A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 6
- 主要终点
- Maximum measured concentration of FVIII:C (Cmax)
研究概览
简要总结
Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.
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 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •The activity of the coagulation factor VIII (FVIII:C) < 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- •Normal prothrombin time or INR < 1.
- •Negative lupus anticoagulant.
排除标准
- •Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).
- •History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.
- •Current FVIII inhibitor-positive or history of FVIII inhibitor-positive.
- •Other coagulation disorder(s) in addition to hemophilia A.
- •Infusion of any products containing FVIII within 72 h prior to administration.
- •Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), BUN > 2×ULN, Cr > 2.0 ULN).
- •One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody.
- •Patients who received any anticoagulant or antiplatelet therapy within one week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials.
- •Patients having major surgery or receiving blood or bood components transfusion within 4 weeks prior screening or having planned major surgery schedule during the study.
- •Patients who previously participated in the other clinical trials within one month prior to administration.
- •Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation.
- •Patient who is considered by the other investigators not suitable for clinical study.
研究组 & 干预措施
Arm 1
Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period.
干预措施: ADVATE (Drug)
Arm 1
Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period.
干预措施: FRSW107 (Drug)
结局指标
主要结局
Maximum measured concentration of FVIII:C (Cmax)
时间窗: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
Time required for the concentration of the drug to reach half of its original value (T1/2)
时间窗: Pre-dose and post dose up to 8 days
Measured by aPTT Clotting Assay.
Area Under the Curve to Infinity (AUC)
时间窗: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL).
时间窗: Pre-dose and post dose up to 8 days.
Measured by aPTT Clotting Assay.
次要结局
- Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.(Post dose up to 32 days.)
- Development of Inhibitor(Pre-dose and post dose up to 32 days.)