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

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
ADVATE
FRSW107
Sponsored by
Jiangsu Gensciences lnc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability

Eligibility Criteria

undefined - 12 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Beijing Children's Hospital,Capital Medical University
  • Shenzhen Children's Hospita
  • Nanfang Hospital of Southern Medical University
  • Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
  • The Affiliated Hospital of Qingdao University
  • Chengdu Women's and Children's Central Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm 1

Arm Description

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.

Outcomes

Primary Outcome Measures

Maximum measured concentration of FVIII:C (Cmax)
Measured by aPTT Clotting Assay.
Time required for the concentration of the drug to reach half of its original value (T1/2)
Measured by aPTT Clotting Assay.
Area Under the Curve to Infinity (AUC)
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).
Measured by aPTT Clotting Assay.

Secondary Outcome Measures

Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
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
Measured by the Nijmegen-Modified Bethesda Assay.

Full Information

First Posted
February 11, 2022
Last Updated
August 21, 2023
Sponsor
Jiangsu Gensciences lnc.
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1. Study Identification

Unique Protocol Identification Number
NCT05251090
Brief Title
A Study to Evaluate the Safety and Pharmacokinetic of Recombinant Human Coagulation Factor VIII ，Fc Fusion Protein for Injection
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
June 16, 2021 (Actual)
Primary Completion Date
November 15, 2021 (Actual)
Study Completion Date
May 9, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jiangsu Gensciences lnc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
Keywords
Hemophilia A;Pharmacokinetics;Child;Safety and Tolerability

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm 1
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
ADVATE
Intervention Description
50 international units (IU)/kg, a single dose.
Intervention Type
Drug
Intervention Name(s)
FRSW107
Other Intervention Name(s)
Recombinant Human Coagulation Factor VIII, Fc Fusion Protein for Injection
Intervention Description
50 IU/kg, a single dose.
Primary Outcome Measure Information:
Title
Maximum measured concentration of FVIII:C (Cmax)
Description
Measured by aPTT Clotting Assay.
Time Frame
Pre-dose and post dose up to 8 days.
Title
Time required for the concentration of the drug to reach half of its original value (T1/2)
Description
Measured by aPTT Clotting Assay.
Time Frame
Pre-dose and post dose up to 8 days
Title
Area Under the Curve to Infinity (AUC)
Description
Measured by aPTT Clotting Assay.
Time Frame
Pre-dose and post dose up to 8 days.
Title
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).
Description
Measured by aPTT Clotting Assay.
Time Frame
Pre-dose and post dose up to 8 days.
Secondary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events as assessed by CTCAE V5.0.
Description
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.
Time Frame
Post dose up to 32 days.
Title
Development of Inhibitor
Description
Measured by the Nijmegen-Modified Bethesda Assay.
Time Frame
Pre-dose and post dose up to 32 days.

10. Eligibility

Sex
Male
Gender Based
Yes
Gender Eligibility Description
Hemophilia A is an X-chromosome-linked recessive inherited bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII).
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Runhui Wu, PhD
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Xiaoling Wang, MA.Sc
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100045
Country
China
Facility Name
Shenzhen Children's Hospita
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518000
Country
China
Facility Name
Nanfang Hospital of Southern Medical University
City
Guangzhou
State/Province
Guangzhou
ZIP/Postal Code
510515
Country
China
Facility Name
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430000
Country
China
Facility Name
The Affiliated Hospital of Qingdao University
City
Qingdao
State/Province
Shandong
ZIP/Postal Code
266000
Country
China
Facility Name
Chengdu Women's and Children's Central Hospital
City
Chengdu
State/Province
Sichuan
ZIP/Postal Code
610000
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

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

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