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Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A (RH-107-III02)

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Sponsored by
Zhengzhou Gensciences Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A focused on measuring Hemophilia A, Factor VIII-Fc Fusion Protein, Phase III Expansion Trial, Efficacy and Safety

Eligibility Criteria

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

Inclusion Criteria:

  • Patients Who have Completed trial of RH-107-001 (previously treated patients) Previously received Recombinant Human Coagulation Factor VIII-Fc prophylactic.
  • The patient and/or guardian or his or her legal representative must be able to read, understand, and provide signed informed consent, And voluntarily signed the Informed Consent Form.
  • The compliance of patients appeared quite well.
  • Patient who is considered by the investigators suitable for ongoing to accept previously treated.

Exclusion Criteria:

  • Subjects who have not completed trial of RH-107-001or who have completed the Phase III clinical trial but not willing to continue receiving treatment.
  • Subjects who did not participate in the Phase III clinical trial of RH-107-001.

Sites / Locations

  • Anhui Provincial Hospital
  • Capital Medical University affiliated Beijing Children's Hospital
  • Chongqing Three Gorges Central Hospital
  • The First Hospital of Lanzhou University
  • The Second Affiliated Hospital of Guangzhou Medical University
  • Nanfang Hospital of Southern Medical University
  • The Affiliated Hospital of Guizhou Medical University
  • Henan provincial People's Hospital
  • Henan Cancer Hospital
  • Xiangya Hospital of Central South University
  • The Affiliated Hospital of Xuzhou Medical College
  • Jiangxi Provincial People's Hospital
  • Jinan central hospital
  • The Affiliated Hospital of Qingdao University
  • The Second Hospital of Shanxi Medical University
  • 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

Arm Description

Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months.

Outcomes

Primary Outcome Measures

Annualized Bleeding Rates (ABR).
Annualized bleeding rate = (number of bleeding episodes during the efficacy, period/total number of days during the efficacy period)*365.25. The efficacy period begins with the first prophylactic dose of FRSW107 and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods are not included in the efficacy period. A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. Any injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.
Number of target joints.
Describe the number and percentage of cases with target joints ≥1 and their 95% confidence intervals before and after drug administration, and compare the change in the number of target joints from baseline after treatment.
Annualized Joint Bleeding Rate (AJBR)
Annualized joint bleeding rate(AJBR)can be calculated using the following formula: Number of joint bleeding episodesevents during efficacy evaluation period/(number of days in treatment period/365.25).

Secondary Outcome Measures

Total Dose Required for Resolution of a Bleeding Episode.
The total dose required to resolve a bleeding episode per participant, based on the efficacy period. The efficacy period begins with the first dose and ends with the last dose (for a bleed). For 'Per bleeding episode' values, for each bleeding episode, the total dose is the sum of the doses (IU/kg) administered across all injections given to treat that bleeding episode. For 'Per participant' values, the total dose (IU/kg) used to resolve each bleed is averaged across all bleeding episodes per participant.
Number of injections required to resolve a bleeding episode.
The number of injections required to resolve a bleeding episode per participant, based on the efficacy period. The efficacy period begins with the first dose and ends with the last dose (for a bleed). All injections given from the initial sign of a bleed, until the last date/time within the bleed window are counted. The resolution of a bleed is defined as no sign of bleeding following injection for the bleed. For 'Per participant' values, the number of injections required to resolve each bleed is averaged across all bleeding episodes per participant.
Quality of life assessment.
Quality of life assessment by Haemophilia Joint Health Score(HJHS 2.1).
Score of bleeding symptoms and Vital signs.
Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale.
Number of participants with inhibitor development
Number of participants who developed a positive FVIII inhibitor level (≥0.6 Bethesda unit [BU]) during the study was summarized and classified as participants developing low titer inhibitor (i.e. ≤ 5.0 BU) and participants developing high titer inhibitor (i.e. > 5.0 BU).
Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).
A test to analyze the formation of antibodies to CHO.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events. (SAEs) as a Measure of Safety and Tolerability.
An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.

Full Information

First Posted
April 9, 2021
Last Updated
August 24, 2023
Sponsor
Zhengzhou Gensciences Inc
Collaborators
Jiangsu Gensciences lnc.
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1. Study Identification

Unique Protocol Identification Number
NCT04845399
Brief Title
Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
Acronym
RH-107-III02
Official Title
A Single-Arm, Multicenter, Open-label Phase III Expansion Trial Evaluating Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in Adolescent and Adult Patients With Hemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Completed
Study Start Date
April 16, 2021 (Actual)
Primary Completion Date
June 26, 2021 (Actual)
Study Completion Date
June 26, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Zhengzhou Gensciences Inc
Collaborators
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
The primary objectives of the study are to further evaluate the efficacy and safety of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (FRSW107) in adolescent and adult patients with 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, Factor VIII-Fc Fusion Protein, Phase III Expansion Trial, Efficacy and Safety

