Conditions: Gaucher Disease
Keywords: enzyme replacement therapy
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 19
Healthy volunteers: no

Brief summary:
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.

Detailed description:

Arms:
arm 1: Subjects randomized to receive 30 units/kg arm 2: Subjects randomized to 60 units/kg

Interventions: intervention 1: Taliglucerase alfa
Intervention descriptions: intervention 1: Taliglucerase infusion every two weeks for 21 months

Protocol document: