •Evidence of vaccination against hepatitis A and B (or presence of antibodies against hepatitis A and B) within 10 years prior to their first dose of Biostate®
- •Written informed consent given
- •Exclusion Criteria (for participation in the PK component):
- •Actively bleeding immediately prior to initial PK period
- •Have received DDAVP or a VWF product in the 5 days prior to their first dose of study product
- •Have Type 2B, 2N or 2M VWD
- •Exclusion Criteria (for all subjects):
- •Requiring a VWF product for a planned surgical procedure at enrolment
- •Have received aspirin or other non-steroidal anti-inflammatory drugs within 7 days prior to their first dose of study product
- •Known history of, or are suspected to have, VWF or FVIII inhibitors
- •Suffering an 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 Biostate®, VWF/FVIII concentrates, or human albumin
- •Impaired liver function at screening
- •Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
- •Participation in a clinical study or use of an investigational compound in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period.
- •Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
排除标准
- 未提供
研究组 & 干预措施
PK
Includes subjects participating in the pharmacokinetic component of the study.
干预措施: Biostate® (Biological)
Prophylaxis
Includes subjects receiving 12 months of prophylactic therapy.
干预措施: Biostate® (Biological)
On-demand
Includes subjects receiving 12 months of on-demand treatment.
干预措施: Biostate® (Biological)
Cross-over to prophylaxis
Includes subjects completing 12 months of on-demand treatment (the "On-demand" arm) who cross-over to prophylactic therapy for an additional 12-month period.
干预措施: Biostate® (Biological)
结局指标
主要结局
Number of spontaneous or traumatic NSB events
时间窗: From Day 1 until final study visit
Pharmacokinetic parameters for vWF and FVIII (PK arm only)
时间窗: Up to 72 hours following infusions on Day 1 and approximately Day 180
Haemostatic efficacy at time of non-surgical bleeding (NSB) event
时间窗: From Day 1 until final study visit
Number of treatments with blood product transfusions required to resolve any bleeding event
时间窗: From Day 1 until final study visit
Haemostatic efficacy overall
时间窗: Monthly (prophylactic therapy) or once every 3 months (for on-demand use)
vWF/FVIII concentrate usage (number of infusions, IU/kg per dose, per event, per month and per year)
时间窗: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
时间窗: From Day 1 until final study visit
次要结局
- Development of vWF inhibitors(From Day 1 until final study visit)
- Development of FVIII inhibitors(From Day 1 until final study visit)