An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A.

Zhengzhou Gensciences

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT04456387

CTR20201212

Details and patient eligibility

About

The primary objectives of the study are to evaluate the efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the Prevention and Treatment of Bleeding in patients with hemophilia A.

The secondary objectives are to evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in the prevention and treatment of bleeding episodes, to investigate the quality of life in patients who used the FRSW107.

Enrollment: 119 patients

Sex: Male

Ages: 12 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 1)Male, aged 12 to 60 years
• 2)Severe hemophilia A. The activity of the coagulation factor VIII (FVIII:C) < 1%, and previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry.
• 3. No history of a positive inhibitor test (< 0.6 BU) or clinical signs of decreased response to FVIII administrations within 2 years before the test or during the screening period. No Family history of inhibitors.
• 4. Non-immune deficiency, with a certain immune capacity (CD4 > 200/μL)
• 5. Platelet count > 100,000 platelets/μL.
• 6. Normal prothrombin time or INR < 1.3.
• 7. Normal previous results of vWF antigen examination.
• 8. Negative lupus anticoagulant.
• 9. the patient has a detailed record of bleeding events for at least 6 months (the subject can be admitted to the on-demand treatment group with spontaneous bleeding ≥3 times within 6 months).
• 10. Capable of understanding and willing to comply with the conditions of the protocol have read (patient and/or guardian).

Exclusion criteria

• 1. Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins).
• 2. History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration.
• 3. Other coagulation disorder(s) in addition to hemophilia A.
• 4. Patients with severe heart disease, including myocardial infarction, heart failure (III or higher level).
• 5. Clinically significant of other systematic diseases: alcoholism, drug abuse, mental disorders and mental retardation.
• 6. Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 3 × upper limit of normal (ULN), BUN > 2×ULN, Cr > 176.8µmol/L).
• 7. 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.
• 8. 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.
• 9. Patients who previously participated in the other clinical trials within 1 month prior screening.
• 10. 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.
• 11. Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation.
• 12. Patient who is considered by the other investigators not suitable for clinical study.

NOTE:Other protocol-defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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119 participants in 2 patient groups

Arm 1-on demand treatment

Experimental group

Description:

Part A-Participants will receive on-demand treatment with Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection for 6 months.

Treatment:

Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Arm 2- prophylaxis treatment

Experimental group

Description:

Part B-Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months. 12 Participants of them will receive PK assessment at ED1 and ED35(Participants will be tested for PK assessment at timepoints throughout the study based on exposure days (ED). One ED is equivalent to a 24 hours period in which drug is dosed.)，measured by one-stage assay, they would not be given prophylaxis treatment until the completion of PK blood collection.

Treatment:

Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection