NCT07684898进行中（未招募）3 期
A Single-Arm, Open-Label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, Immunogenicity and Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Therapy in Patients With Severe Hemophilia A (Adults and Adolescents)
Hangzhou Gensciences Biopharmaceutical Co., Ltd.38 个研究点 分布在 1 个国家目标入组 60 人开始时间: 2026年6月12日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 60
- 试验地点
- 38
- 主要终点
- ABR
研究概览
简要总结
The indication for this product is to control and prophylaxis in patients with Hemophilia A (congenital Factor VIII deficiency):
The Primary Objective： To evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated patients with severe Hemophilia A.
Secondary Objectives： To evaluate the health-related quality of life, pharmacokinetic (PK) profiles, safety and immunogenicity of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) for prophylactic treatment in previously treated subjects 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
- •1.12≤ age ≤65 year-old men; 2.Subjects with clinically confirmed severe hemophilia A, i.e. at screening (central laboratory testing) or previous medical records confirm: FⅧ activity < 1%; 3.Previous documented treatment with any recombinant and/or blood-derived coagulation factor Ⅷ products or cryoprecipitation products and dosed ≥150 exposure days (EDs≥150) ; 4.Normal prothrombin time (PT) or International Normalized Ratio (INR)<1.3; 5.Bleeding events were recorded in detail for at least 6 months prior to screening; 6.Fully understand and know about this study and sign informed consent to participate in the clinical study voluntarily, subject and/or their guardian can cooperate with them for bleeding treatment at home, and have the ability to complete all study procedures
排除标准
- •Known or suspected allergy to the investigational drug or its excipients, including mouse or hamster proteins;
- •Hypersensitivity or anaphylaxis after FⅧ or IgG2 injection in the past;
- •FⅧ inhibitor positive (≥0.6 BU/mL) during the screening period, or have a history of FⅧ inhibitor positive in the past, or a family history of FⅧ inhibitor positive;
- •Von Willebrand factor (vWF) antigen test results were lower than the lower limit of normal value;
- •Severe anemia at the screening stage (hemoglobin < 60 g/L);
- •Platelet count during screening period < 100×109 /L;
- •Abnormal liver function: Alanine aminotransferase (ALT), or aspartate aminotransferase (AST) >3 times upper limit of normal (ULN); or Serum total bilirubin (TBIL) >1.5x ULN;
- •Subjects with abnormal renal function: Creatinine clearance (Ccr) <50 ml/min (according to Cockcroft and Gault formula); or Serum creatinine (Cr) >1.5x ULN;
- •Subjects with active hepatitis C, that is, hepatitis C virus (HCV) antibody positive and HCV RNA positive; Or anti-treponema pallidum specific antibody (TPHA) positive; Or positive for antibodies against the human immunodeficiency virus (HIV);
- •Subjects with coagulation dysfunction other than hemophilia A;
- •Have a medical condition that may increase the risk of bleeding;
- •A history of drug or alcohol abuse;
- •Have a known mental disorder that may affect trial compliance;
- •Subjects who have received transfusions of blood or blood components within 4 weeks prior to screening;
- •Participants who had participated in other Interventional clinical trials within 1 month before screening;
- •Use of any anticoagulant or antiplatelet drugs, off-label maximum dose of non-steroidal anti-inflammatory drugs (NSAID) within 7 days prior to screening; Or subjects who need to be treated with anticoagulant or antiplatelet drugs or off-label maximum doses of SAID during clinical trials;
- •Severe cardiovascular and cerebrovascular disease or major thromboembolic events, such as stroke, myocardial infarction, unstable angina, congestive heart failure (New York Heart Association [NYHA] grade ≥ III), and severe arrhythmias (including QTc interphase > 480 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic ≥ 160 mmHg or diastolic ≥100 mmHg), deep vein thrombosis, etc.
