[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"prion-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:prion-disease":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,49],{"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,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":4,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":58,"briefSummary":60,"conditions":61,"keywords":4,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":72},"100527915","phase-1-prprofile-a-study-to-assess-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-ion717-100527915","NCT06153966","PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717","A Phase 1\u002F2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease","Key Inclusion Criteria\n\n* A confirmed diagnosis of probable or definite prion disease.\n* Early-stage prion disease at the time of Screening.\n* Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.\n* Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.\n* Aged ≥ 18 at the time of informed consent.\n\nKey Exclusion Criteria\n\n* Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.\n* Any contraindication or unwillingness to undergo an MRI.\n* Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.\n* Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.\n* Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.",{"count":57,"type":21},76,[24,59],"PHASE2","The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.",[27],"2026-04-23",{"date":64,"type":40},"2026-04-24",{"date":66,"type":40},"2024-01-04",{"date":68,"type":21},"2030-06",{"name":70,"class":71},"Ionis Pharmaceuticals, Inc.","INDUSTRY",13]