A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Status: | Archived |
---|---|
Conditions: | Metabolic |
Therapuetic Areas: | Pharmacology / Toxicology |
Healthy: | No |
Age Range: | Any |
Updated: | 7/1/2011 |
Start Date: | August 2010 |
End Date: | December 2011 |
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.
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