Ignite Creation Date:
2025-12-24 @ 5:28 PM
Ignite Modification Date:
2025-12-26 @ 3:03 PM
Study NCT ID:
NCT00376168
Status:
COMPLETED
Last Update Posted:
2018-10-04
First Post:
2006-09-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005776', 'term': 'Gaucher Disease'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C582473', 'term': 'taliglucerase alfa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'einata@protalix.com', 'phone': '972-4-9889488', 'title': 'Einat Almon', 'phoneExt': '137', 'organization': 'Protalix Ltd.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': '9 months', 'eventGroups': [{'id': 'EG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks', 'otherNumAtRisk': 16, 'otherNumAffected': 6, 'seriousNumAtRisk': 16, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks', 'otherNumAtRisk': 16, 'otherNumAffected': 4, 'seriousNumAtRisk': 16, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Feeling hot', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}, {'term': 'Glycosuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 16, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 16, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 10.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Spleen Volume Measured by MRI.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'OG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '-26.91', 'spread': '7.79', 'groupId': 'OG000'}, {'value': '-38.01', 'spread': '9.38', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'The primary efficacy analysis was based on two one-sample t-tests (one for each treatment group) to determine if the percent change in spleen volume is different than zero.\n\nWith 12 patients in each treatment group, there was greater than 95% power to detect a change of 20% or more using a one-sample t-test (alpha=0.025, 2-sided test to allow for each group to be tested separately) to evaluate the primary outcome of percent change in spleen volume after nine months.', 'statisticalMethod': 'one-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'one-sample t-test (combined) to test the null hypothesis that percent change = 0'}, {'pValue': '<0.0001', 'groupIds': ['OG001'], 'ciPctValue': '95', 'pValueComment': 'threshold for statistical significance = 0.025', 'groupDescription': 'The primary efficacy analysis was based on two one-sample t-tests (one for each treatment group) to determine if the percent change in spleen volume is different than zero.\n\nWith 12 patients in each treatment group, there was greater than 95% power to detect a change of 20% or more using a one-sample t-test (alpha=0.025, 2-sided test to allow for each group to be tested separately) to evaluate the primary outcome of percent change in spleen volume after nine months.', 'statisticalMethod': 'one-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'statisticalComment': 'one-sample t-test (combined) to test the null hypothesis that percent change = 0'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and 9 months', 'description': 'Calculated as percent change in spleen volume from Baseline to 9 months', 'unitOfMeasure': 'percentage of change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Liver Volume', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'OG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '-10.48', 'spread': '11.27', 'groupId': 'OG000'}, {'value': '-11.11', 'spread': '6.68', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0041', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '<0.0001', 'groupIds': ['OG001'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and 9 months', 'description': 'Calculated as percent change in liver volume from Baseline to 9 months', 'unitOfMeasure': 'percentage of change from baseline', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat'}, {'type': 'SECONDARY', 'title': 'Change in Hemoglobin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'OG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '1.6', 'spread': '1.4', 'groupId': 'OG000'}, {'value': '2.2', 'spread': '1.4', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0010', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '<0.0001', 'groupIds': ['OG001'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Month 9', 'description': 'Absolute change in Hemoglobin concentration from Baseline to Month 9', 'unitOfMeasure': 'g/dL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat'}, {'type': 'SECONDARY', 'title': 'Change in Platelet Count', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}, {'value': '16', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'OG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '11,427', 'spread': '20,214', 'groupId': 'OG000'}, {'value': '41,494', 'spread': '47,063', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0460', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}, {'pValue': '0.0031', 'groupIds': ['OG001'], 'ciPctValue': '95', 'groupDescription': 'For each of the secondary endpoints, a one-sample t-test (% change in liver volume, mean change in hemoglobin and platelet count) was examined