Conditions: Gaucher Disease
Keywords: gaucher disease pediatric
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 11
Healthy volunteers: no

Brief summary:
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.

Detailed description:

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: Taliglucerase alfa
Intervention descriptions: intervention 1: Taliglucerase alfa for infusion every two weeks for 12 months

Protocol document: