An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 108
- 试验地点
- 72
- 主要终点
- Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
研究概览
简要总结
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A. The secondary objectives were to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in PUPs, to evaluate rFVIIIFc consumption for the prevention and treatment of bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune tolerance induction (ITI) in participants with inhibitors.
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
- — 至 5 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Ability of the participant's legally authorized representative (e.g. their parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
- •Weight >=3.5 kg at the time of screening.
- •Severe hemophilia A defined as less than (<) 1 IU/dL (<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period.
排除标准
- •Any exposure to blood components, factor VIII replacement products, including commercially available rFVIIIFc at any time prior to or during screening.
- •History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection).
- •History of hypersensitivity reactions associated with any rFVIIIFc administration.
- •Other coagulation disorder(s) in addition to hemophilia A.
- •Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment.
- •Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.
- •Note: Other protocol defined Inclusion/Exclusion criteria may apply.
结局指标
主要结局
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
时间窗: Up to 3 years
A positive/confirmed inhibitor result occurs when a participant has a value \>=0.6 BU/mL by central laboratory testing using Nijmegen-modified Bethesda assay, that is confirmed on re-testing of a separate sample collected \>=2 weeks after the initial sample. Confirmed inhibitor development was based on all participants who had reached \>=10 EDs and had \>=1 inhibitor test performed at or beyond this milestone or who had an inhibitor. Exposure day (ED) is a 24-hour period in which participant received \>=1 dose of rFVIIIFc injections. Participants who did not develop an inhibitor but reached the milestone number of EDs were included in the denominator during calculation of percentage. Additionally, any participant who developed an inhibitor following the initial rFVIIIFc administration was included in the numerator and denominator during calculation of percentage.
次要结局
- Total Number of Exposure Days (EDs)(Up to 3 years)
- Number of rFVIIIFc Injections With Excellent or Good, Moderate or None Treatment Response Assessed Using a 4-Point Scale(Up to 3 years)
- Number of Participants With Response to Immune Tolerance Induction (ITI)(Up to 3 years)
- Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])(Up to 3 years)
- Annualized Number of Spontaneous Joint Bleeding Episodes(Up to 3 years)
- Number of Injections of rFVIIIFc Required to Resolve a Bleeding Episode(Up to 3 years)
- Average Dose Per Injection of rFVIIIFc Required to Resolve a Bleeding Episode(Up to 3 years)
- Total Annualized rFVIIIFc Consumption Per Participant for the Prevention and Treatment of Bleeding Episodes(Up to 3 years)
- Change From Baseline in rFVIIIFc Incremental Recovery (IR)(Baseline, Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108 and 120)