[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"sanfilippo-syndrome-type-a\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:sanfilippo-syndrome-type-a":43},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100559717",false,"NCT06567769","Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)","A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)","Inclusion Criteria:\n\n* Participants with documented MPS IIIA diagnosis\n* Participants aged ≥ 12 months and ≤ 18 years\n\nExclusion Criteria:\n\n* Participants with significant non-MPS IIIA related central nervous system impairment\n* Participants with previous complication from intraventricular drug administration\n* Participants with contraindications for MRI scans and for neurosurgery\n* Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study\n* Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy","ALL","12 Months","18 Years",{"count":19,"type":20},9,"ESTIMATED","INTERVENTIONAL",[23],"PHASE1","The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).",[26],"Sanfilippo Syndrome Type A",[28,29],"Mucopolysaccharidosis Type III Subtype A","MPS IIIA","RECRUITING","2025-08-21",{"date":33,"type":34},"2025-08-28","ACTUAL",{"date":36,"type":34},"2024-11-21",{"date":38,"type":20},"2027-06",{"name":40,"class":41},"GC Biopharma Corp","INDUSTRY",5,""]