search
Back to results

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

Primary Purpose

Severe Hemophilia A

Status
Completed
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
FRSW117
Sponsored by
Jiangsu Gensciences lnc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Hemophilia A focused on measuring Pharmacokinetics;, Safety;, immunogenicity;, Preliminary efficacy

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • People'S Hospital of Zhengzhou
  • Affiliated Hospital of Jiangnan University
  • The Affiliated Hospital of Qingdao University
  • People'S Hospital of Rizhao
  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm 1 prophylaxis treatment

Arm Description

Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. All subjects are being received PK assessment in ED1 and ED4.

Outcomes

Primary Outcome Measures

Maximum measured concentration of FVIII:C (Cmax)
Measured by One-Stage Clotting Assay
Time required for the concentration of the drug to reach half of its original value (T1/2)
Measured by One-Stage Clotting Assay
Area Under the Curve to Infinity (AUC)
Measured by One-Stage 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 One-Stage Clotting Assay
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
Evaluation of the level of anti-PEG antibody production in participants

Secondary Outcome Measures

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.
Development of Inhibitor
Measured by the Nijmegen-Modified Bethesda Assay
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

Full Information

First Posted
February 22, 2022
Last Updated
May 12, 2023
Sponsor
Jiangsu Gensciences lnc.
search

1. Study Identification

Unique Protocol Identification Number
NCT05265286
Brief Title
A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Completed
Study Start Date
April 14, 2022 (Actual)
Primary Completion Date
August 21, 2022 (Actual)
Study Completion Date
August 21, 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, 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Hemophilia A
Keywords
Pharmacokinetics;, Safety;, immunogenicity;, Preliminary efficacy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm 1 prophylaxis treatment
Arm Type
Experimental
Arm Description
Subjects of high dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. Subjects of low dose group are being received four doses of FRSW117. dosing on day1(ED1), day8(ED2), day15(ED3), day22(ED4) respectively. All subjects are being received PK assessment in ED1 and ED4.
Intervention Type
Drug
Intervention Name(s)
FRSW117
Other Intervention Name(s)
PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Intervention Description
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.
Primary Outcome Measure Information:
Title
Maximum measured concentration of FVIII:C (Cmax)
Description
Measured by One-Stage Clotting Assay
Time Frame
Pre-dose and post dose up to 10 days
Title
Time required for the concentration of the drug to reach half of its original value (T1/2)
Description
Measured by One-Stage Clotting Assay
Time Frame
Pre-dose and post dose up to 10 days
Title
Area Under the Curve to Infinity (AUC)
Description
Measured by One-Stage Clotting Assay
Time Frame
Pre-dose and post dose up to 10 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 One-Stage Clotting Assay
Time Frame
Pre-dose and post dose up to 10 days
Title
Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants
Time Frame
Pre-dose and post dose up to 36 days
Title
Evaluation of the level of anti-PEG antibody production in participants
Time Frame
Pre-dose and post dose up to 36 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 PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0.
Time Frame
Pre-dose and post dose up to 36 days
Title
Development of Inhibitor
Description
Measured by the Nijmegen-Modified Bethesda Assay
Time Frame
Pre-dose and post dose up to 36 days
Title
score of bleeding symptoms and Vital signs
Description
Response to treatment with PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for bleeding episodes, using the 4-point bleeding response scale
Time Frame
Pre-dose and post dose up to 36 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)
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Renchi Yang, PhD
Organizational Affiliation
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
Official's Role
Principal Investigator
Facility Information:
Facility Name
People'S Hospital of Zhengzhou
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450000
Country
China
Facility Name
Affiliated Hospital of Jiangnan University
City
Wuxi
State/Province
Jiangsu
ZIP/Postal Code
214100
Country
China
Facility Name
The Affiliated Hospital of Qingdao University
City
Qingdao
State/Province
Shandong
ZIP/Postal Code
266000
Country
China
Facility Name
People'S Hospital of Rizhao
City
Rizhao
State/Province
Shandong
ZIP/Postal Code
276800
Country
China
Facility Name
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs