A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection

Phase 1

Completed

• Conditions: Hemophilia A
• Interventions: Drug: ADVATE; Drug: FRSW107

• Registration Number: NCT05251090
• Lead Sponsor: Jiangsu Gensciences lnc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-06-16
• Primary Completion: 2021-11-15
• Study First Submitted: 2022-02-11
• Study First Submitted QC: 2022-02-11
• Study First Posted: 2022-02-22
• Study Completion: 2022-05-09
• Status Verified: 2023-08
• Last Update Submitted: 2023-08-21
• Last Update Posted: 2023-08-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 13

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm 1	ADVATE	Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period.
Arm 1	FRSW107	Subjects(up to 12 years of age) received two treatments: 50 IU/kg ADVATE in the first period, followed by 50 IU/kg FRSW107 in the second period.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (6)

The Affiliated Hospital of Qingdao University; 🇨🇳 Qingdao, Shandong, China

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology; 🇨🇳 Wuhan, Hubei, China

Beijing Children's Hospital,Capital Medical University; 🇨🇳 Beijing, Beijing, China

Shenzhen Children's Hospita; 🇨🇳 Shenzhen, Guangdong, China

Chengdu Women's and Children's Central Hospital; 🇨🇳 Chengdu, Sichuan, China

Nanfang Hospital of Southern Medical University; 🇨🇳 Guangzhou, Guangzhou, China