Dataset from A-LONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia A

The primary objectives of this study are: to evaluate the safety and tolerability of rFVIIIFc
administered as a prophylaxis (Arm 1), weekly (Arm 2), on-demand (Arm 3), and surgical
treatment regimen; to evaluate the efficacy of the rFVIIIFc tailored prophylaxis regimen (Arm
1); to evaluate the efficacy of rFVIIIFc administered as an on-demand (Arm 3) and surgical
treatment regimen. The secondary objectives of this study are: to characterize the PK profile
of rFVIIIFc and compare the PK of rFVIIIFc with the currently marketed product, Advate®; to
characterize the range of dose and schedules required to adequately prevent bleeding in a
prophylaxis regimen, maintain hemostasis in a surgical setting, or to treat bleeding episodes
in an on-demand, weekly treatment, or prophylaxis setting.

本研究的主要目标为：评估重组凝血因子Ⅶ-Fc融合蛋白（rFVIIIFc）分别以预防性给药方案（队列1）、每周给药方案（队列2）、按需给药方案（队列3）及外科治疗方案给药时的安全性与耐受性；评估定制化预防性给药方案（队列1）下重组凝血因子Ⅶ-Fc融合蛋白的临床疗效；评估按需给药方案（队列3）及外科治疗方案给予重组凝血因子Ⅶ-Fc融合蛋白的临床疗效。本研究的次要目标为：表征重组凝血因子Ⅶ-Fc融合蛋白的药代动力学特征，并将其与当前上市商品Advate®的药代动力学特征进行对比；明确可在预防性给药方案中充分预防出血、外科场景中维持止血，或在按需给药、每周给药及预防性给药场景中治疗出血发作的剂量范围与给药方案。