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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
11
Taliglucerase alfa for infusion every two weeks for 12 months
Shaare Zedek Medical Center
Jerusalem, Israel
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Barrio Sajonia Asunción, Paraguay
Morningside Medi-Clinic
Morningside, South Africa
Hemoglobin
median and interquartile range for change from baseline in haemoglobin
Time frame: Every 3 months for 12 months
Chitotriosidase
Percent change from baseline in chitotriosidase
Time frame: Every 3 months for 12 months
Spleen Volume
Spleen volume measured by MRI
Time frame: Baseline and Month 12
Platelet Count
Mean and standard deviation of platelet count per cubic mm
Time frame: Baseline and 12 months
Chemokine (C-C Motif) Ligand 18 (CCL18)
Percent change from baseline in CCL18
Time frame: Every 3 months for 12 months
Liver Volume
Liver volume measured by MRI
Time frame: Baseline and Month 12
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