重症血友病AおよびB患者（以前に因子製剤またはバイパス製剤による予防療法を受けていた患者）におけるフィツシランの研究
基本情報
- NCT ID
- NCT03549871
- 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
- 80
- 治験依頼者名
- Sanofi
概要
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA) prophylaxis. Secondary Objectives: * To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis: * the frequency of spontaneous bleeding episodes * the frequency of joint bleeding episodes * health related quality of life (HRQOL) in participants greater than or equal to (\>=) 17 years of age * To characterize the frequency of bleeding episodes during the onset and treatment periods in participants receiving fitusiran. * To characterize the safety and tolerability of fitusiran. * To characterize the annualized weight-adjusted consumption of factor/BPA while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis.