Ignite Creation Date:
2025-12-24 @ 11:40 PM
Ignite Modification Date:
2026-01-26 @ 4:37 PM
Study NCT ID:
NCT02097251
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2017-02-06
First Post:
2014-03-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D016538', 'term': 'Mucopolysaccharidosis VII'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic 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': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000654126', 'term': 'vestronidase alfa'}]}}, 'protocolSection': {'designModule': {'studyType': 'EXPANDED_ACCESS'}, 'statusModule': {'overallStatus': 'NO_LONGER_AVAILABLE', 'statusVerifiedDate': '2017-02', 'lastUpdateSubmitDate': '2017-02-02', 'studyFirstSubmitDate': '2014-03-24', 'studyFirstSubmitQcDate': '2014-03-24', 'lastUpdatePostDateStruct': {'date': '2017-02-06', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2014-03-27', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['Mucopolysaccharidosis Type 7', 'Sly syndrome', 'UX003', 'Enzyme Replacement Therapy', 'rare disease', 'Lysosomal Storage Disease', 'metabolic disorder'], 'conditions': ['Mucopolysaccharidosis Type 7']}, 'descriptionModule': {'briefSummary': 'Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 1 specific patient\n* Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.\n* Written informed consent of parent(s) or legal guardian(s)'}, 'identificationModule': {'nctId': 'NCT02097251', 'briefTitle': 'An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient', 'organization': {'class': 'OTHER', 'fullName': 'Northwell Health'}, 'officialTitle': 'An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (MPS 7) Patient', 'orgStudyIdInfo': {'id': '13-606A'}}, 'armsInterventionsModule': {'interventions': [{'name': 'UX003', 'type': 'DRUG', 'otherNames': ['recombinant human beta glucuronidase', 'rhGUS'], 'description': 'Open Label'}]}, 'contactsLocationsModule': {'locations': [{'zip': '11040', 'city': 'New Hyde Park', 'state': 'New York', 'country': 'United States', 'facility': "Cohen Children's Medical Center", 'geoPoint': {'lat': 40.7351, 'lon': -73.68791}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Joyce Fox', 'class': 'OTHER'}, 'collaborators': [{'name': 'Ultragenyx Pharmaceutical Inc', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': "Chief of Medical Genetics at Cohen Children's Medical Center", 'investigatorFullName': 'Joyce Fox', 'investigatorAffiliation': 'Northwell Health'}}}}