Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
| Status: | Completed |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 10/6/2018 |
| Start Date: | June 2008 |
| End Date: | August 2013 |
A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
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.
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.
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.
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.
Inclusion Criteria:
- Successful completion of Protocol PB-06-001
- The patient signs informed consent
Exclusion Criteria:
- 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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