first for each dose using the step-down approach. Next, a mixed effects model that included dose and time, with subject as a random effect, was fit to examine whether there was a difference between dose groups, for those outcomes that were tested in the step-down approach.', 'statisticalMethod': 'One-sample t-test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Month 9', 'description': 'Change in Platelet count from Baseline to Month 9', 'unitOfMeasure': 'count/mm^3', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Change in Chitotriosidase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'OG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'classes': [{'categories': [{'measurements': [{'value': '-14,548', 'spread': '8,025.8', 'groupId': 'OG000'}, {'value': '-12,538', 'spread': '14,489', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Month 9', 'description': 'Change in Chitotriosidase from Baseline to Month 9', 'unitOfMeasure': 'nmol/ml/hr', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Intent to treat'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'FG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'comment': 'One patient randomized but not treated', 'groupId': 'FG001', 'numSubjects': '16'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}, {'groupId': 'FG001', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '32', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'ELELYSO 30 Units/kg', 'description': 'Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks'}, {'id': 'BG001', 'title': 'ELELYSO 60 Units/kg', 'description': 'Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '31', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '36.3', 'spread': '11.8', 'groupId': 'BG000'}, {'value': '36.0', 'spread': '12.2', 'groupId': 'BG001'}, {'value': '36.2', 'spread': '11.8', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Canada', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Spain', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Chile', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'South Africa', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Israel', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Mexico', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'Serbia', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Spleen Volume', 'classes': [{'categories': [{'measurements': [{'value': '2130.94', 'spread': '1154.72', 'groupId': 'BG000'}, {'value': '2117.38', 'spread': '1356.17', 'groupId': 'BG001'}, {'value': '2120', 'spread': '1200', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Measured by MRI', 'unitOfMeasure': 'mL', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Liver Volume', 'classes': [{'categories': [{'measurements': [{'value': '2880.60', 'spread': '736.12', 'groupId': 'BG000'}, {'value': '2481.31', 'spread': '452.74', 'groupId': 'BG001'}, {'value': '2600', 'spread': '600', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Measured by MRI', 'unitOfMeasure': 'mL', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Hemoglobin', 'classes': [{'categories': [{'measurements': [{'value': '12.2', 'spread': '1.7', 'groupId': 'BG000'}, {'value': '11.4', 'spread': '2.6', 'groupId': 'BG001'}, {'value': '12', 'spread': '2', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Measured in central laboratory', 'unitOfMeasure': 'g/dL', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Platelet Count', 'classes': [{'categories': [{'measurements': [{'value': '75,320', 'spread': '40,861', 'groupId': 'BG000'}, {'value': '65,038', 'spread': '28,668', 'groupId': 'BG001'}, {'value': '70,000', 'spread': '32,000', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Measured in local laboratory', 'unitOfMeasure': 'count/mm^3', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Chitotriosidase', 'classes': [{'categories': [{'measurements': [{'value': '28158', 'spread': '11686', 'groupId': 'BG000'}, {'value': '24702', 'spread': '17428', 'groupId': 'BG001'}, {'value': '26000', 'spread': '14000', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'description': 'Gaucher disease biomarker', 'unitOfMeasure': 'nmol/ml/hr', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 32}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2007-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-09', 'completionDateStruct': {'date': '2009-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-09-05', 'studyFirstSubmitDate': '2006-09-12', 'resultsFirstSubmitDate': '2012-05-11', 'studyFirstSubmitQcDate': '2006-09-12', 'lastUpdatePostDateStruct': {'date': '2018-10-04', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2012-07-25', 'studyFirstPostDateStruct': {'date': '2006-09-14', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2012-07-26', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in Chitotriosidase', 'timeFrame': 'Baseline and Month 9', 'description': 'Change in Chitotriosidase from Baseline to Month 9'}], 'primaryOutcomes': [{'measure': 'Change From Baseline in Spleen Volume Measured by MRI.', 'timeFrame': 'Baseline and 9 months', 'description': 'Calculated