Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein

Launched by JIANGSU GENSCIENCES LNC. · Nov 16, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called recombinant human coagulation factor VIII-Fc fusion protein, or FRSW117, for patients with severe hemophilia A. Hemophilia A is a condition where the blood doesn’t clot properly, which can lead to serious bleeding problems. The trial aims to see how well this new treatment prevents bleeding and ensures safety for patients. It will also look at how the body processes the treatment and whether the body has any reactions to it.

To participate in this study, men aged 12 to 65 who have been diagnosed with severe hemophilia A and have a history of bleeding episodes may be eligible. Participants will receive the treatment and be monitored to see how effective it is and whether there are any side effects. It’s important for potential participants to understand all the details of the study and agree to join voluntarily. This could be a great opportunity for those who are looking for new ways to manage their condition.

Gender

MALE

Eligibility criteria

• • inclusion Criteria：
• • 12≤ age ≤65 year-old men;
• • Patients with clinically confirmed severe hemophilia A, i.e. at screening (central laboratory testing) or previous medical records confirm: FⅧ activity \< 1%；
• • Previous documented treatment with any recombinant and/or blood-derived coagulation factor Ⅷ products or cryoprecipitation products and dosed ≥150 exposure days (EDs≥150)
• • Normal prothrombin time (PT) or International Normalized Ratio (INR)\<1.3
• • Bleeding events were recorded in detail for at least 6 months prior to screening（Participants in the on demand /PPX group were required to have at least 6 episodes of spontaneous bleeding within 6 months）
• • 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
• Exclusion Criteria:
• • 1. Known or suspected allergy to the investigational drug or its excipients, including mouse or hamster proteins;
• • 2. Hypersensitivity or anaphylaxis after FⅧ or IgG2 injection in the past;
• • 3. 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;
• • 4. Von Willebrand factor (vWF) antigen test results were lower than the lower limit of normal value;
• • 5. Severe anemia at the screening stage (hemoglobin \&lt; 60 g/L);
• • 6. Platelet count during screening period \&lt; 100×109 /L;
• 7. 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;
• 8. Patients with abnormal renal function:
• • Creatinine clearance (Ccr) \<50 ml/min (according to Cockcroft and Gault formula); orSerum creatinine (Cr) \>1.5x ULN;
• • 9. People 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);
• • 10. Patients with coagulation dysfunction other than hemophilia A;
• • 11. Have a medical condition that may increase the risk of bleeding;
• • 12. A history of drug or alcohol abuse;
• • 13. Have a known mental disorder that may affect trial compliance;
• • 14. Patients who have received transfusions of blood or blood components within 4 weeks prior to screening;
• • 15. Participants who had participated in other clinical trials within 1 month before screening;
• • 16. 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 patients who need to be treated with anticoagulant or antiplatelet drugs or off-label maximum doses of SAID during clinical trials;
• • 17. 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.
• • 18. Study patients who had used emesezumab within 6 months prior to first administration of the drug;
• • 19. Patients who had used monoclonal antibody therapy, Fc fusion protein products (except FRSW107 and FRSW117), PEG products (except FRSW117), or intravenous immunoglobulin infusion within 3 months before the first administration of the investigational drug;
• • 20. Study patients who underwent major surgery within 3 months prior to initial drug administration (major surgery is defined in 6.2.3 Perioperative management);
• • 21. Study patients who have used FⅧ preparation of any standard half-life (e.g., Bycoch, Coproch, Biinidin, Renjie, NoL, Antaine, etc.) within 3 days or 5 half-lives prior to first administration of the drug (taking the elderly); Patients who have used any other extended half-life preparation FⅧ within 4 days or 5 half-lives prior to first dosing (for the elderly);
• • 22. Study patients with fever, severe active bacterial or viral infection, and allergies within 2 weeks before the first administration of the drug;
• • 23. 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;
• • 24. 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);
• • 25. 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.);
• • 26. Have other serious medical conditions that the researchers said could not benefit from them
• • 27. Subjects deemed unsuitable by other investigators.