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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With

Brief 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.


Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: Number of Participants With Clinical Success at Month 24/Discontinuation [ Time Frame: Month 24 (or at time of discontinuation) ]

Patients are considered to be a clinical success if ALL of the following are met: The patient's hemoglobin does not fall more than 1.25g/dL for women or 1.5 g/dL for men below the patient's baseline value, platelet count does not fall more than 25% below the patient's baseline value or does not fall below 80,000 mm3, liver and spleen volumes are not greater than 20% above the patient's baseline value, no evidence of bone disease progression, including no incidence of pathologic fractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.


Original Primary Outcome: Maintenance of Clinical Response

Current Secondary Outcome:

  • Mean Composite Scores of the SF-36 Health Survey at Baseline [ Time Frame: Baseline ]
    The mean composite scores (0 being worst and 100 being best) for both treatment groups at Baseline. Composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
  • Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation. [ Time Frame: Month 24 (or at time of discontinuation) ]
    The mean composite scores (0 being worst and 100 being best) for both treatment groups at Month 24/Discontinuation. The mean composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
  • Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation [ Time Frame: Baseline and Month 24 (or at time of discontinuation) ]
    The mean composite scores (0 being worst and 100 being best) for both treatment groups approximated those of the general population at baseline. Composite score - The overall composite scores were comprised of a standardized physical and mental component score.


Original Secondary Outcome: Quality of life

Information By: Sanofi

Dates:
Date Received: August 15, 2006
Date Started: December 2001
Date Completion:
Last Updated: March 17, 2015
Last Verified: March 2015