Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 发起方
- 入组人数
- 7
- 试验地点
- 4
- 主要终点
- Number of Participants That Did Not Develop Inhibitors to FVIII (<0.6BU)
研究概览
简要总结
Primary objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject.
Secondary objectives: To assess efficacy and tolerability by monitoring FVIII recovery and adverse events
详细描述
The primary efficacy endpoint is to assess immunogenicity of Optivate® by monitoring plasma inhibitor level for at least 100 EDs for each subject.
FVIII inhibitor evaluation FVIII inhibitor screen data will be listed. FVIII quantitative inhibitor results will be listed. Shift tables will present the number of subjects with positive (≥ 0.6 BU) and negative (< 0.6 BU) results and those for whom the results change during the study. The number of exposure days until development of inhibitors will be summarised.
For the secondary endpoints: Descriptive statistics will be presented on the number of recoveries at each timepoint and for each subject. These will be presented for each visit and for each subject and then for each batch of FVIII/ Optivate® used. All the AE data (from CRF and study diary) will be pooled together and reported in terms of the type, duration, treatment and/or severity.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent or, if less than 18 years of age written assent (where possible) and their parent/guardian's written informed consent.
- •Severe haemophilia A (< 1%# FVIII:C).
- •Previously Treated Patients (PTPs) with > 150 exposure days on prior Factor VIII therapy (of which at least the last 50 EDs or 2 years treatment can be confirmed by way of subject records).
- •Immunocompetent with CD4 count > 200 / µl.
- •HIV negative or a viral load < 200 particles / µl.
- •subjects suffering from severe haemophilia A (<2%) may be enrolled, but only after approval by BPL. Subjects with a Factor VIII of <2% may not constitute more than 50% of the total patient population. A separate statistical evaluation will be conducted for the <1% and <2% populations.
排除标准
- •• History of inhibitor development to FVIII or a positive result on the Nijmegen Bethesda at screening (quantitative result of > 0.6 BU) prior to the administration of Optivate®.
- •Known or suspected hypersensitivity to the investigational medicinal product or its excipients.
- •Clinically significant liver disease, renal disease, or coagulopathy other than haemophilia A.
- •History of unreliability or non cooperation (including not being able to complete the study diary).
- •Participating in, or have taken part in another trial within the last 30 days.
研究组 & 干预措施
Optivate 500IU
Optivate 500IU
干预措施: Optivate 500IU (Biological)
结局指标
主要结局
Number of Participants That Did Not Develop Inhibitors to FVIII (<0.6BU)
时间窗: At least 100 Exposure Days for each subject. Subjects will attend 5 visits over a period of up to 12 months
FVIII inhibitor status at any of the study visits was measured by a Nijmegen Bethesda assay and inhibitor screens. A result of ≥ 0.6 BU confirmed that the subject had developed inhibitors to FVIII. If this occurred, the test was repeated on a separate sample; if both tests were confirmed to be ≥ 0.6 BU, this was to be reported by the Investigator as a serious adverse event (SAE).
次要结局
- Optivate® Therapy to Treat Breakthrough Bleeds Per Subject Per Year in the Protocol Population.(Over a period of 12 months)
- Treatment Emergent Adverse Events (Serious) in Safety Population(Over a period of 12 months)
- Recovery With Prior FVIII Concentrate (Screening Visit) Versus Recovery With First Dose With Optivate® (Visit 1) for the Protocol Population.(Screening and Visit 1 (up to 4 weeks))
- Overall Consumption of Optivate®: Number of Exposure Days for Each Subject Per Year/Subject in the Per Protocol Population.(Over a period of 12 months)
- Treatment Emergent Adverse Events (Non-serious) in the Safety Population(Over a period of 12 months)
- Optivate® Recovery Across Visits 1 to 4 for the Protocol Population.(Visits 1 to 4 (Up to 100 Optivate exposure days))
- Overall Consumption of Optivate®: Total Dose in IU/kg of Optivate® Per Subject for Prophylactic Use.(Over a period of 12 months)
- Overall Consumption of Optivate®: Total Number of Infusions for Prophylactic Use Per Subject in the Protocol Population.(Over a period of 12 months)
- Overall Consumption of Optivate®: Overall Mean Dose in IU/kg of Optivate® Per Subject/Year for Prophylactic Use in the Protocol Population.(Over a period of 12 months)
- Number of Participants With Inhibitor Development in Safety Population (Measured by ≥0.6 Bethesda Units)(Over a period of 12 months)
- Overall Consumption of Optivate®: Total Dose in IU/kg of Optivate® Per Subject to Treat a Bleed in the Protocol Population.(Over a period of 12 months)
- Overall Consumption of Optivate®: Total Number of Infusions to Treat a Bleed Per Subject in the Protocol Population.(Over a period of 12 months)