NCT02954575已完成3 期
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 57
- 试验地点
- 7
- 主要终点
- Total Annualized Bleeding Rate (TABR)
研究概览
简要总结
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Severe hemophilia A (<1% FVIII:C) according to medical history
- •Male patients aged ≥12 years
- •Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
- •Immunocompetence (CD4+ count >200/µL)
- •Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
- •Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted
- •Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).
排除标准
- •Any coagulation disorders other than hemophilia A
- •History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
- •Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
- •Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
- •Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
研究组 & 干预措施
All patients
Experimental
All patients will receive 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: 6 months
The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
次要结局
- Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C(Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection)
- Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)(6 months)
- Immunogenicity of Wilate by Testing for FVIII Inhibitors(6 months)
- Efficacy of Wilate in the Treatment of Breakthrough BEs(6 months)
- Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis(6 months)
- Incremental in Vivo Recovery (IVR) of Wilate Over Time(Baseline, 3 and 6 months)
- Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate(6 months)
- Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C(Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection)
- Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study(6 months)
- Spontaneous Annualized Bleeding Rate (SABR)(6 months)
- Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C(Initial PK assessment (Day -1) and 6 months)
- Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study(6 months)
研究者
研究点 (7)
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