A Phase I, Multicentre, Open-label, Self-control Study to Evaluate the Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 12
- 试验地点
- 5
- 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 at two dose levels in patients with hemophilia A.
Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in patients with hemophilia A.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 12 Years 至 60 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •12 years to 60 years, male.
- •The activity of the coagulation factor VIII (FVIII:C) < 2%, and previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
- •Non-immune deficiency (CD4 > 200/μL).
- •Non-acute hemorrhagic state.
- •No history of a positive inhibitor test (< 0.6 BU) or clinical signs of decreased response to FVIII administrations. No Family history of inhibitors.
- •Platelet count > 100,000 platelets/μL.
- •Normal prothrombin time or INR < 1.
- •Normal thrombin time (TT).
- •Normal previous results of vWF antigen examination.
- •Negative lupus anticoagulant .
- •Capable of understanding and willing to comply with the conditions of the protocol have read (patient and/or guardian).
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 IgG2 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 4 days prior screening or within 72 h prior to administration.
- •Patients with severe heart disease, including myocardial infarction, heart failure (III or higher level).
- •Clinically significant of other systematic diseases: alcoholism, drug abuse, mental disorders and mental retardation.
- •Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 3 × upper limit of normal (ULN) , BUN > 2×ULN, Cr > 2.0 mg/dL).
- •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 1 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.
研究组 & 干预措施
Arm 1
Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a low dose.
干预措施: ADVATE (Drug)
Arm 1
Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a low dose.
干预措施: FRSW107 (Drug)
Arm 2
Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a high dose.
干预措施: ADVATE (Drug)
Arm 2
Participants will receive a single intravenous (i.v.) injection of ADVATE followed by a single intravenous (i.v.) injection of Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection (FRSW107) at a high dose.
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 of FRSW107 up to 10 days.
Measured by the One-stage 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 of FRSW107 up to 10 days.
Measured by the One-stage aPTT Clotting Assay.
Area Under the Curve to Infinity (AUC).
时间窗: Pre-dose and post dose of FRSW107 up to 10 days.
Measured by the One-stage 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 of FRSW107 up to 10 days.
Measured by the One-stage aPTT Clotting Assay.
次要结局
- Development of Inhibitor.(Pre-dose and post dose of FRSW107 up to 28 days.)
- Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.(Post dose of FRSW107 up to 28.)