A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

• Status: Recruiting
• Trial end date: 2022-07-30
• Target enrollment: 0
• Participant gender: Male

Summary

The primary objectives of the study are to evaluate the Pharmacokinetics，Safety and tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW117) in patients with severe hemophilia A.

The secondary objectives are to monitor anti-durg antibodies and anti-PEG antibodies levels in patients with severe hemophilia A

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Jiangsu Gensciences lnc.
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Patients with clinically confirmed hemophilia A (coagulation factor VIII <1%) and previous medical records confirming exposure to coagulation factor VIII for ≥150 days (EDs ≥150).

• Non-immunodeficient, with some immunity (CD4 > 200/μL).

• Platelet count >100×10^9/L.

• Normal prothrombin time (PT) or international normalized ratio (INR) <1.3.

• Negative lupus anticoagulant.

• Fully understand, informed about this study and sign the informed consent form, voluntarily participate in the clinical study and have the ability to complete all study procedures

Exclusion Criteria:

• Hypersensitivity to the test substance or its excipients (including rodent or hamster protein).

• Pre-existing hypersensitivity or allergic reactions to FVIII or IgG2 injection therapy.

• Positive FVIII inhibitor at screening (≥0.6 BU/mL), or previous history of FVIII inhibitor Positive history, or family history of inhibitors.

• Patients with other coagulation disorders in addition to hemophilia A.

• The results of vWF antigen examination lower than normal.

• Severe anemia and need blood transfusion (hemoglobin < 60g/L).

• Patients who have received any standard half-lives FVIII formulations (e.g., Kogenate, Kovaltry, Advate, Xyntha, etc.) or received any other half-life-extending FVIII formulations within 4 days or 5 half-lives (whichever is longer) before administration.

• Patients who had used emecizumab within 6 months prior to administration.

• Patients with fever, upper respiratory tract infection or allergy symptoms within the previous 2 weeks before screening.

• Suffer from other diseases that may increase the risk of bleeding or the risk of thrombosis.

• Severe cardiovascular and cerebrovascular diseases: such as cerebral hemorrhage, stroke, myocardial infarction, unstable angina pectoris, congestive heart failure(the current New York Heart Association cardiac function grade III, Hypertension that cannot be controlled with drug treatment: systolic blood pressure> 160 mmHg or diastolic blood pressure> 95 mmHg.

• 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)).

• Abnormal kidney function: BUN > 2×ULN, Cr > 2.0mg/dL.

• One or more clinically significant tests for Hepatitis B Virus Surface Antigen, Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody.

• Patients who received any anticoagulant or antiplatelet therapy within a week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials.

• Systemic immunomodulators (e.g., corticosteroids [equivalent dose of 10 mg/ day prednisone], A-interferon, immunoglobulin, cyclophosphamide, cyclosporine, etc.) were used within 14 days prior to administration or during the study period.

• Patients having major surgery or receiving blood or blood 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 a 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.