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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: | NCT00391625 |
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 long term safety of enzyme replacement therapy with DRX008A (GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.
Condition | Intervention | Phase |
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Gaucher Disease, Type 1 |
Biological: Gene-Activated® human glucocerebrosidase (GA-GCB) |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy |
Enrollment: | 9 |
Study Start Date: | April 2005 |
Estimated Study Completion Date: | April 2009 |
Estimated Primary Completion Date: | April 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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GA-GCB: Experimental |
Biological: Gene-Activated® human glucocerebrosidase (GA-GCB)
IV infusion, 30 or 60 U/kg every other week for the duration of the study
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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 (velaglucerase alfa) contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Israel | |
Shaare Zedek Medical Center | |
Jerusalem, Israel | |
Romania | |
Maria Sklodwska Curie Children's Hospital | |
Bucharest, Romania, 75544 | |
Serbia | |
Mother and Child Health Care Institute of Serbia | |
Belgrade, Serbia |
Principal Investigator: | Ari Zimran, M.D. | Gaucher Clinic, Shaare Zedek Medical Center |
Principal Investigator: | Florea Iordachescu, MD | Maria Sklodowska Curie Children's Hospital |
Principal Investigator: | Maja Djordjevic, M.D. | Mother and Child Health Care Institute of Serbia |
Responsible Party: | Shire Human Genetic Therapies, Inc. ( Tiffany Crump, Senior Medical Affairs Associate ) |
Study ID Numbers: | TKT025EXT |
Study First Received: | October 20, 2006 |
Last Updated: | July 31, 2008 |
ClinicalTrials.gov Identifier: | NCT00391625 |
Health Authority: | United States: Food and Drug Administration; Israel: Israeli Health Ministry Pharmaceutical Administration; Romania: State Institute for Drug Control; Serbia and Montenegro: Agency for Drugs and Medicinal Devices |
Gaucher disease, Enzyme Replacement Therapy |
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 Metabolic disorder Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Nervous System Diseases |