NCT00486278已完成2 期
A Multi-centre, Randomised, Double-blinded, Controlled, Dose-escalation Trial on Safety and Efficacy of Activated Recombinant FVII Analogue (NN1731) in the Treatment of Joint Bleeds in Congenital Haemophilia Patients With Inhibitors
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 1
- 主要终点
- Number of Adverse Events (AEs)
研究概览
简要总结
This trial is conducted in Africa, Asia, Europe, Japan, and North and South America.
The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated)) in haemophilia patients with inhibitors.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •12 years of age or older (at least 18 years in Croatia, France and United Kingdom (UK))
- •Clinical diagnosis of congenital haemophilia A or B with a current positive inhibitor titre and a known peak inhibitor of above 5 Bethesda units (BU) (present or in the past) to human FVIII or IX and known antihuman FVIII or IX anamnestic response
- •Minimum of 2 joint bleeds (haemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 6 months, or at least 4 joint bleeds (hemarthroses of ankles, knees, or elbows) requiring haemostatic drug treatment within the previous 12 months at trial entry
排除标准
- •Known allergy to rFVIIa, and/or suspected allergy to trial product
- •Platelet count lower than 50,000 mm^3 based on medical records at trial entry (visit 1)
- •Any clinical signs or history of thromboembolic events
- •Advanced atherosclerotic disease
- •Severe liver disease based on medical records within the past 12 months at trial entry (Visit 1), as defined by alanine aminotransferase (ALAT) above 3 times the upper limit of normal reference range
- •Known active pseudo tumours (documented bleeding requiring treatment within the last 3 months
- •Subject had any (major) surgical procedure in the 30 days prior to screening into the trial. a. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject
研究组 & 干预措施
vatreptacog alfa 5 mcg/kg
Experimental
干预措施: vatreptacog alfa (activated) (Drug)
vatreptacog alfa 10 mcg/kg
Experimental
干预措施: vatreptacog alfa (activated) (Drug)
vatreptacog alfa 20 mcg/kg
Experimental
干预措施: vatreptacog alfa (activated) (Drug)
vatreptacog alfa 40 mcg/kg
Experimental
干预措施: vatreptacog alfa (activated) (Drug)
vatreptacog alfa 80 mcg/kg
Experimental
干预措施: vatreptacog alfa (activated) (Drug)
rFVIIa 90 mcg/kg
Experimental
干预措施: eptacog alfa (activated) (Drug)
结局指标
主要结局
Number of Adverse Events (AEs)
时间窗: Monitoring of adverse events was performed from start of the trial to approximately 4 weeks after administration of trial product.
Adverse event is defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
次要结局
- F1 + 2 (Prothrombin Fragments 1+2)(pre-dose - 12 hours after trial product administration)
- Prothrombin Time (PT)(pre-dose - 12 hours after trial product administration)
- Biochemistry: ALAT (Alanine Aminotransferase)(screening visit, pre-dose and 12 hours after dosing)
- Activated Recombinant Human Factor VII Analogue Activity in the Blood(0-24 hours after trial product administration)
- Number of Subjects With Need for Additional Haemostatic Agents(within 24 hours after successful control of bleeding episode with trial product)
- Pharmacokinetic Parameters Based on FVIIa Activity: AUC 0-t (Area Under the Plasma FVIIa Activity-time Curve From Time Zero to the Time (t) )(0-24 hours after trial product administration)
- Activated Partial Thromboplastin Time (aPTT)(pre-dose - 12 hours after trial product administration)
- Cessation of Bleeding: Number of Doses Needed to Control Bleeding(Within 9 hours after first trial product administration or need of additional haemostatic medication within 9 hours after first trial administration additional haemostatic agents required to control bleed (treatment failure))
- Pharmacokinetic Parameters Based on FVIIa Activity: AUC(0-inf) (Area Under the Plasma FVIIa Activity-time Curve From Time Zero to Infinity)(0-24 hours after trial product administration)
- Pharmacokinetic Parameters Based on FVIIa Activity: MRT (Mean Residence Time)(0-24 hours after trial product administration)
- Pharmacokinetic Parameters Based on FVIIa Activity: t½ (Terminal Half-life)(0-24 hours after trial product administration)
- Pharmacokinetic Parameters Based on FVIIa Activity: CL (Total Clearance)(0-24 hours after trial product administration)
- Pharmakokinetic Parameters Based on FVIIa Activity: Vss (Distribution Volume at Steady State)(0-24 hours after trial product administration)
- Haematology: Packed Cell Volume(screening visit, pre-dose and 12 hours after dosing)
- Immunogenicity (Inhibitor Development)(Monitoring of adverse events was performed from start of the trial to approximately 4 weeks after administration of trial product.)
- Biochemistry: Creatinine(screening visit, pre-dose and 12 hours after dosing)
- Haematology: Haemoglobin(screening visit, pre-dose and 12 hours after dosing)
- Haematology: Platelet Count(screening visit, pre-dose and 12 hours after dosing)
- Haematology: Red Cell Count(screening visit, pre-dose and 12 hours after dosing)
- Haematology: White Cell Count(screening visit, pre-dose and 12 hours after dosing)
研究者
研究点 (1)
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