Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
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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 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 1 Phase 2 |
| 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 |
- Evaluation of long term safety [ Time Frame: duration of study ] [ Designated as safety issue: Yes ]
- Evaluation of hematological parameters and organomegaly [ Time Frame: Duration of study ] [ Designated as safety issue: Yes ]
| 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
|
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 (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:
- Patients who have completed through Week 41 visit in the TKT025 study.
- Patients must have voluntarily signed an IRB/EC approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
- Patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
- Female and male patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Female patients must have a negative serum pregnancy test on enrollment.
Exclusion Criteria:
- Patient has received treatment with non-Gaucher disease related investigational drug or device within the past 30 days prior to study entry; such use during the study is not permitted.
- Patient has a clinically relevant medical condition (e.g., HIV, hepatitis B or C) that would make implementation of the protocol difficult and/or confound an assessment of the effects of the experimental therapy and its adverse events.
- Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope and possible consequences of the study.
- Patient is unable to comply with the protocol, e.g. uncooperative attitude, medical condition, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.
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
No publications provided by Shire Human Genetic Therapies, Inc.
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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 |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
Gaucher disease, Enzyme Replacement Therapy |
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