Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients

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追踪信息

首次提交日期 ^ICMJE

October 3, 2018

首次发布日期e ^ICMJE

October 4, 2018

最后更新发布日期

October 4, 2018

预计研究开始日期 ^ICMJE

February 13, 2018

预计主要完成日期

June 2022 (主要结果测量的最终数据收集日期)

目前主要观察指标 ^ICMJE

Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment[ Time Frame: 100 exposure days ]

Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs

Incidence of Adverse Drug Reactions (ADRs)[ Time Frame: 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.

原始主要观察测量 ^ICMJE

与当前相同

目前的二级观察 ^ICMJE

Dosage of FVIII concentrates[ Time Frame: 100 exposure days ]
For each individual FVIII injection the dose will be recorded.
Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians[ Time Frame: 100 exposure days ]
At the end of the postoperative period, treating physicians will assess the effectiveness of surgical prophylaxis using a scale including the four items: 'excellent,' 'good,' moderate,' and 'none'.

描述性信息

简略标题 ^ICMJE

Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients

正式标题 ^ICMJE

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

简要概况

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.

详细说明

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.

研究类型 ^ICMJE

Observational

研究阶段

研究设计 ^ICMJE

分配:
干预模型:
干预模型描述:
盲法: Observational
盲法描述:
主要目的:

适用条件 ^ICMJE

干预项目 ^ICMJE

研究工具

: Nuwiq
All patients receiving Nuwiq (recombinant FVIII)
: Octanate
All patients receiving Octanate (plasma derived FVIII)
: Wilate
All patients receiving Wilate (plasma derived FVIII/von Willebrand factor [VWF])

招募信息

招募状态 ^ICMJE

Recruiting

预计入组 ^ICMJE

140

原始预计入组 ^ICMJE

与当前相同

预计研究完成日期

June 2022

预计主要完成日期

June 2022 (主要结果测量的最终数据收集日期)

合格标准 ^ICMJE

Inclusion Criteria: - 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 - Either - 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 - their first ED occurred after 1st January 2015, 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 - 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) Exclusion Criteria: - 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

性别

参与研究的性别:

All

年龄

最小年龄：N/A ，最大年龄：N/A

接受健康的志愿者

没有

可入组国家 ^ICMJE

管理信息

数据检测委员会

研究涉及美国FDA监管的产品

研究美国FDA监管的药品: No
研究涉及美国FDA监管的设备产品: No

IPD 共享声明

计划分享 IPD:

责任方

，

研究赞助商 ^ICMJE

Octapharma

合作者 ^ICMJE

研究员 ^ICMJE

Study Director: Sigurd Knaub, PhD Octapharma

PRS 账户

验证日期

October 2018

^ICMJE 国际医学期刊编辑委员会和世界卫生组织 ICTRP 要求的元素

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