Hemophilia APhase 3RH-107-III02NCT04845399
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
A Phase 3 study testing Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in people with Hemophilia A, run by Zhengzhou Gensciences Inc at 16 sites in China.
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. Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 101 participants
- Where
- 16 sitesChina
- Study dates
- 16 Apr 2021to 26 Jun 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
China16 sites
Capital Medical University affiliated Beijing Children's Hospital
Beijing, Beijing Municipality, 100045
Xiangya Hospital of Central South University
Changsha, Hunan, 410013
Chongqing Three Gorges Central Hospital
Chongqing, Chongqing Municipality, 404000
The Second Affiliated Hospital of Guangzhou Medical University
Guangzhou, Guangzhou, 510260
Nanfang Hospital of Southern Medical University
Guangzhou, Guangzhou, 510515
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004
Anhui Provincial Hospital
Hefei, Anhui, 230001
Jinan central hospital
Ji'nan, Shandong, 250013
The First Hospital of Lanzhou University
Lanzhou, Gansu, 730000
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 330006
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266000
The Second Hospital of Shanxi Medical University
Taiyuan, Shanxi, 030001
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
Tianjin, Tianjin Municipality, 300020
The Affiliated Hospital of Xuzhou Medical College
Xuzhou, Jiangsu, 221002
Henan provincial People's Hospital
Zhengzhou, He'nan, 450003
Henan Cancer Hospital
Zhengzhou, He'nan, 450008
About this study
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
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.
- Conditions
- Hemophilia A
- Being tested
- Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
- Keywords
- Hemophilia A, Factor VIII-Fc Fusion Protein, Phase III Expansion Trial, Efficacy and Safety
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months. Receives: Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: For the duration of study participation, 6 months.
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.
Measured: For the duration of study participation, 6 months.
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).
Measured: For the duration of study participation, 6 months.
Secondary outcomes (7)
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.
Measured: For the duration of study participation, 6 months.
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.
Measured: For the duration of study participation, 6 months.
Quality of life assessment.
Quality of life assessment by Haemophilia Joint Health Score(HJHS 2.1).
Measured: For the duration of study participation, 6 months.
Score of bleeding symptoms and Vital signs.
Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale.
Measured: For the duration of study participation, 6 months.
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).
Measured: For the duration of study participation, 6 months.
Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).
A test to analyze the formation of antibodies to CHO.
Measured: For the duration of study participation, 6 months.
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.
Measured: For the duration of study participation, 6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2021
- Study start16 Apr 2021
- Primary completion26 Jun 2021
- Study completion26 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zhengzhou Gensciences Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Renchi Yang, Principal investigator, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04845399
- Sponsor's protocol ID
- CTR20210593
- Acronym
- RH-107-III02
- Status last verified
- Apr 2021
- First submitted
- 9 Apr 2021
- Last updated
- 29 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04845399
- Acronym / sponsor ID
- RH-107-III02 / CTR20210593
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Factor VIII-Fc Fusion Protein, Phase III Expansion Trial, Efficacy and Safety
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- Zhengzhou Gensciences IncIndustry (pharma, biotech, devices)
- Collaborators
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
- Study start
- 16 Apr 2021 (actual)
- Primary completion
- 26 Jun 2021 (actual)
- Study completion
- 26 Jun 2021 (actual)
- First posted
- 15 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionParticipants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionParticipants received prophylaxis treatment at 50 IU/ kg every three days.30-50 IU/kg is recommended to administration while bleeding occurs during the experiment. | Drug | Arm 1 |
Primary outcome
Annualized Bleeding Rates (ABR).
Time frame: For the duration of study participation, 6 months.
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.
Time frame: For the duration of study participation, 6 months.
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)
Time frame: For the duration of study participation, 6 months.
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).
7 secondary outcomes
Total Dose Required for Resolution of a Bleeding Episode.
Time frame: For the duration of study participation, 6 months.
Number of injections required to resolve a bleeding episode.
Time frame: For the duration of study participation, 6 months.
Quality of life assessment.
Time frame: For the duration of study participation, 6 months.
Score of bleeding symptoms and Vital signs.
Time frame: For the duration of study participation, 6 months.
Number of participants with inhibitor development
Time frame: For the duration of study participation, 6 months.
Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).
Time frame: For the duration of study participation, 6 months.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events. (SAEs) as a Measure of Safety and Tolerability.
Time frame: For the duration of study participation, 6 months.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Renchi Yang principal investigator
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College.
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in people with Hemophilia A, run by Zhengzhou Gensciences Inc at 16 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Bleeding Rates (ABR). (time frame: For the duration of study participation, 6 months.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Aug 2023.