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Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 95

Last updated 2015-04-03

Study results available
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Summary

This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.

Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.

Conditions

  • Gaucher Disease, Type 1
  • Cerebroside Lipidosis Syndrome
  • Glucocerebrosidase Deficiency Disease
  • Glucosylceramide Beta-Glucosidase Deficiency Disease
  • Gaucher Disease, Non-Neuronopathic Form

Interventions

DRUG

Cerezyme

Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Edward Kaye, M.D. · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2001-12-31
Completion
2007-02-28

Countries

  • United States
  • Brazil
  • Canada
  • Italy
  • Poland
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00364858 on ClinicalTrials.gov