NCT02137850已完成3 期
An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia 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
- 入组人数
- 124
- 试验地点
- 95
- 主要终点
- Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
研究概览
简要总结
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
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
- 0 Years 至 6 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
- •Male, age below 6 years of age at the time of signing informed consent
- •Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results
- •No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable)
排除标准
- •Any history of FVIII inhibitor (defined by medical records) - Known or suspected hypersensitivity to trial product or related products
- •Previous participation in this trial. Participation is defined as first dose administered of trial product
- •Receipt of any investigational medicinal product within 30 days before screening
- •Congenital or acquired coagulation disorder other than haemophilia A
- •Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
- •Patient's parent(s')/legally acceptable representative (LAR(s')) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
研究组 & 干预措施
50 EDs (exposure days)
Experimental
干预措施: turoctocog alfa pegol (Drug)
结局指标
主要结局
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
时间窗: From start of the treatment up to 7 years
Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.
次要结局
- Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest(From start of the treatment up to 8.9 years)
- Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)(From start of the treatment up to 8.9 years)
- Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)(From start of the treatment up to 8.9 years)
- Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None")(From start of the treatment up to 8.9 years)
- Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))(From start of the treatment up to 8.9 years)
- Consumption of N8-GP for Prophylaxis (Number of Injections)(From start of the treatment up to 8.9 years)
- Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed))(From start of the treatment up to 8.9 years)
- Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections)(From start of the treatment up to 8.9 years)
- Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value(From start of the treatment up to 8.9 years)
- Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other")(From start of the treatment up to 8.9 years)
研究者
研究点 (95)
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