Ignite Creation Date:
2025-12-25 @ 1:20 AM
Ignite Modification Date:
2025-12-25 @ 11:29 PM
Study NCT ID:
NCT00004293
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
1999-10-18
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005776', 'term': 'Gaucher Disease'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}], 'ancestors': [{'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': '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'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005962', 'term': 'Glucosylceramidase'}], 'ancestors': [{'id': 'D005959', 'term': 'Glucosidases'}, {'id': 'D006026', 'term': 'Glycoside Hydrolases'}, {'id': 'D006867', 'term': 'Hydrolases'}, {'id': 'D004798', 'term': 'Enzymes'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'primaryPurpose': 'TREATMENT'}, 'enrollmentInfo': {'count': 24}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '1999-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2003-12', 'lastUpdateSubmitDate': '2005-06-23', 'studyFirstSubmitDate': '1999-10-18', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2005-06-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ["Gaucher's disease", 'inborn errors of metabolism', 'rare disease', 'sphingolipidoses'], 'conditions': ["Gaucher's Disease"]}, 'descriptionModule': {'briefSummary': 'OBJECTIVES:\n\nI. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.', 'detailedDescription': 'PROTOCOL OUTLINE:\n\nPatients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay At least 3 organ systems affected, based on the following criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary compromise Symptoms compromise daily activities or risk longevity No neurologic disease'}, 'identificationModule': {'nctId': 'NCT00004293', 'briefTitle': 'Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease', 'organization': {'class': 'NIH', 'fullName': 'National Center for Research Resources (NCRR)'}, 'orgStudyIdInfo': {'id': '199/11725'}, 'secondaryIdInfos': [{'id': 'UPITTS-M1230'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'glucocerebrosidase', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '15260', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'John Barranger', 'role': 'CONTACT', 'phone': '412-624-4623'}], 'facility': 'University of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}], 'overallOfficials': [{'name': 'John Barranger', 'role': 'STUDY_CHAIR', 'affiliation': 'University of Pittsburgh'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'National Center for Research Resources (NCRR)', 'class': 'NIH'}, 'collaborators': [{'name': 'University of Pittsburgh', 'class': 'OTHER'}]}}}