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A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
This study has been completed.
Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00376168
First received: September 12, 2006
Last updated: July 25, 2012
Last verified: July 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 the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
| Condition | Intervention | Phase |
|---|---|---|
| Gaucher Disease | Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD) | Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy 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:
Gaucher disease
MedlinePlus related topics:
Gaucher Disease
U.S. FDA Resources
Further study details as provided by Protalix:
Primary Outcome Measures:
- Change From Baseline in Spleen Volume Measured by MRI. [ Time Frame: Baseline and 9 months ]Calculated as percent change in spleen volume from Baseline to 9 months
Secondary Outcome Measures:
- Change From Baseline in Liver Volume [ Time Frame: Baseline and 9 months ]Calculated as percent change in liver volume from Baseline to 9 months
- Change in Hemoglobin [ Time Frame: Baseline and Month 9 ]Absolute change in Hemoglobin concentration from Baseline to Month 9
- Change in Platelet Count [ Time Frame: Baseline and Month 9 ]Change in Platelet count from Baseline to Month 9
Other Outcome Measures:
- Change in Chitotriosidase [ Time Frame: Baseline and Month 9 ]Change in Chitotriosidase from Baseline to Month 9
| Enrollment: | 32 |
| Study Start Date: | August 2007 |
| Study Completion Date: | October 2009 |
| Primary Completion Date: | September 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: prGCD 30 Units/kg |
Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Intravenous infusion every two weeks for 9 months
Other Name: Taliglucerase alfa
|
| Experimental: prGCD 60 Units/kg |
Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Intravenous infusion every 2 weeks for 9 months
Other Name: Taliglucerase alfa
|
Detailed Description:
This will be a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in 30 untreated patients with Gaucher disease. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the study will be nine months. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.
There will be two treatment groups, 15 patients in each treatment group.
Treatment Group I: 30 units/kg every 2 weeks. Treatment Group II: 60 units/kg every 2 weeks.
All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD.
Eligibility| Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Males and females, 18 years or older
- Confirmed enzymatic diagnosis of Gaucher disease
- Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
- Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
- Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
- Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
- Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
- Ability to provide a written informed consent.
Exclusion Criteria:
- Currently taking another experimental drug for any condition
- Pregnant or nursing
- Presence of HIV and/or, HBsAg and/or hepatitis C infections
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
- Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
- History of allergy to carrots.
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00376168
Please refer to this study by its ClinicalTrials.gov identifier: NCT00376168
Locations
| United States, Florida | |
| University Research Foundation for Lysosomal Storage Diseases | |
| Coral Springs, Florida, United States, 33065 | |
| United States, Georgia | |
| Division of Medical Genetics, Emory University School of Medicine | |
| Decatur, Georgia, United States, 30033 | |
| United States, New York | |
| New York University Medical Center | |
| New York, New York, United States, 10016 | |
| 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, 91031 | |
| Italy | |
| Universita "La Sapienza" | |
| Rome, Italy, 00161 | |
| South Africa | |
| Morningside Medi-Clinic | |
| Morningside, South Africa, 2196 | |
| Spain | |
| Hospital Universitario Miguel Servet | |
| Zaragoza, Spain, 50009 | |
| 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
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00376168 History of Changes |
| Other Study ID Numbers: |
PB-06-001 |
| Study First Received: | September 12, 2006 |
| Results First Received: | May 11, 2012 |
| Last Updated: | July 25, 2012 |
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 July 17, 2017