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 ]

median and interquartile range for change from baseline in haemoglobin


Original Primary Outcome: Hemoglobin [ Time Frame: Every 3 months for 12 months ]

median percentage and the interquartile range for change from baseline in haemoglobin


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