Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 43
- 试验地点
- 14
- 主要终点
- Total Annualized Bleeding Rate (TABR)
研究概览
简要总结
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.
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
- 6 Years 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:
- •Aged ≥6 years at the time of screening
- •VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (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 on-demand treatment with a VWF-containing product with at least 1, and an average of ≥2, documented spontaneous BEs per month in the last 6 months, with at least 2 of these BEs requiring treatment with a VWF-containing product
- •Availability of records to reliably evaluate type, frequency, and treatment of BEs for at least 6 months of on-demand treatment before screening
- •Female patients of child-bearing potential must have a negative urine pregnancy test at screening and agree to use adequate birth control measures; in case hormonal contra-ception is used, the medication class should remain unchanged for the duration of the study
- •All patients to provide voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted
排除标准
- •Patients who meet any of the following criteria are not eligible for the study:
- •Having received on-demand or prophylactic treatment with a VWF-containing product but having no records available to reliably evaluate the type, frequency, and treatment of BEs over a period of at least 6 months of on-demand treatment
- •History, or current suspicion, of VWF or FVIII inhibitors
- •Medical history of a thromboembolic event within 1 year before enrolment
- •Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)
- •Platelet count <100,000/µL at screening (except for VWD type 2B)
- •Body weight <20 kg at screening
- •Patients receiving, or scheduled to receive, immunosuppressant drugs (other than an-tiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
- •Pregnant or breast-feeding at the time of enrolment
- •Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia)
- •Treatment with any IMP in another interventional clinical study currently or within 4 weeks before enrolment
- •Other coagulation disorders or bleeding disorders due to anatomical reasons
- •Known hypersensitivity to any of the components of the study drug
研究组 & 干预措施
All patients
Patients with type 3, type 2 (except 2N), or severe type 1 VWD aged ≥6 years at screening receiving Wilate for prophylactic treatment.
干预措施: Wilate (Drug)
结局指标
主要结局
Total Annualized Bleeding Rate (TABR)
时间窗: 12 months
The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds (except menstrual bleeds) occurring in the time period between first dose of the investigational medicinal product (IMP) and the Study Completion Visit, divided by the duration (in years) between first dose of IMP and the Study Completion Visit.
Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29
时间窗: 12 Months
Estimated TABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
次要结局
- Spontaneous Annualized Bleeding Rate (SABR)(12 months)
- Wilate Consumption for Prophylaxis (mFAS Population)(12 months)
- Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo)(From baseline and 12-month visit)
- Incremental In Vivo Recovery (IVR) of FVIII(Baseline and 12-month visit)
- Wilate Exposure for Prophylaxis (mFAS Population)(12 months)
- Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29.(12 Months)
- Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)(12 months)