A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz 112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme

INTERVENTION: see 'background of the study' CONDITION: ; lysosomal storage disease ; metabolic disease 10018849 10021605 10027424 PRIMARY OUTCOME: The primary efficacy endpoint will be the percentage (%) of patients who remain ; stable for 52 weeks (the primary analysis period) assessed for both treatment ; groups separately along with a difference between the two treatment groups. ; For a patient to be considered to have demonstrated a clinically meaningful ; response to treatment with Genz 112638 or Cerezyme, patients must remain stable ; in hematological parameters (hemoglobin levels and platelet counts), and organ ; volumes (spleen, when applicable, and liver volumes in multiples of normal ; [MN]). A blinded Independent Adjudication Board (IAB) will review and confirm ; that failure to meet the primary endpoint is attributed to a decline in Gaucher ; disease.; SECONDARY OUTCOME: The secondary efficacy endpoints will include the following: Total T‐ and ; Z‐scores for bone mineral density (dual‐energy X‐ray absorptiometry [DXA]) of ; femur and lumbar spine, hemoglobin level, platelet count, and spleen and liver ; volumes (in MN) (assessed by magnetic resonance imaging [MRI]). ; ; The tertiary efficacy endpoints include the following: Biomarkers (chemokine CC ; motif ligand 18 [CCL18] and chitotriosidase); bone disease assessments (X‐ray, ; MRI and bone marrow burden score); Gaucher assessments (mobility, bone crisis, ; and bone pain); Quality of Life (QOL) (Brief Pain Inventory [BPI], Fatigue ; Severity Score [FSS], Short Form‐36 Health Survey (SF‐36®), and treatment ; preference (oral vs intravenous therapy). ; ; Exploratory endpoints include Gaucher disease Severity Score System (DS3) and ; the percent changes from Baseline in investigational biomarkers including ; glucosylceramide (GL‐1) assayed from dried blood spots [DBS] on filter paper ; and from plasma, as well as ceramide, high‐sensitivity C‐reactive protein ; (hsCRP), apolipoprotein‐B‐100, sphingomyelin, and macrophage inflammatory ; protein‐1 beta (MIP1‐*) (assayed from plasma). ; INCLUSION CRITERIA: 1. The patient is willing and able to provide signed informed consent prior to any study‐related procedures to be performed. 2. The patient is 18 to 65 years of age at the time of randomization. 3. The patient*s Tanner Stage should be * 4 prior to randomization. 4. The patient has a diagnosis of Gaucher disease type 1 confirmed by a documented deficiency of acid *‐glucosidase activity by enzyme assay. 5. The patient consents to provide a blood sample for genotyping for Gaucher disease (unless the patient*s Gaucher genotype is already available), chitotriosidase, and for genotyping of cytochrome P450 2D6 (CYP2D6) to categorize the patient*s predicted rate of metabolism. 6. The patient has received treatment with Cerezyme for at least 3 years at a prescribed dose of * 20 U/kg to * 60 U/kg (± 5 U/kg) q2w during the last year of treatment and has not had a dose reduction, regimen change, or treatment interruption for greater than 6 consecutive mon