Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Octapharma
- 入组人数
- 200
- 试验地点
- 100
- 主要终点
- Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment
研究概览
简要总结
International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.
详细描述
Octapharma's FVIII concentrates have been tested in clinical trials and registered for treatment of haemophilia A; however, as haemophilia A is a rare disease, the numbers of patients treated in studies so far are limited. For previously untreated patients (PUPs), who are typically young children, and for minimally treated patients (MTPs), who have been exposed to only minimal FVIII dosages, there is a general interest to increase the body of data on treatment effectiveness and safety, particularly related to inhibitor development. Also, specifically for PUPs, treatment algorithms are not standardized, e.g. with respect to utilisation, dosage, frequency or optimal start age of FVIII prophylaxis. Real world evidence derived from a non-interventional study (NIS) can describe product utilisation and demonstrate value over a product's life cycle and facilitate benefit-risk assessments. The purpose of this study is thus to evaluate product utilisation, effectiveness and safety, including inhibitor development information, in severe haemophilia A PUPs and MTPs, who have been prescribed Octapharma's FVIII concentrates.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients of any age and ethnicity
- •Severe haemophilia A (FVIII:C<1%)
- •Decision to prescribe Octapharma's FVIII concentrate before enrollment into the study
- •No previous treatment with FVIII concentrates or other blood products containing FVIII (PUPs) OR
- •Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood products containing FVIII (MTPs), if
- •data are available on all previous treatment, AND
- •they did not develop an inhibitor at any time point, OR
- •they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate (in the presence or absence of emicizumab).
- •Voluntarily given, fully informed written and signed consent obtained before any study-related data documentation is conducted (obtained from the patient's parent/legal guardian)
排除标准
- •Diagnosis with a coagulation disorder other than haemophilia A
- •Concomitant treatment with any systemic immunosuppressive drug
- •Participation in an interventional clinical trial during the time period evaluated
- •Participation in another non-interventional study of Octapharma
结局指标
主要结局
Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment
时间窗: 100 exposure days
Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs
Incidence of Adverse Drug Reactions (ADRs)
时间窗: 100 exposure days
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.
次要结局
- Dosage of FVIII concentrates(100 exposure days)
- Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians(100 exposure days)