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| 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 |
| 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 |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations
Hide Study 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 | |
| Study Director: | Edward Kaye, M.D. | Genzyme |
More Information
| 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 |
|
Type 1 Gaucher Disease Glucocerebrosidase Deficiency Disease |
|
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 |