Ignite Creation Date:
2025-12-24 @ 2:06 PM
Ignite Modification Date:
2026-02-24 @ 2:29 PM
Study NCT ID:
NCT00787995
Status:
TERMINATED
Last Update Posted:
2014-12-08
First Post:
2008-11-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}], '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': '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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 353}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2008-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-12', 'completionDateStruct': {'date': '2014-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-12-05', 'studyFirstSubmitDate': '2008-11-06', 'studyFirstSubmitQcDate': '2008-11-06', 'lastUpdatePostDateStruct': {'date': '2014-12-08', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-11-10', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Endurance', 'timeFrame': 'Study Visit'}, {'measure': 'Respiratory Function', 'timeFrame': 'Study Visit'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Mucopolysaccharidosis IV type A', 'MPS IV Type A', 'Mucopolysaccharidosis IVA', 'MPS IVA', 'Morquio A Syndrome', 'Lysosomal Storage Disorder', 'LSD', 'N-acetylgalactosamine-6-sulfatase', 'galactose-6-sulfatase', 'GALNS', 'enzyme replacement therapy', 'ERT'], 'conditions': ['MPS IV A', 'Mucopolysaccharidosis IVA', 'Morquio A Syndrome']}, 'referencesModule': {'references': [{'pmid': '21877895', 'type': 'DERIVED', 'citation': 'Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.'}], 'seeAlsoLinks': [{'url': 'http://www.bmrn.com', 'label': 'BioMarin Pharmaceutical Inc. website'}]}, 'descriptionModule': {'briefSummary': 'This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Subjects diagnosed with MPS IVA', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Willing and able to provide written, signed informed consent, or in the case of subjects age \\< 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.\n* Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.\n* Willing and able to comply with all study procedures.\n\nExclusion Criteria:\n\n* Use of any investigational product or investigational medical device within 30 days prior to screening.\n* Previous hematopoietic stem cell transplant (HSCT).\n* Concurrent disease or condition that would interfere with study participation or pose a safety concern.'}, 'identificationModule': {'nctId': 'NCT00787995', 'briefTitle': 'A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)', 'orgStudyIdInfo': {'id': 'MOR-001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'MPS IVA', 'description': 'Subjects with mucopolysaccharidosis IVA (Morquio Syndrome)'}]}, 'contactsLocationsModule': {'locations': [{'city': 'Oakland', 'state': 'California', 'country': 'United States', 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'city': 'Córdoba', 'country': 'Argentina', 'geoPoint': {'lat': -31.40648, 'lon': -64.18853}}, {'city': 'Porto Alegre', 'country': 'Brazil', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}, {'city': 'São Paulo', 'country': 'Brazil', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'city': 'Lyon', 'country': 'France', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Paris', 'country': 'France', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Mainz', 'country': 'Germany', 'geoPoint': {'lat': 49.98185, 'lon': 8.28008}}, {'city': 'Monza', 'country': 'Italy', 'geoPoint': {'lat': 45.58005, 'lon': 9.27246}}, {'city': 'Amsterdam', 'country': 'Netherlands', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'city': 'Taipei', 'country': 'Taiwan', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'city': 'Birmingham', 'country': 'United Kingdom', 'geoPoint': {'lat': 52.48142, 'lon': -1.89983}}, {'city': 'London', 'country': 'United Kingdom', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Manchester', 'country': 'United Kingdom', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'overallOfficials': [{'name': 'Celeste Decker, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'BioMarin Pharmaceutical'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}