Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
This study is enrolling participants by invitation only.
Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00705939
First received: June 25, 2008
Last updated: January 18, 2012
Last verified: January 2012
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Purpose
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease |
Drug: Plant Cell Expressed Recombinant Human Glucocerebrosidase |
Phase 3 |
Access to an investigational treatment associated with this study is available outside the clinical trial. More info ...
| 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: | A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Farber lipogranulomatosis
Gaucher disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Gaucher's Disease
U.S. FDA Resources
Further study details as provided by Protalix:
Primary Outcome Measures:
- Spleen Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Liver Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 60 |
| Study Start Date: | June 2008 |
| Estimated Study Completion Date: | December 2012 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 1
Continue treatment from previous study
|
Drug: Plant Cell Expressed Recombinant Human Glucocerebrosidase
Intravenous infusion every 2 weeks
Other Name: prGCD
|
Detailed Description:
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Successful completion of Protocol PB-06-001
- The patient signs informed consent
Exclusion Criteria:
- Currently taking another experimental drug for any condition
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
- Pregnant or nursing
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00705939
Locations
| United States, Georgia | |
| Department of Human Genetics, Emory University School of Medicine | |
| Decatur, Georgia, United States, 30033 | |
| United States, New York | |
| Neurogenetics, NYU at Rivergate | |
| New York, New York, United States, 10016 | |
| Australia, Victoria | |
| Bone Marrow Transplant Service, The Royal Melbourne Hospital | |
| Parkville, Victoria, Australia | |
| Canada, Ontario | |
| Mount Sinai Hospital | |
| Toronto, Ontario, Canada, M5G 1X5 | |
| Chile | |
| Pontificia Universidad Catolica de Chile | |
| Santiago, Chile | |
| Israel | |
| Rambam Medical Center | |
| Haifa, Israel, 31096 | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel | |
| South Africa | |
| Morningside Medi-Clinic | |
| Morningside, South Africa, 2196 | |
| Spain | |
| Hospital Universitario Miguel Servet | |
| Zaragoza, Spain, 50009 | |
| United Kingdom | |
| Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust | |
| Cambridge, United Kingdom | |
| Royal Free Hospital | |
| London, United Kingdom, NW3 2QG | |
Sponsors and Collaborators
Protalix
Investigators
| Principal Investigator: | Ari Zimran, MD | Shaare Zedek Medical Center, Jerusalem, Israel |
More Information
No publications provided
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00705939 History of Changes |
| Other Study ID Numbers: | PB-06-003 |
| Study First Received: | June 25, 2008 |
| Last Updated: | January 18, 2012 |
| Health Authority: | United States: Food and Drug Administration Israel: Ministry of Health |
Keywords provided by Protalix:
|
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 June 18, 2013