The indication for this product is to control bleeding in patients with hemophilia A (congenital deficiency of factor VIII).
The primary objective:
Evaluation of the efficacy of recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW107) as an on-demand treatment in previously treated patients with severe hemophilia A.
Secondary objectives:
Evaluation of the safety and immunogenicity of FRSW107 as an on-demand therapy in previously treated patients with severe hemophilia A.
Evaluate the on-demand treatment's PK profile of FRSW107 in previously treated patients with severe hemophilia A based on population pharmacokinetic (PopPK) methods ; preliminarily investigate the exposure-response (E-R) relationship of FRSW107 on-demand treatment in these patients if data permit.
Males aged 12 or younger to 65 years old.
Patients clinically diagnosed with severe hemophilia A, i.e., those confirmed through screening or previous medical records: FⅧ Activity < 1%.
Previous records confirm receipt of any recombinant and/or blood-derived coagulation factor VIII products or cold precipitate products, with a treatment of ≥150 exposure days (EDs ≥150).
The participants have fully understood and been informed of this study, signed the informed consent form, and voluntarily enrolled in the clinical trial. The trial participants and/or their guardians are capable of cooperating with the hemostatic treatment and have the ability to complete all study procedures.
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;
Single group
FRSW107
DrugTreatment for 6 months as needed.
The haemostatic effective rate
The haemostatic effect of FRSW107 when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.
Number of infusions and IU/kg of FRSW107 needed for the treatment of bleeding episodes
FVIII activity during on-demand: activity recovery rate
FVIII activity recovery rate on Day1's haemostatic therapy.
Hemophilia Joint Health Score (HJHS 2.1) during on-demand treatment
total score and each subscale score of HJHS 2.1, as well as their changes over time.
incidence of lack of drug efficacy
Lack of drug efficacy is defined as: during on-demand treatment, hemostatic efficacy is assessed as "ineffective" or "poor/no response" following two consecutive administrations within 72 hours for the same bleeding episode. incidence of lack of drug efficacy=number of lack of drug efficacy/number of evaluated treatment\*100%.
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