重症血友病A患者を対象とした、静脈内投与の組換え凝固第VIII因子Fc-フォン・ヴィレブランド因子-XTEN融合タンパク質、エファネソクトコグアルファ（BIVV001）の第3相オープンラベル介入試験
基本情報
- NCT ID
- NCT04161495
- ステータス
- 完了
- 試験のフェーズ
- 第3相
- 試験タイプ
- 介入
- 目標被験者数
- 159
- 治験依頼者名
- Sanofi
概要
Primary Objective: \- To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for the prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life outcomes. * To evaluate the efficacy of BIVV001 for perioperative management. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial thromboplastin time (aPTT) and 2-stage chromogenic coagulation factor VIII (FVIII) activity assays.