A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Severe Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05265286
• Other study ID #: CTR20220283
• Status: Completed
• Phase: Phase 2
• Start date: April 14, 2022
• Est. completion date: August 21, 2022

Study information

• Verified date: May 2023
• Source: Jiangsu Gensciences lnc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: August 21, 2022
• Est. primary completion date: August 21, 2022
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 65 Years

Eligibility

Key Inclusion Criteria:

• The activity of the coagulation factor VIII (FVIII:C) < 1%.

• 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.• Significant hepatic or renal impairment (ALT and AST > 2×ULN; serum bilirubin level > 2 × upper limit of normal (ULN), Urea /BUN > 2×ULN, Cr > 176.8 µmol/L)

• 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 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

Other protocol-defined inclusion/exclusion Criteria May Apply.

Related Conditions & MeSH terms

• Hemophilia A
• Severe Hemophilia A

Intervention

Drug:
• FRSW117
Subjects of high dose group are being received four doses(50 IU/kg,once a week or every 7 days) of FRSW117. Subjects of low dose group (40 IU/kg,once a week or every 7 days)are being received four doses of FRSW117.

Locations

Country	Name	City	State
China	The Affiliated Hospital of Qingdao University	Qingdao	Shandong
China	People'S Hospital of Rizhao	Rizhao	Shandong
China	Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.	Tianjin	Tianjin
China	Affiliated Hospital of Jiangnan University	Wuxi	Jiangsu
China	People'S Hospital of Zhengzhou	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
Jiangsu Gensciences lnc.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Maximum measured concentration of FVIII:C (Cmax)	Measured by One-Stage Clotting Assay	Pre-dose and post dose up to 10 days
Primary	Time required for the concentration of the drug to reach half of its original value (T1/2)	Measured by One-Stage Clotting Assay	Pre-dose and post dose up to 10 days
Primary	Area Under the Curve to Infinity (AUC)	Measured by One-Stage Clotting Assay	Pre-dose and post dose up to 10 days
Primary	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)	Measured by One-Stage Clotting Assay	Pre-dose and post dose up to 10 days
Primary	Evaluation of the level of anti-PEG-rF?Fc antibody production in participants		Pre-dose and post dose up to 36 days
Primary	Evaluation of the level of anti-PEG antibody production in participants		Pre-dose and post dose up to 36 days
Secondary	Number of participants with treatment-related adverse events as assessed by CTCAE V5.0	Adverse events related to PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.	Pre-dose and post dose up to 36 days
Secondary	Development of Inhibitor	Measured by the Nijmegen-Modified Bethesda Assay	Pre-dose and post dose up to 36 days
Secondary	score of bleeding symptoms and Vital signs	Response to treatment with PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for bleeding episodes, using the 4-point bleeding response scale	Pre-dose and post dose up to 36 days