Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 66
- 试验地点
- 20
- 主要终点
- Annualized Number of Bleeding Episodes (BE) in Phase II
研究概览
简要总结
To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.
详细描述
There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Severe haemophilia A (FVIII:C < 1%) according to medical history.
- •Male patients ≥ 18 years old.
- •Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).
- •Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.
- •Immunocompetence (CD4+ count > 200/microliter).
- •HIV-negative, if positive, viral load < 200 particles/microliter or < 400,000 copies/mL.
- •Freely given written informed consent
排除标准
- •Any coagulation disorder other than haemophilia A.
- •Present or past FVIII inhibitor activity (> 0.6 Bethesda Unit [BU])
- •Severe liver or kidney disease.
研究组 & 干预措施
Human-cl rhFVIII
Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.
干预措施: Human-cl rhFVIII (Biological)
结局指标
主要结局
Annualized Number of Bleeding Episodes (BE) in Phase II
时间窗: Beginning to the end of Phase II (6 months)
The annualized number of total BEs was calculated for each participant as follows: d\*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196).
次要结局
- Dosage Per Week in Phase II(Beginning to the end of Phase II (6 months))
- Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week(Beginning to the end of Phase II (6 months))
- Median Dosing Interval During Individually Tailored Prophylaxis(Beginning to the end of Phase II (6 months))
- Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II(Beginning to the end of Phase II (6 months))