A Phase II, Multicentre, Open-label Study to Evaluate the Pharmacokinetic, Safety and Preliminary Efficacy of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Subjects With Severe Hemophilia A
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 5
- 主要终点
- Time required for the concentration of the drug to reach half of its original value (T1/2)
研究概览
简要总结
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With 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
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 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%.
- •Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry
- •Normal prothrombin time or INR < 1.3
- •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.• Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), Urea /BUN > 2×ULN, Cr > 176.8 µmol/L)
- •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 screening
- •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
- •Other protocol-defined inclusion/exclusion Criteria May Apply.
研究组 & 干预措施
Arm 1 prophylaxis treatment
Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively.
Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively.
All subjects are being received PK assessment in ED1 and ED4.
干预措施: FRSW117 (Drug)
结局指标
主要结局
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 10 days
Measured by One-Stage Clotting Assay
Maximum measured concentration of FVIII:C (Cmax)
时间窗: Pre-dose and post dose up to 10 days
Measured by One-Stage Clotting Assay
Area Under the Curve to Infinity (AUC)
时间窗: Pre-dose and post dose up to 10 days
Measured by One-Stage Clotting Assay
Evaluation of the level of anti-PEG antibody production in participants
时间窗: Pre-dose and post dose up to 36 days
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
时间窗: Pre-dose and post dose up to 36 days
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 10 days
Measured by One-Stage Clotting Assay
次要结局
- score of bleeding symptoms and Vital signs(Pre-dose and post dose up to 36 days)
- Number of participants with treatment-related adverse events as assessed by CTCAE V5.0(Pre-dose and post dose up to 36 days)
- Development of Inhibitor(Pre-dose and post dose up to 36 days)