A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 56
- 试验地点
- 15
- 主要终点
- Total Annualized Bleeding Rate (TABR)
研究概览
简要总结
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 66 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who meet all of the following criteria are eligible for the study:
- •Male or female patients aged ≥5.5 years at the time of enrolment
- •VWD type 1 (baseline von Willebrand factor activity [VWF:RCo], <30 IU/dL), 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding
- •Currently receiving frequent on-demand treatment with a VWF-containing product
- •In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation
- •Voluntarily given, fully informed written and signed consent obtained before collection of any patient data
排除标准
- •Patients who meet any of the following criteria are not eligible for the study:
- •Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds
- •Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months
- •History, or current suspicion, of VWF or FVIII inhibitors
- •Medical history of a thromboembolic event within 6 months before enrolment
- •Severe liver or kidney diseases as described in the medical records
- •Female patients with an existing or suspected pregnancy or who are breast-feeding at the time of enrolment
- •Change in hormonal contraception within 6 months before enrolment
- •Cervical or uterine conditions causing abnormal uterine bleeding (including infection or dysplasia)
- •Other coagulation disorders or bleeding disorders due to anatomical reasons
- •Participation in an interventional clinical study during the 6-month of study period
- •Inability to complete the patient diary to reliably evaluate the type, frequency, and treatment of BEs during the 6-month study period
研究组 & 干预措施
Patients undergoing treatment with a VWF-containing product
Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months
干预措施: Von Willebrand Factor-Containing Product (Drug)
结局指标
主要结局
Total Annualized Bleeding Rate (TABR)
时间窗: Screening through study completion (6 months)
The total annualized bleeding rate (TABR) will be calculated as the total number of spontaneous bleeds, traumatic BEs, and other BEs occurring in the time period between the start of data collection for each patient and the Study Completion Visit, divided by the duration (in years) between the start of data collection and the Study Completion Visit. Surgery periods, and BEs occurring within these surgery periods, will be excluded from the calculation of TABR.
次要结局
- Spontaneous Annualized Bleeding Rate (SABR)(Screening through study completion (6 months))
- Consumption of the VWF-containing Product(Screening through study completion (6 months))
- Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10)(At screening)
- Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale(Screening through study completion (6 months))
- Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)(At screening visit)
- Number of Surgery With Successful/Unsuccessful Efficacy Assessment(From start of surgery until end of post-operative period (within 8 days after surgery))
- Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)(At screening visit)
- Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)(At screening)
- Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score(Screening through study completion (6 months))
- Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate(Screening through study completion (6 months))