Hemophilia A
Conditions
Keywords
Hemophilia A, Factor VIII-Fc Fusion Protein, Phase III Expansion Trial, Efficacy and Safety
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.
Interventions
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.
Sponsors
Jiangsu Gensciences lnc.
Zhengzhou Gensciences Inc
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Bleeding Rates (ABR). | 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. | 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) | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Score of bleeding symptoms and Vital signs. | For the duration of study participation, 6 months. | Response to treatment with rFVIIIFc for bleeding episodes, using the 4-point bleeding response scale. |
| Number of participants with inhibitor development | For the duration of study participation, 6 months. | 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). |
| Quality of life assessment. | For the duration of study participation, 6 months. | Quality of life assessment by Haemophilia Joint Health Score(HJHS 2.1). |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events. (SAEs) as a Measure of Safety and Tolerability. | For the duration of study participation, 6 months. | 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. |
| Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO). | For the duration of study participation, 6 months. | A test to analyze the formation of antibodies to CHO. |
| Total Dose Required for Resolution of a Bleeding Episode. | For the duration of study participation, 6 months. | 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. | For the duration of study participation, 6 months. | 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. |
Countries
China
Outcome results
None listed