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Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
This study has been completed.
First Received: August 15, 2006   Last Updated: September 3, 2009   History of Changes
Sponsor: Genzyme
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00364858
  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 IV

Study Type: Interventional
Study Design: Treatment, Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study
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:

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

  Hide Study Locations
Locations
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
University Research Foundation for Lysosomal Storage Disease, Inc.
Coral Springs, Florida, United States, 33065
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Midwest Cancer Research Group, Inc.
Skokie, Illinois, United States, 60076
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, New Jersey
Institute for Genetics Medicine Saint Peter's University Hospital
New Brunswick, New Jersey, United States, 08903
Holy Name Hospital
Teaneck, New Jersey, United States, 07666
United States, New York
Hemophilia Center of Western New York
Buffalo, New York, United States, 14215
New York Oncology/Hematology PC
Latham, New York, United States, 12110
New York University
New York, New York, United States, 10016
Mt. Sinai Medical Center
New York, New York, United States, 10029
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Ohio
Children's Hospital Research Foundation
Cincinnati, Ohio, United States, 45229
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15261
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
Brazil
Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
Rio de Janeiro, Brazil, 20211
Canada, Ontario
Mount Sinai Hospital
Toronto, Ontario, Canada, ON M5G 1X5
Italy
Universita degli Studi di Napoli "Federico II"
Naples, Italy, 80131
Istituto per l'Infanzia Burlo-Garofolo
Trieste, Italy, 34137
Istituto Giannina Gaslini
Genova, Italy
Poland
Instytut Pomnik Centrum Zdrowia Dzeicka
Warsaw, Poland, 04-736
Spain
Hospital Vall d´Hebrón
Barcelona, Spain, 08035
United Kingdom
Royal Free Hospital
London, United Kingdom, NW3 2QG
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Edward Kaye, M.D. Genzyme
  More Information

Additional Information:
No publications provided

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

Keywords provided by Genzyme:
Type 1 Gaucher Disease
Glucocerebrosidase Deficiency Disease

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

ClinicalTrials.gov processed this record on November 27, 2009