Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
NCT00705939
ABOUT THIS STUDY
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- Successful completion of Protocol PB-06-001
- The patient signs informed consent
- 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
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Descriptive Information | |||
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Brief Title ICMJE | Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial | ||
Official Title ICMJE | A Multicenter, Double-Blind, Extension Trial 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 an extension trial to Study NCT00376168 and NCT00712348. | ||
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. | ||
Study Type ICMJE | Interventional | ||
Study Phase ICMJE | Phase 3 | ||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment | ||
Condition ICMJE | Gaucher Disease | ||
Intervention ICMJE | Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks Other Names:
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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 | 45 | ||
Original Estimated Enrollment ICMJE | 25 | ||
Actual Study Completion Date ICMJE | August 2013 | ||
Actual Primary Completion Date | May 2012 (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 | Australia, Canada, Chile, Israel, South Africa, Spain, United Kingdom, United States | ||
Removed Location Countries | |||
Administrative Information | |||
NCT Number ICMJE | NCT00705939 | ||
Other Study ID Numbers ICMJE | PB-06-003 | ||
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 |