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| Sponsor: | Shire Human Genetic Therapies, Inc. |
|---|---|
| Information provided by (Responsible Party): | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00391625 |
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 enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Drug: GA-GCB |
Phase I Phase II |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
| Study Completion Date: | December 2011 |
| Primary Completion Date: | December 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: GA-GCB
15-60 U/kg every other week via intravenous infusion
|
Drug: GA-GCB
15-60 U/kg every other week via intravenous infusion
Other Name: VPRIV®, velaglucerase alfa, gene-activated® glucocerebrosidase,DRX008
|
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 (the long term safety of enzyme replacement therapy with DRX008A (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
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| Israel | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel | |
| 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: | Maja Djordjevic, M.D. | Mother and Child Health Care Institute of Serbia |
More Information
| Responsible Party: | Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00391625 History of Changes |
| Other Study ID Numbers: | TKT025EXT |
| Study First Received: | October 20, 2006 |
| Last Updated: | February 6, 2012 |
| 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 |
|
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 |