A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase
alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to
treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV)
infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12
months. At the end of the 12-month treatment period eligible subjects will be offered
enrollment in an open-label extension study if taliglucerase alfa is not commercially
available.

本研究为一项多中心、双盲临床试验，旨在评估他利苷酶α（taliglucerase alfa）用于2岁至未满18岁初治戈谢病（Gaucher disease）受试者的安全性与有效性。受试者将被随机分配至30单位/千克或60单位/千克两种剂量之一接受治疗，每两周接受一次他利苷酶α的静脉（intravenous，IV）输注。总治疗周期为12个月。在12个月治疗期结束后，若他利苷酶α尚未实现商业化供应，将为符合入组标准的受试者提供开放标签扩展研究的入组资格。