Clinical Trial: A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
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 Summary:
Sponsor: Protalix
Current Primary Outcome: Hemoglobin [ Time Frame: Every 3 months for 12 months ]
Original Primary Outcome: Hemoglobin [ Time Frame: Every 3 months for 12 months ]
Current Secondary Outcome:
- Chitotriosidase [ Time Frame: Every 3 months for 12 months ]Percent change from baseline in chitotriosidase
- Spleen Volume [ Time Frame: Baseline and Month 12 ]Spleen volume measured by MRI
- Platelet Count [ Time Frame: Baseline and 12 months ]Mean and standard deviation of platelet count per cubic mm
- Chemokine (C-C Motif) Ligand 18 (CCL18) [ Time Frame: Every 3 months for 12 months ]Percent change from baseline in CCL18
- Liver Volume [ Time Frame: Baseline and Month 12 ]Liver volume measured by MRI
Original Secondary Outcome:
- Chitotriosidase or CCL18 [ Time Frame: Every 3 months for 12 months ]Percent change from baseline in chitotriosidase or CCL18
- Spleen and liver volume [ Time Frame: Baseline and Month 12 ]Percent change in spleen and liver volume measured by MRI (or ultrasound)
- Platelet count [ Time Frame: Every 3 months for 12 months ]Percent change from baseline in platelet count
- Anti-taliglucerase alfa antibodies [ Time Frame: Every 3 months for 12 months ]Occurrence of positive antibody response
Information By: Protalix
Dates:
Date Received: May 26, 2010
Date Started: August 2010
Date Completion:
Last Updated: November 10, 2014
Last Verified: November 2014