A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Cerebroside Lipidosis Syndrome Clinical Trial

Status Completed

Clinical Trial Summary

This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.

The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.

Clinical Trial Description

n/a

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cerebroside Lipidosis Syndrome
• Clucocerebrosidase Deficiency Disease
• Deficiency Diseases
• Gaucher Disease
• Gaucher Disease Type I
• Gaucher Disease, Non-Neuronopathic Form
• Glucosylceramide Beta-Glucosidase Deficiency Disease

• NCT number: NCT00365131
• Study type: Interventional
• Source: Sanofi
• Status: Completed
• Phase: Phase 4
• Start date: December 1997
• Completion date: July 2004