as percent change in spleen volume from Baseline to 9 months'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Liver Volume', 'timeFrame': 'Baseline and 9 months', 'description': 'Calculated as percent change in liver volume from Baseline to 9 months'}, {'measure': 'Change in Hemoglobin', 'timeFrame': 'Baseline and Month 9', 'description': 'Absolute change in Hemoglobin concentration from Baseline to Month 9'}, {'measure': 'Change in Platelet Count', 'timeFrame': 'Baseline and Month 9', 'description': 'Change in Platelet count from Baseline to Month 9'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Gaucher Disease']}, 'referencesModule': {'references': [{'pmid': '26053270', 'type': 'DERIVED', 'citation': 'Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.'}, {'pmid': '21900191', 'type': 'DERIVED', 'citation': 'Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Munoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6.'}, {'pmid': '18729283', 'type': 'DERIVED', 'citation': "Winckler T. [Enzyme replacement therapy for Gaucher's Disease]. Pharm Unserer Zeit. 2008;37(5):352-3. doi: 10.1002/pauz.200890067. No abstract available. German."}]}, 'descriptionModule': {'briefSummary': '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.\n\nThis 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.', 'detailedDescription': '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.\n\nThere will be two treatment groups, 15 patients in each treatment group.\n\nTreatment Group I: 30 units/kg every 2 weeks. Treatment Group II: 60 units/kg every 2 weeks.\n\nAll patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males and females, 18 years or older\n* Confirmed enzymatic diagnosis of Gaucher disease\n* 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\n* Female patients of child-bearing potential who agree to use a medically acceptable method of contraception\n* 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).\n* 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.\n* Patients who have not received substrate reduction therapy (SRT) in the past 12 months.\n* Ability to provide a written informed consent.\n\nExclusion Criteria:\n\n* Currently taking another experimental drug for any condition\n* Pregnant or nursing\n* Presence of HIV and/or, HBsAg and/or hepatitis C infections\n* Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.\n* Previous anaphylactoid reaction to Cerezyme® or Ceredase®.\n* History of allergy to carrots.'}, 'identificationModule': {'nctId': 'NCT00376168', 'briefTitle': 'A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pfizer'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'PB-06-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'prGCD 30 Units/kg', 'interventionNames': ['Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)']}, {'type': 'EXPERIMENTAL', 'label': 'prGCD 60 Units/kg', 'interventionNames': ['Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)']}], 'interventions': [{'name': 'Plant cell expressed recombinant glucocerebrosidase (prGCD)', 'type': 'DRUG', 'otherNames': ['Taliglucerase alfa'], 'description': 'Intravenous infusion every two weeks for 9 months', 'armGroupLabels': ['prGCD 30 Units/kg']}, {'name': 'Plant cell expressed recombinant glucocerebrosidase (prGCD)', 'type': 'DRUG', 'otherNames': ['Taliglucerase alfa'], 'description': 'Intravenous infusion every 2 weeks for 9 months', 'armGroupLabels': ['prGCD 60 Units/kg']}]}, 'contactsLocationsModule': {'locations': [{'zip': '33065', 'city': 'Coral Springs', 'state': 'Florida', 'country': 'United States', 'facility': 'University Research Foundation for Lysosomal Storage Diseases', 'geoPoint': {'lat': 26.27119, 'lon': -80.2706}}, {'zip': '30033', 'city': 'Decatur', 'state': 'Georgia', 'country': 'United States', 'facility': 'Division of Medical Genetics, Emory University School of Medicine', 'geoPoint': {'lat': 33.77483, 'lon': -84.29631}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': 'M5G 1X5', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Mount Sinai Hospital', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Santiago', 'country': 'Chile', 'facility': 'Pontificia Universidad Catolica de Chile', 'geoPoint': {'lat': -33.45694, 'lon': -70.64827}}, {'zip': '31096', 'city': 'Haifa', 'country': 'Israel', 'facility': 'Rambam Medical Center', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'zip': '91031', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Shaare Zedek Medical Center', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '00161', 'city': 'Rome', 'country': 'Italy', 'facility': 'Universita "La Sapienza"', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '2196', 'city': 'Morningside', 'country': 'South Africa', 'facility': 'Morningside Medi-Clinic', 'geoPoint': {'lat': -29.82021, 'lon': 31.01736}}, {'zip': '50009', 'city': 'Zaragoza', 'country': 'Spain', 'facility': 'Hospital Universitario Miguel Servet', 'geoPoint': {'lat': 41.65606, 'lon': -0.87734}}, {'zip': 'NW3 2QG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pfizer', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}