A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Information source: Protalix
Information obtained from ClinicalTrials.gov on November 03, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease
Intervention: Human Glucocerebrosidase (prGCD) (Drug)
Phase: Phase 3
Status: Recruiting
Sponsored by: Protalix Official(s) and/or principal investigator(s):
Ari Zimran, MD, Principal Investigator, Affiliation: Shaare Zedek Medical Center, Jerusalem, Israel
Overall contact:
Raul Chertkoff, MD, Phone: +972-4-9889488, Email: raul@protalix.com
Summary
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.
Clinical Details
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
Study design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Dose Comparison, Parallel Assignment, Safety/Efficacy Study
Primary outcome: Change from baseline in spleen volume measured by MRI.
Secondary outcome: Change from baseline in: Liver volume, Hemoglobin measurement, Platelet count, Biomarkers (chitotriosidase and pulmonary and activation-regulated chemokine (PARC/ CCL18)
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
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
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.
Locations and Contacts
Raul Chertkoff, MD, Phone: +972-4-9889488, Email: raul@protalix.com
Shaare Zedek Medical Center, Jerusalem 91031, Israel; Recruiting
Ari Zimran, MD
Ari Zimran, MD, Principal Investigator
Morningside Medi-Clinic, Morningside 2196, South Africa; Recruiting
Rene Heitner, MD, Phone: +27-11-883-3408
Rene Heitner, MD, Principal Investigator
Royal Free Hospital, London NW3 2QG, United Kingdom; Recruiting
Atul Mehta, MD, Phone: 44-2078302814, Email: atul.mehta@royalfree.nhs.uk
Atul Mehta, MD, Principal Investigator
University Research Foundation for Lysosomal Storage Diseases, Coral Springs, Florida 33065, United States; Recruiting
Neal J Weinreb, MD, Phone: 954-755-1904, Email: bonneal@hotmail.com
Neil J Weinreb, MD, Principal Investigator
Division of Medical Genetics, Emory University School of Medicine, Decatur, Georgia 30033, United States; Recruiting
Paul M Fernhoff, MD, Phone: 404-778-8528, Email: pferhoff@genetics.emory.edu
Paul M Ferhnoff, MD, Principal Investigator
New York University Medical Center, New York, New York 10016, United States; Recruiting
Gregory M Pastores, MD, Phone: 212-263-1018
Michelle Ford, Phone: 212-263-1018
Gregory M Pastores, MD, Principal Investigator
Additional Information
Starting date: August 2007
Ending date: May 2009
Last updated: June 25, 2008
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