[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100627126":3},{"organization":4,"outcomesModule":7,"designInfo":36,"detailedDescription":43,"studyPopulation":11,"armGroups":44,"interventions":55,"overallOfficials":65,"centralContacts":69,"locations":75,"responsibleParty":184,"collaborators":186,"id":190,"slug":11,"hasResults":191,"nctId":192,"briefTitle":193,"officialTitle":194,"acronym":195,"eligibilityCriteria":196,"healthyVolunteers":191,"sex":197,"minAge":198,"maxAge":11,"enrollmentInfo":199,"targetDuration":11,"studyType":202,"phases":203,"briefSummary":205,"conditions":206,"keywords":208,"overallStatus":78,"whyStopped":11,"lastUpdateSubmitDate":216,"lastUpdatePostDateStruct":217,"startDateStruct":220,"completionDateStruct":222,"leadSponsor":224,"locationsCount":225},{"fullName":5,"class":6},"Broad Institute of MIT and Harvard","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"Frequency of adverse events",null,"Baseline to week 24",[14,18,20,22,24,28,30,31,34],{"measure":15,"description":16,"timeFrame":17},"CSF PrP concentration","Prion protein (PrP) concentration in cerebrospinal fluid (CSF), a pharmacodynamic (PD) biomarker for PrP-siRNA activity","4 weeks post-dose",{"measure":15,"description":16,"timeFrame":19},"8 weeks post-dose",{"measure":15,"description":16,"timeFrame":21},"12 weeks post-dose",{"measure":15,"description":16,"timeFrame":23},"24 weeks post-dose",{"measure":25,"description":26,"timeFrame":27},"Plasma concentration of PrP-siRNA","A pharmacokinetic (PK) measurement of investigational drug concentration in plasma","4 hours post-dose",{"measure":25,"description":26,"timeFrame":29},"24 hours post-dose",{"measure":25,"description":26,"timeFrame":17},{"measure":32,"description":33,"timeFrame":17},"CSF concentration of PrP-siRNA","A pharmacokinetic (PK) measurement of investigational drug concentration in cerebrospinal fluid (CSF)",{"measure":35,"description":11,"timeFrame":12},"Change in CSF PrP over time",{"allocation":37,"interventionModel":38,"interventionModelDescription":39,"primaryPurpose":40,"observationalModel":11,"timePerspective":11,"maskingInfo":41},"NON_RANDOMIZED","SEQUENTIAL","Single ascending dose","TREATMENT",{"masking":42,"maskingDescription":11,"whoMasked":11},"NONE","This is a first-in-human, open label, single ascending dose study in participants with prion disease. The study will consist of a screening period of up to 2 weeks, administration of a single intrathecal dose of PrP-siRNA, and a 24-week follow-up period. Multiple dose levels will be tested. This trial also includes an observational arm in which participants will not receive investigational drug, and will be followed for an 8-week period after baseline.",[45,49],{"label":46,"type":47,"description":48,"interventionNames":11},"Arm 1: Observational","NO_INTERVENTION","In Arm 1, participants will undergo lumbar punctures and other study activities at baseline (Week 0) and at Week 4 and Week 8. Investigational drug will not be administered. We will prioritize enrollment in Arm 2; Arm 1 will be open to enrollment whenever Arm 2 is not open to enrollment.",{"label":50,"type":51,"description":52,"interventionNames":53},"Arm 2: Single ascending dose","EXPERIMENTAL","In Arm 2, participants will be admitted to the clinical trial center and receive a single intrathecal dose of PrP-siRNA. Dose levels to be sequentially evaluated are 50, 100, and 200 mg. Patients will be discharged on Day 2 and then periodically return to the study center on an outpatient basis at Week 1, 2, 4, 8, 12 and 24 for safety monitoring and study activities through the 24 week follow up period.",[54],"Drug: PrP-siRNA",[56],{"type":57,"name":58,"description":59,"armGroupLabels":60,"otherNames":61},"DRUG","PrP-siRNA","Intrathecally administered divalent siRNA designed to target the PRNP mRNA. The structure has been published in DOI: 10.1101\u002F2024.12.05.627039",[50],[62,63,64],"2439-s4","2439-exNA","divalent siRNA 2439-exNA",[66],{"name":67,"affiliation":5,"role":68},"Eric V