[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Broad Institute of MIT and Harvard\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":48},{"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":14,"eligibilityCriteria":15,"healthyVolunteers":10,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100627126",false,"NCT07444580","PrP-targeting siRNA Safety & Mechanism Study","An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adult Patients Diagnosed With Symptomatic Prion Disease.","PRiSM","Key inclusion criteria:\n\n1. clinically manifested symptoms of prion disease, in the opinion of the investigator;\n2. a diagnosis of probable prion disease according to CDC criteria;\n3. a positive CSF RT-QuIC or PRNP genetic test;\n4. no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15; and\n5. availability of a study partner to assist with study procedures.\n\nKey exclusion criteria:\n\n1. pregnancy;\n2. contraindication to LP; or\n3. recent participation in a different prion disease clinical trial.\n\nAdditional inclusion and exclusion criteria apply and will be evaluated at screening.","ALL","18 Years",{"count":19,"type":20},30,"ESTIMATED","INTERVENTIONAL",[23],"PHASE1","The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.",[26],"Prion Disease",[28,29,30,31,32,33,34],"prion","Creutzfeldt-Jakob disease","Fatal familial insomnia","Gerstmann-Straussler-Scheinker disease","CJD","FFI","GSS","RECRUITING","2026-06-04",{"date":38,"type":39},"2026-06-05","ACTUAL",{"date":41,"type":39},"2026-05-15",{"date":43,"type":20},"2029-08-14",{"name":45,"class":46},"Broad Institute of MIT and Harvard","OTHER",5,""]