- •Subjects who have received emicizumab within 6 months prior to the first administration of study drug, or have previously received fitusiran (siRNA, brand name: CEPHEIN®) or gene therapy;
- •Subjects who have received monoclonal antibody therapy, Fc fusion protein products, or intravenous immunoglobulin within 3 months prior to the first administration of study drug;
- •Subjects who have undergone major surgery within 3 months prior to the first administration of study drug, or those who plan to receive surgery during the study period;
- •Subjects who have received any standard half-life FⅧ preparations (e.g., Advate, Kovaltry, Octate, Recombinate, NovoEight, Anjiyin, etc.) within 3 days or 5 half-lives (whichever is longer) prior to the first administration of study drug; patients who have received any other extended half-life FⅧ preparations (e.g., Noxyte) within 4 days or 5 half-lives (whichever is longer) prior to the first administration of study drug;
- •Study patients with fever, severe active bacterial or viral infection, and allergies within 2 weeks before the first administration of the drug;
- •Systemic immunomodulators (such as glucocorticoids [> 10 mg/ day equivalent dose of prednisone], alpha-interferon, immunoglobulin, cyclophosphamide, cyclosporin, etc.) used within 14 days prior to the first administration of the study drug or planned during the study period were allowed to be inhaled, nasal spray, or topical corticosteroids;
- •Those who had been vaccinated within 4 weeks prior to initial administration of the study drug; Or who plan to be vaccinated during PK blood collection (only for subjects in the PK subgroup);
- •Plan to have a child or sperm donation during the entire trial period and within 3 months after the last dose, or do not want to use effective physical contraception (such as condoms, diaphragms, Iuds, etc.);
- •Have other serious medical conditions that the researchers said could not benefit from them
- •Subjects deemed unsuitable by other investigators.
研究组 & 干预措施
prophylactic treatment
Experimental
Subjects in PK Subgroup receive a single and multiple dose of 50 IU/kg FRSW107 at Visit 1 and Visit 5, respectively. PK samples will be collected up to 72 hours after the start of administration.After completion of PK blood sampling for the first dose and prior to availability of the corresponding PK data, subjects may continue prophylactic treatment with FRSW107 at a dose of 50 IU/kg every 3 days until their PK data are obtained.Once the first-dose PK data of a subject are available, individualized prophylactic treatment with FRSW107 will be implemented based on the PK results. On the premise of maintaining a trough FVIII activity level of ≥1%, the investigator will determine the appropriate individualized prophylactic regimen for the subject. The recommended prophylactic dosing interval is Q3D, with an optional dose range of 25-50 IU/kg.
For subjects in the non-PK subgroup, the investigator will select the initial prophylactic dose within the recommended range of 25-50 IU/kg.
干预措施: FRSW107 (Drug)
结局指标
主要结局
ABR
时间窗: 6 months
Annual rate of bleeding (ABR) during preventive treatment = Number of bleeding episode during the efficacy evaluation period/(number of treatment days /365.25)
次要结局
- Safety Evaluation(6 months)
- Immunogenicity Evaluation(6 months)
- Peak activity (Cmax)(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
- Effective rate of hemostatic treatment(6 months)
- Annualized rate of spontaneous bleeds and annualized rate of traumatic bleeds.(6 months)
- Annualized Joint Bleed Rate (AJBR)(6 months)
- Number of target joints.(6 months)
- Dosing parameters of prophylactic treatment(6 months)
- Factor VIII incremental recovery and trough levels during prophylactic treatment.(6 months)
- Time interval between each bleeding episode and the prior prophylactic dose during prophylaxis.(6 months)
- Dosing parameters for rescue hemostatic treatment of breakthrough bleeds during prophylaxis(6 months)
- Hemophilia Joint Health Score version 2.1 (HJHS 2.1)(6 months)
- EuroQol 5-Dimension 5-Level Questionnaire (EQ-5D-5L) and EuroQol Visual Analogue Scale (EQ VAS) .(6 months)
- Incidence of insufficient therapeutic response(6 months)
- time to peak (Tmax)(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
- area under the concentration-time curve from time zero to the last quantifiable time point (AUC₀-ₗₐₛₜ)(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
- elimination half-life (t₁/₂)(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
- incremental recovery(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
- The time for FⅧ activity to decline to 15%, 5%, 3% and 1% .(At Visit 1 (Day 0 through Day 4) and Visit 5 (Day 160 through Day 164).)
研究者
研究点 (38)
Loading locations...
相似试验
进行中（未招募）
3 期
Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-Demand TreatmentSevere Hemophilia ANCT07663903Hangzhou Gensciences Biopharmaceutical Co., Ltd.60
尚未招募
不适用
Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in IndiaHemophilia ANCT07446010Bayer33
招募中
3 期
Long-term Follow-up Study of Male Adults with Hemophilia B Previously Treated with Etranacogene Dezaparvovec (CSL222)Hemophilia B2023-503765-37-00CSL Behring LLC28
尚未招募
2/3 期
Effect of Praval bhasma, Lakshadi guggulu and local application of Dashang Lepa with pinda tail on knee joint pain in Hemophilia patientsCTRI/2024/01/061535Central Council for Research in Ayurvedic Sciences60
已完成
3 期
A Clinical Trial Evaluating the Safety of Hydronidone Capsules in Patients With Hepatic Fibrosis and Liver CirrhosisNCT07343778Beijing Continent Pharmaceutical Co, Ltd.337