Status:
COMPLETED
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Lead Sponsor:
Pfizer
Conditions:
Gaucher Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief 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...
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 receiv...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2009
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT00376168
Start Date
August 1 2007
End Date
October 1 2009
Last Update
October 4 2018
Active Locations (11)
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1
University Research Foundation for Lysosomal Storage Diseases
Coral Springs, Florida, United States, 33065
2
Division of Medical Genetics, Emory University School of Medicine
Decatur, Georgia, United States, 30033
3
New York University Medical Center
New York, New York, United States, 10016
4
Mount Sinai Hospital
Toronto, Ontario, Canada, M5G 1X5