[Translation] A multicenter, open-label study to evaluate the safety, efficacy, and pharmacokinetics of velaglucerase alfa in Chinese subjects with type 1 Gaucher disease
主要目的:
本研究的主要目的是通过评估 VPRIV 在中国 1 型戈谢病受试者中的 EOW IV 输注,最长给药 51 周的治疗中严重治疗相关不良事件 (TEAE) 的发生率,从而评价 VPRIV 的安全性。
次要目的:
本研究的次要目的是评估: VPRIV 的其他安全性参数(包括 TEAE 和 IRR 的发生率和抗体形成率) VPRIV 对血液学表现的影响 VPRIV 对肝脏和脾脏体积的影响 VPRIV 对 QoL 的影响 VPRIV 的 PK VPRIV 对疾病生物标志物的影响
[Translation] Primary Objective:
The primary objective of this study is to evaluate the safety of VPRIV by assessing the incidence of serious treatment-related adverse events (TEAEs) during treatment with EOW IV infusion in Chinese type 1 Gaucher disease subjects for up to 51 weeks.
Secondary Objectives:
The secondary objectives of this study are to evaluate: Other safety parameters of VPRIV (including the incidence of TEAEs and IRRs and the rate of antibody formation) Effect of VPRIV on hematological manifestations Effect of VPRIV on liver and spleen volumes Effect of VPRIV on QoL PK of VPRIV Effect of VPRIV on disease biomarkers