Prospective, Multinational, Non-interventional Post-authorisation Study to Document the Long-term Immunogenicity, Safety, and Efficacy of Human-cl rhFVIII (Simoctocog Alfa) in Patients With Haemophilia A Treated in Routine Clinical Practice
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 80
- 试验地点
- 39
- 主要终点
- Number of Patients With FVIII Inhibitors
研究概览
简要总结
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Haemophilia A (FVIII:C ≤ 2%) based on medical history; at least 100 patients should have severe haemophilia A (FVIII:C < 1%)
- •Male patients of any age
- •Previous treatment with a FVIII concentrate for more than 150 EDs
- •Availability of detailed documentation (patient diary, log book, etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e., prophylaxis, on-demand, recent surgery, or immune tolerance induction)
- •Inhibitor negative (< 0.6 BU) at study entry as confirmed by a recovery test with previous FVIII product and inhibitor test in a central laboratory
- •Immunocompetence (CD4+ count > 200/µL), HIV-negative, or having a viral load < 200 particles/µL or < 400,000 copies/mL
- •Decision to prescribe Human-cl rhFVIII before enrolment into the study
- •Written informed consent by the patient or the patient's parent or legal guardian
排除标准
- •Patients treated with any investigational medicinal product (IMP) except FVIII IMP within 30 days prior to the Screening Visit or patients planning to undergo treatment with any IMP other than Human-cl rhFVIII are not eligible for enrolment into the study.
结局指标
主要结局
Number of Patients With FVIII Inhibitors
时间窗: Screening through to study completion (minimum 1.7 months; maximum 31.6 months)
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Number of Patients With Adverse Drug Reactions
时间窗: Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.
次要结局
- Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment(Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months))
- Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale(Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months))
- Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians(From start of surgery until end of post-operative period)
- Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale(Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months))