Ignite Creation Date:
2025-12-24 @ 5:12 PM
Ignite Modification Date:
2026-02-28 @ 7:09 PM
Study NCT ID:
NCT02067650
Status:
COMPLETED
Last Update Posted:
2018-01-24
First Post:
2013-08-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}], '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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'CROSS_SECTIONAL', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 18}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-01', 'completionDateStruct': {'date': '2016-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-01-23', 'studyFirstSubmitDate': '2013-08-13', 'studyFirstSubmitQcDate': '2014-02-19', 'lastUpdatePostDateStruct': {'date': '2018-01-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2014-02-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Power Doppler', 'timeFrame': 'one time exam', 'description': 'The primary objective is to determine whether patients with various types of MPS and at various stages of the disease exhibit abnormalities of the finger, wrist and knee joints as assessed by grey scale and Power Doppler ultrasonography.'}], 'secondaryOutcomes': [{'measure': 'Ultrasound scanning technique', 'timeFrame': 'one time scan', 'description': 'To determine the optimal ultrasound scanning technique for MPS patients given the potentially advanced joint contractures'}, {'measure': 'Findings of synovitis or tenosynovitis', 'timeFrame': 'one time scan', 'description': 'To determine whether there are ultrasound findings indicative of synovitis or tenosynovitis'}, {'measure': 'Differences between MPS I and IV', 'timeFrame': 'one time scan', 'description': 'To determine whether differences exist between MPS I and MPS IV'}, {'measure': 'Differences between ERT and BMT', 'timeFrame': 'one time scan', 'description': 'To determine whether differences exist between patients with or without Enzyme Replacement Therapy (ERT) or Bone Marrow Transplants'}, {'measure': 'Differences between severe and attenuated phenotypes', 'timeFrame': 'one time scan', 'description': 'To determine whether differences exist between severe and attenuated phenotypes'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['MPS I', 'MPS IV']}, 'descriptionModule': {'briefSummary': 'Hardly any imaging studies specifically addressing the articular and periarticular structures in MPS patients are available at this point. The investigators propose to do for the first time a pilot study using musculoskeletal ultrasound in patients with various types of MPS.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '99 Years', 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with MPS I and IV', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria\n\n* Diagnosis of Mucopolysaccharidosis\n* Ability to collaborate with the ultrasound assessment\n* Written informed consent from parents and patients over 14 years of age, assent from children 7 to 14 years\n\nExclusion Criteria\n\n* Inability to collaborate with the ultrasound assessment\n* Acute trauma to a joint that will be assessed in the 3 months preceding the study\n* Coexistence of other pathology that is known to affect the respective joints'}, 'identificationModule': {'nctId': 'NCT02067650', 'briefTitle': 'Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis', 'organization': {'class': 'OTHER', 'fullName': "Children's Hospital of Eastern Ontario"}, 'officialTitle': 'Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis', 'orgStudyIdInfo': {'id': 'MPSUS'}}, 'contactsLocationsModule': {'locations': [{'zip': 'K1H8L1', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital of Eastern Ontario", 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}], 'overallOfficials': [{'name': 'Johannes Roth, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children's Hospital of Eastern Ontario"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Children's Hospital of Eastern Ontario", 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Johannes Roth', 'investigatorAffiliation': "Children's Hospital of Eastern Ontario"}}}}