NCT01445197终止3 期
A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to Evaluate Plasma-Derived Antihaemophilic Factor/Von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects With Haemophilia A (≤ 2%) Who Have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors)
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- CSL Behring
- 入组人数
- 1
- 试验地点
- 7
- 主要终点
- Response to immune tolerance induction (ITI) treatment
研究概览
简要总结
This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tolerance therapy.
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
- 28 Days 至 11 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects diagnosed with haemophilia A (≤ 2% FVIII level in the absence of factor replacement, according to their medical history).
- •Age 28 days to <12 years.
- •Subject is eligible for immune tolerance induction (ITI) therapy
排除标准
- •The subject has received ITI previously.
- •Subjects with a historical peak inhibitor titre of ≥ 200 BU/mL.
- •Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons.
- •High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator.
- •Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined at screening).
研究组 & 干预措施
Biostate
Experimental
干预措施: Biostate (Biological)
结局指标
主要结局
Response to immune tolerance induction (ITI) treatment
时间窗: 30 months
Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.
次要结局
- Time to complete response (success)(Up to 65 months)
- Number of bleeding events per patient(Up to 65 months)
- FVIII inhibitor titre(Up to 65 months)
- Time to inhibitor titer <0.6 BU/mL for the first time(Up to 65 months)
- Severity of bleeding events per patient(Up to 65 months)
- Catheter-related complications(Up to 65 months)
- Thromboembolic complications(Up to 65 months)
- Frequency of bleeding events(Up to 65 months)
研究者
研究点 (7)
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