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
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 101
- 试验地点
- 16
- 主要终点
- Annualized Bleeding Rates (ABR).
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Arm 1
Participants will receive prophylaxis treatment with Recombinant Human Coagulation Factor VIII-Fc fusion for 6 months.
干预措施: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection (Drug)
结局指标
主要结局
Annualized Bleeding Rates (ABR).
时间窗: 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.
Annualized Joint Bleeding Rate (AJBR)
时间窗: 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).
Number of target joints.
时间窗: 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.
次要结局
- Number of participants with inhibitor development(For the duration of study participation, 6 months.)
- Total Dose Required for Resolution of a Bleeding Episode.(For the duration of study participation, 6 months.)
- Quality of life assessment.(For the duration of study participation, 6 months.)
- Number of Participants With Incidence of Antibody Formation to CHINESE HAMSTER OVARY (CHO).(For the duration of study participation, 6 months.)
- Score of bleeding symptoms and Vital signs.(For the duration of study participation, 6 months.)
- Number of injections required to resolve a bleeding episode.(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.(For the duration of study participation, 6 months.)