Ignite Creation Date:
2025-12-25 @ 2:17 AM
Ignite Modification Date:
2026-03-08 @ 11:03 PM
Study NCT ID:
NCT02520934
Status:
UNKNOWN
Last Update Posted:
2019-03-29
First Post:
2015-08-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Miglustat on Gaucher Disease Type IIIB
Sponsor:
Organization:
Raw JSON
{'hasResults': False, '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': 'C059896', 'term': 'miglustat'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 19}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2015-07'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2019-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2019-03-28', 'studyFirstSubmitDate': '2015-08-06', 'studyFirstSubmitQcDate': '2015-08-11', 'lastUpdatePostDateStruct': {'date': '2019-03-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-08-13', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improve in Purdue Pegboard test speed', 'timeFrame': '24 months'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Enzyme Replacement Therapy', 'Miglustat', 'Gaucher Disease'], 'conditions': ['Gaucher Disease']}, 'descriptionModule': {'briefSummary': 'evaluate the combination therapy with Miglustat and enzyme replacement therapy (ERT) on Gaucher disease', 'detailedDescription': 'understand if Miglustat (glucosylceramide synthase inhibitor) could improve neuropathy in patients with Gaucher disease'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Case\\_Miglustat\n\nInclusion Criteria:\n\n1. Confirmed diagnosis of Gaucher Disease: blood test shown lack of beta- glucocerebrosidase, and found L444P homozygous on GBA gene.\n2. Aged 6 years old or above.\n3. Already have regular ERT (30-120 IU/kg/ every 2 weeks) at least a year; dosage and frequency of ERT had not been changed in recent 3 months.\n\nExclusion Criteria:\n\n1. History of tremor and abnormal extremities perception ( pain, numbness, tingle etc.)\n2. Abnormal kidney function.\n3. Pregnant or plan to have a baby ( potentially pregnant patient need to be transferred to gynecologist for the test and promise to have proper contraception measures).\n4. Allergic to Miglustat.\n\nControl\\_normal Inclusion Criteria\n\n1. Age 6-18 years\n2. No significant physical, mental, or psychiatric problems\n\nExclusion criteria\n\n1\\. Children with eye disease (not include myopia, hyperopia, Astigmatism)'}, 'identificationModule': {'nctId': 'NCT02520934', 'briefTitle': 'Miglustat on Gaucher Disease Type IIIB', 'organization': {'class': 'OTHER', 'fullName': 'National Taiwan University Hospital'}, 'officialTitle': 'Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB', 'orgStudyIdInfo': {'id': '201503076MIPD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Case_Miglustat', 'description': 'Besides regular ERT, patients in this group also need to take Miglustat for 24 months.', 'interventionNames': ['Drug: Miglustat', 'Drug: ERT']}, {'type': 'NO_INTERVENTION', 'label': 'Control', 'description': 'Patients will be tested for their pupil cycle time.'}], 'interventions': [{'name': 'Miglustat', 'type': 'DRUG', 'otherNames': ['Zavesca'], 'description': 'combine miglustat and ERT to see if neurologic manifestations can be improved', 'armGroupLabels': ['Case_Miglustat']}, {'name': 'ERT', 'type': 'DRUG', 'description': 'enzyme replacement therapy', 'armGroupLabels': ['Case_Miglustat']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10041', 'city': 'Taipei', 'country': 'Taiwan', 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}], 'overallOfficials': [{'name': 'Yin-Hsiu Chien, M.D., Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'National Taiwan University Hospital'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Taiwan University Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'Actelion', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}