Minikel, PhD","PRINCIPAL_INVESTIGATOR",[70],{"name":71,"role":72,"phone":73,"phoneExt":11,"email":74},"Broad Institute","CONTACT","617 714 7000","priontrials@broadinstitute.org",[76,101,122,142,163],{"facility":77,"status":78,"city":79,"state":80,"zip":81,"country":82,"countryCode":83,"cosmosGeoPoint":84,"geoPoint":89,"contacts":90},"Massachusetts General Hospital","RECRUITING","Boston","Massachusetts","02114","United States","US",{"type":85,"coordinates":86},"Point",[87,88],-71.05977,42.35843,{"lat":88,"lon":87},[91,95,99],{"name":92,"role":72,"phone":93,"phoneExt":11,"email":94},"Alison McManus, DNP","617-643-4848","ajmcmanus@mgh.harvard.edu",{"name":96,"role":72,"phone":97,"phoneExt":11,"email":98},"Kelli Devitte-McKee, MSN","617-643-7945","kdevitte-mckee@mgb.org",{"name":100,"role":68,"phone":11,"phoneExt":11,"email":11},"Steven E Arnold, MD",{"facility":102,"status":78,"city":103,"state":104,"zip":105,"country":82,"countryCode":83,"cosmosGeoPoint":106,"geoPoint":110,"contacts":111},"Mayo Clinic","Rochester","Minnesota","55905",{"type":85,"coordinates":107},[108,109],-92.4699,44.02163,{"lat":109,"lon":108},[112,116,120],{"name":113,"role":72,"phone":114,"phoneExt":11,"email":115},"Sherry Klingerman, CCRP","507-284-0451","klingerman.sherry@mayo.edu",{"name":117,"role":72,"phone":118,"phoneExt":11,"email":119},"Brenda Nelson","507-293-9237","nelson.brenda6@mayo.edu",{"name":121,"role":68,"phone":11,"phoneExt":11,"email":11},"Vijay K Ramanan, MD PhD",{"facility":123,"status":78,"city":124,"state":124,"zip":125,"country":82,"countryCode":83,"cosmosGeoPoint":126,"geoPoint":130,"contacts":131},"Columbia University Medical Center","New York","10032",{"type":85,"coordinates":127},[128,129],-74.00597,40.71427,{"lat":129,"lon":128},[132,136,140],{"name":133,"role":72,"phone":134,"phoneExt":11,"email":135},"Katrina Cuasay, BA","212-305-2077","kc2305@cumc.columbia.edu",{"name":137,"role":72,"phone":138,"phoneExt":11,"email":139},"Lambrini Whitney, RN","212-342-5615","lnw2109@cumc.columbia.edu",{"name":141,"role":68,"phone":11,"phoneExt":11,"email":11},"Lawrence Honig, MD PhD",{"facility":143,"status":78,"city":144,"state":145,"zip":146,"country":82,"countryCode":83,"cosmosGeoPoint":147,"geoPoint":151,"contacts":152},"University Hospitals Cleveland Medical Center","Cleveland","Ohio","44106",{"type":85,"coordinates":148},[149,150],-81.69541,41.4995,{"lat":150,"lon":149},[153,157,161],{"name":154,"role":72,"phone":155,"phoneExt":11,"email":156},"Mary Andrews, BA, RRT, CCRP","216-844-2386","mary.andrews@uhhospitals.org",{"name":158,"role":72,"phone":159,"phoneExt":11,"email":160},"Prerna Vatsa, BS, MS","216-844-2319","prerna.vatsa@uhhospitals.org",{"name":162,"role":68,"phone":11,"phoneExt":11,"email":11},"Brian S Appleby, MD",{"facility":164,"status":78,"city":165,"state":166,"zip":167,"country":82,"countryCode":83,"cosmosGeoPoint":168,"geoPoint":172,"contacts":173},"Vanderbilt University Medical Center","Nashville","Tennessee","37232",{"type":85,"coordinates":169},[170,171],-86.78444,36.16589,{"lat":171,"lon":170},[174,178,182],{"name":175,"role":72,"phone":176,"phoneExt":11,"email":177},"Kelly Harper, BPS","615-322-2538","kelly.a.harper@vumc.org",{"name":179,"role":72,"phone":180,"phoneExt":11,"email":181},"Leeza Kopaeva, BS","615-421-4100","leeza.kopaeva@vumc.org",{"name":183,"role":68,"phone":11,"phoneExt":11,"email":11},"Amanda Peltier, MD",{"type":185,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[187],{"name":188,"class":189},"National Institute of Neurological Disorders and Stroke (NINDS)","NIH","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":200,"type":201},30,"ESTIMATED","INTERVENTIONAL",[204],"PHASE1","The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.",[207],"Prion Disease",[209,210,211,212,213,214,215],"prion","Creutzfeldt-Jakob disease","Fatal familial insomnia","Gerstmann-Straussler-Scheinker disease","CJD","FFI","GSS","2026-06-04",{"date":218,"type":219},"2026-06-05","ACTUAL",{"date":221,"type":219},"2026-05-15",{"date":223,"type":201},"2029-08-14",{"name":5,"class":6},5]