An Open-label, Multi-centre Post-marketing Study to Assess the Efficacy and Safety of Voncento® in Subjects With Von Willebrand Disease
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 26
- 试验地点
- 12
- 主要终点
- Annual bleeding rate
研究概览
简要总结
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor (VWF) product is required as on-demand therapy, for prophylactic therapy, or during surgery. Subjects will be treated with Voncento either as an on-demand regimen (eg, to treat a non-surgical spontaneous or traumatic bleeding event) or prevention regimen (eg, to prevent an anticipated bleeding event) at a dose prescribed by the Investigator in accordance with the Voncento Summary of Product Characteristics (SmPC), or with a prophylaxis regimen (regular treatment with Voncento at a frequency of 1-3 times per week). Voncento will also be given to prevent and treat any surgical bleeding events.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of severe type 1, 2A, or 3 VWD where Von Willebrand Factor: Ristocetin Cofactor (VWF:RCo) is <20% at screening
- •Desmopressin acetate treatment is ineffective, contraindicated, or not available for subject (type 3 VWD subjects only).
- •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.
- •Require a VWF product to control a non-surgical bleeding (NSB) event or for ongoing prophylactic therapy.
排除标准
- •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 Voncento, VWF / FVIII concentrates, or human albumin.
- •Participated in another interventional clinical study within 30 days before the first administration of Voncento or at any time during the study.
- •Females who are pregnant, breast-feeding or who have a positive pregnancy test at screening
- •Alcohol, drug, or medication abuse within 1 year before the study.
- •Currently receiving a therapy not permitted during the study.
- •Previous participation in a Voncento / Biostate study.
结局指标
主要结局
Annual bleeding rate
时间窗: For the duration of the subject's participation in the study (approximately 12 months).
The number of bleeding events per year
Haemostatic efficacy - NSB event
时间窗: Assessed daily by the subject until the bleed stops, for the duration of the subject's participation in the study (approximately 12 months). Each bleeding event is also to be assessed retrospectively by the Investigator.
Subject's and investigator's assessment of haemostatic efficacy of Voncento in its usage for a non-surgical bleeding (NSB) event. Assessments of haemostatic efficacy will be based on a 4-point ordinal scale of Excellent, Good, Moderate or None.
Number of infusions - NSB event
时间窗: For the duration of the subject's participation in the study (approximately 12 months).
Number of infusions of Voncento required to treat an NSB event.
Total dose of Voncento - NSB event
时间窗: For the duration of the subject's participation in the study (approximately 12 months).
Total dose of Voncento (in international units of Von Willebrand Factor: Ristocetin Cofactor) required to treat an NSB event.
Number of NSB events per month
时间窗: From Day 1 until final study visit, approximately 12 months.
次要结局
- Number of infusions - surgical bleeding event(For the duration of the subject's participation in the study (approximately 12 months).)
- Number of subjects with VWF or FVIII inhibitors(At screening, Day 1 and approximately Months 3, 6, 9 and 12, for each subject.)
- Assessment of blood loss during a surgical procedure(During surgery, for any surgical procedure during the subject's participation in the study (approximately 12 months).)
- Overall adverse events(From Day 1 until the final study visit for each subject (approximately 12 months))
- Haemostatic efficacy - surgical event(Assessed during and after surgery until the bleeding stops, for any surgical procedure during the subject's participation in the study (approximately 12 months).)
- Haemostatic efficacy - prophylaxis(Approximately every month (subject assessment) and every 3 months (Investigator assessment) for the duration of the subject's participation in the study (approximately 12 months).)
- Total dose of Voncento - surgical bleeding event(For the duration of the subject's participation in the study (approximately 12 months).)