[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100658812":3},{"organization":4,"outcomesModule":7,"designInfo":15,"detailedDescription":18,"studyPopulation":19,"armGroups":20,"interventions":30,"overallOfficials":36,"centralContacts":41,"locations":11,"responsibleParty":50,"collaborators":11,"id":52,"slug":53,"hasResults":54,"nctId":55,"briefTitle":56,"officialTitle":57,"acronym":11,"eligibilityCriteria":58,"healthyVolunteers":54,"sex":59,"minAge":11,"maxAge":11,"enrollmentInfo":60,"targetDuration":11,"studyType":63,"phases":11,"briefSummary":64,"conditions":65,"keywords":70,"overallStatus":73,"whyStopped":11,"lastUpdateSubmitDate":74,"lastUpdatePostDateStruct":75,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":11},{"fullName":5,"class":6},"Ultragenyx Pharmaceutical Inc","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":11,"otherOutcomes":11},[9,13],{"measure":10,"description":11,"timeFrame":12},"Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy",null,"10 Years",{"measure":14,"description":11,"timeFrame":12},"Bayley Scales of Infant and Toddler Development Cognitive Raw Score Over Time",{"allocation":11,"interventionModel":11,"interventionModelDescription":11,"primaryPurpose":11,"observationalModel":16,"timePerspective":17,"maskingInfo":11},"COHORT","PROSPECTIVE","The UX111-CL401 MPS IIIA Disease Monitoring Program is a prospective, multicenter, long term outcomes study to obtain safety and clinical outcomes data for 10 years after the administration of rebisufligene etisparvovec in either a clinical trial or post-marketing setting.","The DMP population comprises patients with MPS IIIA who received rebisufligene etisparvovec in a clinical trial or have been prescribed (or plan to receive) rebisufligene etisparvovec in a post marketing setting.",[21,26],{"label":22,"type":11,"description":23,"interventionNames":24},"Group 1: Rebisufligene Etisparvovec in Prior Clinical Study","Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in prior clinical study involving rebisufligene etisparvovec",[25],"Other: No Intervention",{"label":27,"type":11,"description":28,"interventionNames":29},"Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting","Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in a post-marketing setting",[25],[31],{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":11},"OTHER","No Intervention","No investigational\u002Fstudy product will be administered in this DMP.",[22,27],[37],{"name":38,"affiliation":39,"role":40},"Medical Director","Ultragenyx Pharmaceuticals Inc.","STUDY_DIRECTOR",[42,47],{"name":43,"role":44,"phone":45,"phoneExt":11,"email":46},"Patients Contact: Trial Recruitment","CONTACT","1-888-756-8657","trialrecruitment@ultragenyx.com",{"name":48,"role":44,"phone":45,"phoneExt":11,"email":49},"HCPs Contact: Medical Information","medinfo@ultragenyx.com",{"type":51,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100658812","mps-iiia-disease-monitoring-program-100658812",false,"NCT07855562","MPS IIIA Disease Monitoring Program","Mucopolysaccharidosis Type IIIA (MPS IIIA) Disease Monitoring Program","Inclusion Criteria:\n\n* Confirmed diagnosis of MPS IIIA\n* Patient who:\n\n  1. Group 1: Received rebisufligene etisparvovec in a prior interventional clinical trial.\n\n     May be enrolled upon completion of Study ABT-001 (Cohort 4) or end of study (EOS) or early termination (ET) visit from the prior clinical trial (Study ABT-003 or Study LTFU-ABO-102). OR\n  2. Group 2: Received (or plans to receive) rebisufligene etisparvovec in a postmarketing setting.\n* Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.\n\nExclusion Criteria:\n\n* Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.","ALL",{"count":61,"type":62},55,"ESTIMATED","OBSERVATIONAL","The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.",[66,67,68,69],"MPS IIIA","Sanfilippo Syndrome","Sanfilippo A","Mucopolysaccharidosis III",[66,71,72],"Sanfilippo","Gene Therapy","NOT_YET_RECRUITING","2026-10-01",{"date":76,"type":77},"2026-10-02","ACTUAL",{"date":79,"type":62},"2026-11",{"date":81,"type":62},"2038-10",{"name":5,"class":6}]