[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100461082":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":34,"responsibleParty":51,"collaborators":54,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":64,"eligibilityCriteria":65,"healthyVolunteers":60,"sex":66,"minAge":10,"maxAge":10,"enrollmentInfo":67,"targetDuration":10,"studyType":70,"phases":10,"briefSummary":71,"conditions":72,"keywords":76,"overallStatus":37,"whyStopped":10,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":91},{"fullName":5,"class":6},"Nemours Children's Clinic","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Mucopolysaccharidosis IVA",null,"Patients affected by MPS IVA. The diagnosis of MPS will be confirmed by deficient enzyme activity of \\\u003C 5% of normal activity level as measured in plasma, leukocytes, or fibroblasts.",[13],"Diagnostic Test: Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":10},"DIAGNOSTIC_TEST","Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.","This study includes 15 major assessments: clinical assessment procedures; anthropometric measurements; activity of daily living and quality-of-life questionnaires; gait kinematics and kinetics analysis; pulmonary function tests (PFT); skeletal radiographs and dual-energy x-ray absorptiometry (DXA); MRI in cervical spine, temporal bones, and hip; computed tomography angiography (CTA) for tracheal obstruction; CT for temporal bones; anesthetic encounters; joint mobility; hearing function; biochemical analyses; and pathological analyses.",[9],[21],{"name":22,"affiliation":23,"role":24},"Shunji Tomatsu, MD PhD","Nemours Children's Care","PRINCIPAL_INVESTIGATOR",[26,30],{"name":22,"role":27,"phone":28,"phoneExt":10,"email":29},"CONTACT","3022987336","stomatsu@nemours.org",{"name":31,"role":27,"phone":32,"phoneExt":10,"email":33},"Greg Stets","302-298-6504","gstets@nemours.org",[35],{"facility":36,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":49},"Nemours Children's Health, Delaware Valley","RECRUITING","Wilmington","Delaware","19803","United States","US",{"type":44,"coordinates":45},"Point",[46,47],-75.54659,39.74595,{"lat":47,"lon":46},[50],{"name":31,"role":27,"phone":32,"phoneExt":10,"email":33},{"type":24,"investigatorFullName":52,"investigatorTitle":53,"investigatorAffiliation":5,"oldNameTitle":10,"oldOrganization":10},"Shunji Tomatsu","Principal research scientist and Professor",[55],{"name":56,"class":57},"Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)","NIH","100461082","non-invasive-functional-assessment-and-pathogenesis-of-morquio-a-100461082",false,"NCT05284006","Non-invasive Functional Assessment and Pathogenesis of Morquio A","Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)","NIFAMA","Inclusion Criteria:\n\n* Patients affected by MPS IVA. The diagnosis of MPS IVA is confirmed by deficient enzyme activity of \\\u003C 5% of normal activity level as measured in plasma or leukocytes.\n\nExclusion Criteria:\n\n* No","ALL",{"count":68,"type":69},60,"ESTIMATED","OBSERVATIONAL","Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal\u002Fpulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.",[73,74,75],"Mucopolysaccharidosis IV Type A","Morquio A Syndrome","MPS IVA",[77,78,79,80,81],"skeletal dysplasia","GALNS","keratan sulfate","tracheal narrowing","short stature","2026-05-11",{"date":84,"type":85},"2026-05-13","ACTUAL",{"date":87,"type":85},"2021-05-01",{"date":89,"type":69},"2027-04-30",{"name":5,"class":6},1]