[{"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:":49},{"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":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100627126","phase-1-prp-targeting-sirna-safety--mechanism-study-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":20,"type":21},30,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.",[27],"Prion Disease",[29,30,31,32,33,34,35],"prion","Creutzfeldt-Jakob disease","Fatal familial insomnia","Gerstmann-Straussler-Scheinker disease","CJD","FFI","GSS","RECRUITING","2026-06-04",{"date":39,"type":40},"2026-06-05","ACTUAL",{"date":42,"type":40},"2026-05-15",{"date":44,"type":21},"2029-08-14",{"name":46,"class":47},"Broad Institute of MIT and Harvard","OTHER",5,""]