A Phase I, Multicentre, Open-label Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection in Children With Severe Hemophilia A

CTR20220279

Primary objective: To assess the pharmacokinetics of Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection (FRSW107) Secondary objectives: To assess Safety and Tolerability by monitoring FVIII recovery and adverse events in Severe Hemophilia A.

2021-06-16

2021-11-15

2022-05-09

Completed

Early phase I

13

Key Inclusion Criteria: * The activity of the coagulation factor VIII (FVIII:C) \< 1%. Less than 6 years old Patients previously treated with FVIII concentrate (s) for a minimum of 50 exposure days (EDs) prior to study entry. 6 years old to 12 years old Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry. * Normal prothrombin time or INR \< 1.3. * Negative lupus anticoagulant. Key Exclusion Criteria: * 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 II immunoglobulin 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 72 h prior to administration. * Significant hepatic or renal impairment (ALT and AST \> 2×ULN; serum bilirubin level \> 2 × upper limit of normal (ULN), BUN \> 2×ULN, Cr \> 2.0 ULN). * 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 one month prior to administration. * 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.

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2023-08-22