NCT03093480已完成4 期
A Non-controlled, Open-Label, Multicenter, Study of Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Subjects With Inhibitors Undergoing the First ITI Treatment
Bioverativ, a Sanofi company74 个研究点 分布在 10 个国家目标入组 16 人开始时间: 2017年12月8日最近更新:
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 16
- 试验地点
- 74
- 主要终点
- Time to Tolerization With rFVIIIFc
研究概览
简要总结
The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.
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
- Male
- 接受健康志愿者
- 否
入选标准
- •Ability of the participant or his legally authorized representative (e.g., parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local participant privacy regulations
- •Male participants of any age diagnosed with severe hemophilia A (as confirmed from the medical record)
- •Currently diagnosed with high titer inhibitors (historical peak greater than or equal to (>=) 5 Bethesda units per milliliter (BU/mL), according to medical records)
- •Previously treated with any plasma-derived or recombinant conventional or Extended Half-Life FVIII
排除标准
- •Other coagulation disorder(s) in addition to hemophilia A
- •Previous immune tolerance induction (ITI)
- •History of hypersensitivity or anaphylaxis associated with any factor VIII (FVIII) administration
- •Planned major surgery scheduled during the study unless deferred until after study completion (minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed)
- •Abnormal renal function (serum creatinine >1.5 milligram per deciliter (mg/dL) or 2 × upper limit of normal (ULN) for participant age based on local laboratory range) as assessed by local laboratory
- •Serum alanine aminotransferase or aspartate aminotransferase > 5 × upper limit of normal (ULN) as assessed by local laboratory
结局指标
主要结局
Time to Tolerization With rFVIIIFc
时间窗: Up to 48 Weeks
Time required for participants to achieve immune tolerance induction (ITI) success where ITI success is defined as achieving all 3 of the following criteria: confirmed negative titers consisting of 2 consecutive negative inhibitor assessments within 2 weeks (less than \[\<\] 0.6 Bethesda units/milliliter \[mL\] by the Nijmegen-modified Bethesda assay); incremental recovery (IR) greater than or equal to (\>=) 66 percent (%) of the expected IR in 2 consecutive assessments; half-life (t½) \>= 7 hours.
次要结局
- Annualized rFVIIIFc Consumption for Overall Study Period(Up to 2 Years)
- Annualized Bleeding Rates During ITI Period(Up to 48 weeks)
- Number of Participants With Immune Tolerance Induction (ITI) Success(Up to 48 Weeks)
- Number of Participants Who Experienced Relapse(Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period))
- Average Number of Days Missed From Work or School Per Month During ITI Period(Up to 48 weeks)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (TESAEs) as a Measure of Safety and Tolerability(Up to 2 Years)
- Average Number of Days Missed From Work or School Per Month After ITI Period(Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period))
- Annualized Number of Hospitalization Days During ITI Period(Up to 48 weeks)
- Annualized Number of Hospitalization Days After ITI Period(Up to 48 weeks (16 weeks Tapering period & 32 weeks Follow-up period))
- Annualized Bleeding Rates After ITI Period(Up to 48 weeks (16 weeks Tapering period and 32 weeks follow-up period))
- Adherence to Treatment Regimen Overall Study Period(Up to 2 Years)
研究者
研究点 (74)
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