Trial record 3 of 58 for:    GBA

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

This study has been completed.
Sponsor:
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00364858
First received: August 15, 2006
Last updated: September 3, 2009
Last verified: May 2009
  Purpose

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.


Condition Intervention Phase
Gaucher Disease, Type 1
Cerebroside Lipidosis Syndrome
Glucocerebrosidase Deficiency Disease
Glucosylceramide Beta-Glucosidase Deficiency Disease
Gaucher Disease, Non-Neuronopathic Form
Drug: Cerezyme
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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 Type 1 Gaucher Disease

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Number of Participants With Clinical Success at Month 24/Discontinuation [ Time Frame: Month 24 (or at time of discontinuation) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Mean Composite Scores of the SF-36 Health Survey at Baseline [ Time Frame: Baseline ] [ Designated as safety issue: No ]
  • Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation. [ Time Frame: Month 24 (or at time of discontinuation) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]

Enrollment: 95
Study Start Date: December 2001
Study Completion Date: February 2007
Arms Assigned Interventions
Q2 Cerezyme
Patients receiving Cerezyme one infusion every 2 weeks (Q2).
Drug: Cerezyme
Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).
Q4 Cerezyme
Patients receiving Cerezyme one infusion every 4 weeks (Q4).
Drug: Cerezyme
Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient must provide written informed consent prior to undergoing any study-related procedures.
  • The patient has a confirmed diagnosis of Gaucher disease with a documented deficiency of glucocerebrosidase by enzyme assay
  • The patient has been genotyped or will have genotyping performed within 3 months of study enrollment.
  • The patient has been treated with Cerezyme for at least 2 years prior to study enrollment.
  • The patient has been on a stable dose of between 20-60 U/kg every 2 weeks for at least 6 months prior to study enrollment.
  • The patient is at least 18 years old.
  • The patient has a hemoglobin value of ≥ 11.0 g/dL for women and ≥ 12.0 g/dL for men and a platelet count of ≥ 100,000 mm^3.
  • The patient's liver volume is ≤ 1.8 x normal confirmed by MRI or CT within 6 months of randomization.
  • The patient's spleen volume is ≤ 10 x normal confirmed by MRI or CT within 6 months of randomization.
  • The patient has a serum creatinine < 2.0 mg/dL, an ASTand ALT < 2 x upper limit of normal and a total bilirubin < 2.0 x upper limit of normal.
  • Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to randomization into the study.

Exclusion Criteria:

  • The patient is pregnant.
  • The patient has evidence of neurologic or pulmonary involvement with Gaucher disease confirmed by medical history.
  • The patient has evidence of current or prior bleeding varices or liver infarction requiring hospitalization confirmed by medical history.
  • The patient has evidence of pathologic bone fractures, medullary infarctions, lytic lesions or avascular necrosis secondary to Gaucher disease confirmed by skeletal evaluation within 6 months of randomization.
  • The patient has had a bone crisis (defined as pain with acute onset which requires immobilization of the affected area, narcotics for relief of pain and may be accompanied by periosteal elevation, increased white cell count, fever or debilitation of > 3 days) within 12 months of randomization.
  • Patient has received an investigational drug within 30 days of the start of their participation in this trial. Patients may not receive any other investigational product throughout the course of the study.
  • The patient has a clinically significant disease (with the exception of symptoms relating to Gaucher disease), including clinically significant cardiovascular, hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival
  • Patient has a medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00364858

  Show 26 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Edward Kaye, M.D. Genzyme, a Sanofi Company
  More Information

Additional Information:
No publications provided

Responsible Party: Medical Monitor, Genzyme Coporation
ClinicalTrials.gov Identifier: NCT00364858     History of Changes
Other Study ID Numbers: CZ-011-01
Study First Received: August 15, 2006
Results First Received: May 28, 2009
Last Updated: September 3, 2009
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme, a Sanofi Company:
Type 1 Gaucher Disease
Glucocerebrosidase Deficiency Disease

Additional relevant MeSH terms:
Deficiency Diseases
Gaucher Disease
Lipidoses
Malnutrition
Nutrition Disorders
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on August 27, 2014