Phase 3
Completed N=44
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Source: ClinicalTrials.gov NCT00705939 ↗Enrolled (actual)
44
Serious AEs
15.9%
Results posted
Jul 2014
Primary outcomePrimary: Spleen Volume — 2324.0; 2120.0; 778.0; 1707.7 mL
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Spleen Volume |
2324.0; 2120.0; 778.0; 1707.7; 1267.9; 883.7 | — |
| SECONDARY Liver Volume |
2999.7; 2470.5; 1775.7; 2515.6; 2118.7; 1788.9 | — |
| SECONDARY Hemoglobin |
12.5; 11.4; 13.6; 14.2; 13.6; 13.6 | — |
| SECONDARY Platelet Count |
64900; 69043; 163833; 80325; 122857; 145250 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00705939). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.