A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
NCT00376168
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- 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.
- 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.
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Descriptive Information | |||
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Brief Title ICMJE | A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease | ||
Official Title ICMJE | 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 | ||
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 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. | ||
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. | ||
Study Type ICMJE | Interventional | ||
Study Phase ICMJE | Phase 3 | ||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment | ||
Condition ICMJE | Gaucher Disease | ||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||
Recruitment Information | |||
Recruitment Status ICMJE | Completed | ||
Actual Enrollment ICMJE | 32 | ||
Original Enrollment ICMJE | 30 | ||
Actual Study Completion Date ICMJE | October 2009 | ||
Actual Primary Completion Date | September 2009 (Final data collection date for primary outcome measure) | ||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||
Accepts Healthy Volunteers ICMJE | No | ||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||
Listed Location Countries ICMJE | Canada, Chile, Israel, Italy, South Africa, Spain, United Kingdom, United States | ||
Removed Location Countries | |||
Administrative Information | |||
NCT Number ICMJE | NCT00376168 | ||
Other Study ID Numbers ICMJE | PB-06-001 | ||
Has Data Monitoring Committee | Yes | ||
U.S. FDA-regulated Product | Not Provided | ||
IPD Sharing Statement ICMJE | Not Provided | ||
Responsible Party | Pfizer | ||
Study Sponsor ICMJE | Pfizer | ||
Collaborators ICMJE | Not Provided | ||
Investigators ICMJE | Not Provided | ||
PRS Account | Pfizer | ||
Verification Date | September 2018 | ||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |