An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
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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 3 |
| Study Type: | Interventional |
| Study Design: | 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 |
- Evaluation of safety assessments [ Time Frame: Duration of the study ] [ Designated as safety issue: Yes ]
- The evaluation of hematological parameters and organomegaly [ Time Frame: Duration of the study ] [ Designated as safety issue: Yes ]
| Enrollment: | 95 |
| Study Start Date: | May 2008 |
| Study Completion Date: | December 2012 |
| Primary Completion Date: | December 2012 (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:
|
Detailed Description:
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:
- The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
- Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.
- Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
- The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
- The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
Exclusion Criteria:
- The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.
- The patient is pregnant or lactating.
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
- The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).
- The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator
Contacts and Locations
Show 21 Study Locations| Study Director: | Eric Crombez, M.D. | Shire Human Genetic Therapies, Inc. |
More Information
No publications provided
| Responsible Party: | 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: | January 24, 2013 |
| 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 of the Russian Federation Spain: Ministry of Health and Consumption Tunisia: Ministry of Public Health |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
VPRIV Enzyme Replacement Therapy Gaucher disease glucocerebrosidase beta-glucocerebrosidase |
Acid beta-glucocerebrosidase glucosylceramidase D-glucosyl-N-acylsphingosine glucohydrolase gene activation human |
Additional relevant MeSH terms:
|
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 |
ClinicalTrials.gov processed this record on May 23, 2013