A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Number of Treatment Emergent (Serious and Non-serious) Adverse Events
研究概览
简要总结
This trial will be conducted in Asia, Europe and the United States of America (USA).
The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.
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
- 1 Year 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed participation in trial F13CD-3760 (NCT01230021)
排除标准
- •Known or suspected hypersensitivity to trial product or related products
- •Known history of development of inhibitors against FXIII (factor XIII)
- •Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
- •Platelet count (thrombocytes) less than 50X10e9 / L
- •Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
- •Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
- •Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction
研究组 & 干预措施
rFXIII 35 IU/kg
干预措施: catridecacog (Drug)
结局指标
主要结局
Number of Treatment Emergent (Serious and Non-serious) Adverse Events
时间窗: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.
次要结局
- Clinical Laboratory Assessments: Biochemistry: Urea(Every 6th month, week 24 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)(Every 6th month, from week 24 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Haematology: Haemoglobin(Every 6th month, from week 0 to end of trial visit (week 173).)
- Vital Signs: Pulse(Week 0 to end of trial visit (week 173).)
- Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.(Week 0 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Biochemistry: Creatinine(Every 6th month, from week 24 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)(Every 6th month, from week 24 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Haematology: Thrombocytes(Every 6th month, from week 0 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Haematology: Erythrocytes(Every 6th month, from week 0 to end of trial visit (week 173).)
- Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.(Weeks 0 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Haematology: Leucocytes(Every 6th month, from week 0 to end of trial visit (week 173).)
- Clinical Laboratory Assessments: Haematology: Haematocrit(Every 6th month, from week 0 to end of trial visit (week 173).)
- Physical Examinations(Week 0 to end of trial visit (week 173).)
- Vital Signs: Systolic BP (Blood Pressure)(Week 0 to end of trial visit (week 173).)
- Vital Signs: Diastolic BP (Blood Pressure)(Week 0 to end of trial visit (week 173).)