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

Hemophilia A Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04845399
• Other study ID #: CTR20210593
• Status: Completed
• Phase: Phase 3
• Start date: April 16, 2021
• Est. completion date: June 26, 2021

Study information

• Verified date: April 2021
• Source: Zhengzhou Gensciences Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 101
• Est. completion date: June 26, 2021
• Est. primary completion date: June 26, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
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.

Locations

Country	Name	City	State
China	Capital Medical University affiliated Beijing Children's Hospital	Beijing	Beijing
China	Xiangya Hospital of Central South University	Changsha	Hunan
China	Chongqing Three Gorges Central Hospital	Chongqing	Chongqing
China	Nanfang Hospital of Southern Medical University	Guangzhou	Guangzhou
China	The Second Affiliated Hospital of Guangzhou Medical University	Guangzhou	Guangzhou
China	The Affiliated Hospital of Guizhou Medical University	Guiyang	Guizhou
China	Anhui Provincial Hospital	Hefei	Anhui
China	Jinan central hospital	Ji'nan	Shandong
China	The First Hospital of Lanzhou University	Lanzhou	Gansu
China	Jiangxi Provincial People's Hospital	Nanchang	Jiangxi
China	The Affiliated Hospital of Qingdao University	QingDao	Shandong
China	The Second Hospital of Shanxi Medical University	Taiyuan	Shanxi
China	Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.	Tianjin	Tianjin
China	The Affiliated Hospital of Xuzhou Medical College	Xuzhou	Jiangsu
China	Henan Cancer Hospital	Zhengzhou	Henan
China	Henan provincial People's Hospital	Zhengzhou	Henan

Sponsors (2)

Lead Sponsor	Collaborator
Zhengzhou Gensciences Inc	Jiangsu Gensciences lnc.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	For the duration of study participation, 6 months.
Primary	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.	For the duration of study participation, 6 months.
Primary	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).	For the duration of study participation, 6 months.
Secondary	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.	For the duration of study participation, 6 months.
Secondary	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.	For the duration of study participation, 6 months.
Secondary	Quality of life assessment.	Quality of life assessment by Haemophilia Joint Health Score(HJHS 2.1).	For the duration of study participation, 6 months.
Secondary	Score of bleeding symptoms and Vital signs.	Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale.	For the duration of study participation, 6 months.
Secondary	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).	For the duration of study participation, 6 months.
Secondary	Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).	A test to analyze the formation of antibodies to CHO.	For the duration of study participation, 6 months.
Secondary	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.	For the duration of study participation, 6 months.