NCT01132690

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Pfizer · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Taliglucerase alfa	Taliglucerase alfa for infusion every two weeks for 12 months

Timeline

• Start date: 2010-08-01
• Primary completion: 2012-05-01
• Completion: 2012-07-01
• First posted: 2010-05-28
• Last updated: 2018-10-05
• Results posted: 2014-11-11

Locations

3 sites across 3 countries: Israel, Paraguay, South Africa