Ignite Creation Date:
2025-12-25 @ 4:17 AM
Ignite Modification Date:
2025-12-26 @ 3:17 AM
Study NCT ID:
NCT00048620
Status:
COMPLETED
Last Update Posted:
2006-11-03
First Post:
2002-11-04
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009087', 'term': 'Mucopolysaccharidosis VI'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020051', 'term': 'N-Acetylgalactosamine-4-Sulfatase'}], 'ancestors': [{'id': 'D001192', 'term': 'Arylsulfatases'}, {'id': 'D013429', 'term': 'Sulfatases'}, {'id': 'D004950', 'term': 'Esterases'}, {'id': 'D006867', 'term': 'Hydrolases'}, {'id': 'D004798', 'term': 'Enzymes'}, {'id': 'D045762', 'term': 'Enzymes and Coenzymes'}, {'id': 'D002811', 'term': 'Chondroitinsulfatases'}, {'id': 'D002810', 'term': 'Chondroitinases and Chondroitin Lyases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2000-09'}, 'statusVerifiedDate': '2006-11', 'completionDateStruct': {'date': '2005-11'}, 'lastUpdateSubmitDate': '2006-11-02', 'studyFirstSubmitDate': '2002-11-04', 'studyFirstSubmitQcDate': '2002-11-05', 'lastUpdatePostDateStruct': {'date': '2006-11-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2002-11-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Lysosomal storage disease'}]}, 'conditionsModule': {'conditions': ['Mucopolysaccharidosis VI']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.bmrn.com', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patient consent\n* Patient must be five years of age or older\n* Patient must have documented diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI\n* Leukocyte ASB enzyme activity level less than 20% of the normal range\n* Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.\n* Ability to perform all protocol tests\n* Ability to stand independently for six minutes\n* Sexually active subjects must agree to use an adequate form of contraception\n\nExclusion Criteria:\n\n* History of bone marrow transplantation\n* Pregnant or lactating patient\n* Use of an investigational drug or device within 30 days prior to study participation.\n* A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up\n* Known hypersensitivity to rhASB or to components of the study drug\n* History of cancer (except low grade and fully resolved skin malignancy)'}, 'identificationModule': {'nctId': 'NCT00048620', 'briefTitle': 'Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'Double-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI', 'orgStudyIdInfo': {'id': 'ASB-00-01'}}, 'armsInterventionsModule': {'interventions': [{'name': 'N-acetylgalactosamine 4-sulfatase', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '94949', 'city': 'Novato', 'state': 'California', 'country': 'United States', 'facility': 'BioMarin Pharmaceutical Inc.', 'geoPoint': {'lat': 38.10742, 'lon': -122.5697}}], 'overallOfficials': [{'name': 'Stuart J Swiedler, MD, Ph.D.', 'role': 'STUDY_DIRECTOR', 'affiliation': 'BioMarin Pharmaceutical'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}}}}