Hemophilia A
Conditions
Keywords
Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability
Brief summary
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.
Interventions
Sponsors
Jiangsu Gensciences lnc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
No minimum to 12 Years
Healthy volunteers
No
Inclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Maximum measured concentration of FVIII:C (Cmax) | Pre-dose and post dose up to 8 days. | Measured by aPTT Clotting Assay. |
| Time required for the concentration of the drug to reach half of its original value (T1/2) | Pre-dose and post dose up to 8 days | Measured by aPTT Clotting Assay. |
| Area Under the Curve to Infinity (AUC) | Pre-dose and post dose up to 8 days. | Measured by aPTT Clotting Assay. |
| The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL). | Pre-dose and post dose up to 8 days. | Measured by aPTT Clotting Assay. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE V5.0. | Post dose up to 32 days. | Adverse events related to Recombinant Human Coagulation Factor VIII-Fc fusion protein for Injection according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0. |
| Development of Inhibitor | Pre-dose and post dose up to 32 days. | Measured by the Nijmegen-Modified Bethesda Assay. |
Countries
China
Outcome results
None listed