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
101 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arm 1
Arm Type
Experimental
Arm Description
Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months.
Intervention Type
Drug
Intervention Name(s)
Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Intervention Description
Participants received prophylaxis treatment at 50 IU/ kg every three days.30-50 IU/kg is recommended to administration while bleeding occurs during the experiment.
Primary Outcome Measure Information:
Title
Annualized Bleeding Rates (ABR).
Description
Annualized bleeding rate = (number of bleeding episodes during the efficacy, period/total number of days during the efficacy period)*365.25. The efficacy period begins with the first prophylactic dose of FRSW107 and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods are not included in the efficacy period. A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, within which any symptoms of bleeding at the same location or injections less than or equal to 72 hours apart were considered the same bleeding episode. Any injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.
Time Frame
For the duration of study participation, 6 months.
Title
Number of target joints.
Description
Describe the number and percentage of cases with target joints ≥1 and their 95% confidence intervals before and after drug administration, and compare the change in the number of target joints from baseline after treatment.
Time Frame
For the duration of study participation, 6 months.
Title
Annualized Joint Bleeding Rate (AJBR)
Description
Annualized joint bleeding rate(AJBR)can be calculated using the following formula: Number of joint bleeding episodesevents during efficacy evaluation period/(number of days in treatment period/365.25).
Time Frame
For the duration of study participation, 6 months.
Secondary Outcome Measure Information:
Title
Total Dose Required for Resolution of a Bleeding Episode.
Description
The total dose required to resolve a bleeding episode per participant, based on the efficacy period. The efficacy period begins with the first dose and ends with the last dose (for a bleed). For 'Per bleeding episode' values, for each bleeding episode, the total dose is the sum of the doses (IU/kg) administered across all injections given to treat that bleeding episode. For 'Per participant' values, the total dose (IU/kg) used to resolve each bleed is averaged across all bleeding episodes per participant.
Time Frame
For the duration of study participation, 6 months.
Title
Number of injections required to resolve a bleeding episode.
Description
The number of injections required to resolve a bleeding episode per participant, based on the efficacy period. The efficacy period begins with the first dose and ends with the last dose (for a bleed). All injections given from the initial sign of a bleed, until the last date/time within the bleed window are counted. The resolution of a bleed is defined as no sign of bleeding following injection for the bleed. For 'Per participant' values, the number of injections required to resolve each bleed is averaged across all bleeding episodes per participant.
Time Frame
For the duration of study participation, 6 months.
Title
Quality of life assessment.
Description
Quality of life assessment by Haemophilia Joint Health Score(HJHS 2.1).
Time Frame
For the duration of study participation, 6 months.
Title
Score of bleeding symptoms and Vital signs.
Description
Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale.
Time Frame
For the duration of study participation, 6 months.
Title
Number of participants with inhibitor development
Description
Number of participants who developed a positive FVIII inhibitor level (≥0.6 Bethesda unit [BU]) during the study was summarized and classified as participants developing low titer inhibitor (i.e. ≤ 5.0 BU) and participants developing high titer inhibitor (i.e. > 5.0 BU).
Time Frame
For the duration of study participation, 6 months.
Title
Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).
Description
A test to analyze the formation of antibodies to CHO.
Time Frame
For the duration of study participation, 6 months.
Title
Number of Participants With Adverse Events (AEs) and Serious Adverse Events. (SAEs) as a Measure of Safety and Tolerability.
Description
An AE is any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition.
Time Frame
For the duration of study participation, 6 months.

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients Who have Completed trial of RH-107-001 (previously treated patients) Previously received Recombinant Human Coagulation Factor VIII-Fc prophylactic. The patient and/or guardian or his or her legal representative must be able to read, understand, and provide signed informed consent, And voluntarily signed the Informed Consent Form. The compliance of patients appeared quite well. Patient who is considered by the investigators suitable for ongoing to accept previously treated. Exclusion Criteria: Subjects who have not completed trial of RH-107-001or who have completed the Phase III clinical trial but not willing to continue receiving treatment. Subjects who did not participate in the Phase III clinical trial of RH-107-001.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Renchi Yang
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
Anhui Provincial Hospital
City
Hefei
State/Province
Anhui
ZIP/Postal Code
230001
Country
China
Facility Name
Capital Medical University affiliated Beijing Children's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100045
Country
China
Facility Name
Chongqing Three Gorges Central Hospital
City
Chongqing
State/Province
Chongqing
ZIP/Postal Code
404000
Country
China
Facility Name
The First Hospital of Lanzhou University
City
Lanzhou
State/Province
Gansu
ZIP/Postal Code
730000
Country
China
Facility Name
The Second Affiliated Hospital of Guangzhou Medical University
City
Guangzhou
State/Province
Guangzhou
ZIP/Postal Code
510260
Country
China
Facility Name
Nanfang Hospital of Southern Medical University
City
Guangzhou
State/Province
Guangzhou
ZIP/Postal Code
510515
Country
China
Facility Name
The Affiliated Hospital of Guizhou Medical University
City
Guiyang
State/Province
Guizhou
ZIP/Postal Code
550004
Country
China
Facility Name
Henan provincial People's Hospital
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450003
Country
China
Facility Name
Henan Cancer Hospital
City
Zhengzhou
State/Province
Henan
ZIP/Postal Code
450008
Country
China
Facility Name
Xiangya Hospital of Central South University
City
Changsha
State/Province
Hunan
ZIP/Postal Code
410013
Country
China
Facility Name
The Affiliated Hospital of Xuzhou Medical College
City
Xuzhou
State/Province
Jiangsu
ZIP/Postal Code
221002
Country
China
Facility Name
Jiangxi Provincial People's Hospital
City
Nanchang
State/Province
Jiangxi
ZIP/Postal Code
330006
Country
China
Facility Name
Jinan central hospital
City
Ji'nan
State/Province
Shandong
ZIP/Postal Code
250013
Country
China
Facility Name
The Affiliated Hospital of Qingdao University
City
QingDao
State/Province
Shandong
ZIP/Postal Code
266000
Country
China
Facility Name
The Second Hospital of Shanxi Medical University
City
Taiyuan
State/Province
Shanxi
ZIP/Postal Code
030001
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

Learn more about this trial

Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A

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