[{"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":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100559717","phase-1-phase-1-study-of-gc1130a-in-patients-with-sanfilippo-syndrome-type-a-mps-iiia-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":20,"type":21},9,"ESTIMATED","INTERVENTIONAL",[24],"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).",[27],"Sanfilippo Syndrome Type A",[29,30],"Mucopolysaccharidosis Type III Subtype A","MPS IIIA","RECRUITING","2025-08-21",{"date":34,"type":35},"2025-08-28","ACTUAL",{"date":37,"type":35},"2024-11-21",{"date":39,"type":21},"2027-06",{"name":41,"class":42},"GC Biopharma Corp","INDUSTRY",5]