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| Sponsor: | Shire Human Genetic Therapies, Inc. |
|---|---|
| Information provided by: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00635427 |
Purpose
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 every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Biological: velaglucerase alfa |
Phase III |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease |
| Estimated Enrollment: | 102 |
| Study Start Date: | May 2008 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | December 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: velaglucerase alfa
VPRIV GA-GCB Gene activated human glucocerebrosidase
|
Biological: velaglucerase alfa
IV infusion, 15 to 60 U/kg every other week for the duration of the study
Other Names:
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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 long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations
Show 21 Study Locations| Study Director: | Eric Crombez, M.D. | Shire Human Genetic Therapies, Inc. |
More Information
| Responsible Party: | Tiffany Crump, Senior Medical Affairs Associate, Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00635427 History of Changes |
| Other Study ID Numbers: | HGT-GCB-044 |
| Study First Received: | March 6, 2008 |
| Last Updated: | August 4, 2011 |
| Health Authority: | United States: Food and Drug Administration; Paraguay: Ministerio de Salud Pública y Bienestar Social; Israel: Ministry of Health; Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica; Poland: Ministry of Health; United Kingdom: Medicines and Healthcare Products Regulatory Agency; India: Drugs Controller General of India; South Korea: Korea Food and Drug Administration (KFDA); Russia: Ministry of Health and Social Development of the Russian Federation; Spain: Ministry of Health and Consumption; Tunisia: Ministry of Public Health |
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VPRIV Enzyme Replacement Therapy Gaucher disease glucocerebrosidase beta-glucocerebrosidase |
Acid beta-glucocerebrosidase glucosylceramidase D-glucosyl-N-acylsphingosine glucohydrolase gene activation human |
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Gaucher Disease 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 Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |