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Sponsored by: |
Shire Human Genetic Therapies, Inc. |
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Information provided by: | Shire Human Genetic Therapies, Inc. |
ClinicalTrials.gov Identifier: | NCT00478647 |
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in patients with type 1 Gaucher disease who were previously treated with imiglucerase.
Condition | Intervention | Phase |
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Gaucher Disease, Type 1 |
Biological: velaglucerase alfa, (Gene-Activated® human glucocerebrosidase) |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase |
Estimated Enrollment: | 40 |
Study Start Date: | July 2007 |
Estimated Study Completion Date: | July 2009 |
Estimated Primary Completion Date: | July 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental |
Biological: velaglucerase alfa, (Gene-Activated® human glucocerebrosidase)
IV infusion, 15 to 60 U/kg every other week
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Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme.
GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the safety of GA-GCB in men, women, and children with Type 1 Gaucher disease who were previously treated with imiglucerase. Each patient's duration of treatment will be 12 months.
Ages Eligible for Study: | 2 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Includes:
Exclusion Criteria:
Includes:
United States, California | |
Children's Hospital Oakland | |
Oakland, California, United States, 94609 | |
Regional Metabolic Center | |
Los Angeles, California, United States, 90027 | |
United States, Georgia | |
Emory University | |
Decatur, Georgia, United States, 30033 | |
United States, Illinois | |
Feinberg School of Medicine | |
Chicago, Illinois, United States, 60614 | |
United States, Minnesota | |
Children's of Minnesota | |
Minneapolis, Minnesota, United States, 55404 | |
United States, Missouri | |
Children's Mercy Hospital and Clinic | |
Kansas City, Missouri, United States, 64108 | |
United States, New York | |
NYU School of Medicine | |
New York, New York, United States, 10016 | |
United States, Ohio | |
Cincinatti Children's Hospital | |
Cincinnati, Ohio, United States, 45229 | |
United States, Texas | |
Texas Children's Hospital | |
Houston, Texas, United States, 77030 | |
United States, Utah | |
Medical Genetics/Pediatrics | |
Salt Lake City, Utah, United States, 84132 | |
United States, Wisconsin | |
Children's Hospital of Wisconsin | |
Milwaukee, Wisconsin, United States, 53226 | |
Israel | |
Shaare Zedek Medical Center | |
Jerusalem, Israel | |
Poland | |
Children's Memorial Health Institute | |
Warszawa, Poland | |
Spain | |
Hospital Universitario Miguel Servet | |
Zaragoza, Spain, 500009 | |
United Kingdom | |
The Royal Free Hospital | |
London, United Kingdom |
Study Director: | Eric Crombez, M.D. | Shire Human Genetic Therapies, Inc. |
Responsible Party: | Shire Human Genetic Therapies, Inc. ( Tiffany Crump, Senior Medical Affairs Associate ) |
Study ID Numbers: | TKT034 |
Study First Received: | May 23, 2007 |
Last Updated: | July 31, 2008 |
ClinicalTrials.gov Identifier: | NCT00478647 History of Changes |
Health Authority: | United States: Food and Drug Administration; Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; Israel: Ministry of Health; United Kingdom: Medicines and Healthcare Products Regulatory Agency; Spain: Spanish Agency of Medicines |
Enzyme Replacement Therapy Gaucher disease glucocerebrosidase beta-glucocerebrosidase Acid beta-glucocerebrosidase |
glucosylceramidase D-glucosyl-N-acylsphingosine glucohydrolase gene activation human |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Lysosomal Storage Diseases Sphingolipidosis Central Nervous System Diseases Brain Diseases Lymphatic Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Metabolic Disorder Lipid Metabolism Disorders Brain Diseases, Metabolic |
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |