[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100527915":3},{"organization":4,"outcomesModule":7,"designInfo":29,"detailedDescription":38,"studyPopulation":11,"armGroups":39,"interventions":54,"overallOfficials":11,"centralContacts":64,"locations":69,"responsibleParty":209,"collaborators":11,"id":211,"slug":11,"hasResults":212,"nctId":213,"briefTitle":214,"officialTitle":215,"acronym":11,"eligibilityCriteria":216,"healthyVolunteers":212,"sex":217,"minAge":218,"maxAge":11,"enrollmentInfo":219,"targetDuration":11,"studyType":222,"phases":223,"briefSummary":226,"conditions":227,"keywords":11,"overallStatus":72,"whyStopped":11,"lastUpdateSubmitDate":229,"lastUpdatePostDateStruct":230,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":238},{"fullName":5,"class":6},"Ionis Pharmaceuticals, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"Incidence of treatment-emergent adverse events.",null,"Baseline up to Week 33",[14,17,19,21,24,27],{"measure":15,"description":11,"timeFrame":16},"Maximum Observed Plasma Concentration (Cmax) of ION717","on Day 1 and Week 9",{"measure":18,"description":11,"timeFrame":16},"Area Under the Plasma Concentration-time Curve (AUC) of ION717",{"measure":20,"description":11,"timeFrame":16},"Half-life (t1\u002F2λz) of ION717 in Plasma",{"measure":22,"description":11,"timeFrame":23},"Cerebrospinal fluid (CSF) Concentration of ION717","Pre-dose and at multiple points post-dose up to Week 33",{"measure":25,"description":11,"timeFrame":26},"Amount of ION717 Excreted in Urine","Post-dose on Day 1",{"measure":28,"description":11,"timeFrame":23},"Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF",{"allocation":30,"interventionModel":31,"interventionModelDescription":11,"primaryPurpose":32,"observationalModel":11,"timePerspective":11,"maskingInfo":33},"RANDOMIZED","PARALLEL","TREATMENT",{"masking":34,"maskingDescription":11,"whoMasked":35},"DOUBLE",[36,37],"PARTICIPANT","INVESTIGATOR","This is a first-in-human, multi-center study in participants with prion disease. The study will consist of a screening period of up to 6 weeks, a 30-week treatment period, a 142-week open-label extension period and a 32-week post-treatment period. Multiple dose levels will be tested.\n\nThe trial consists of three Regimens. Regimens 1 and 2 are fully enrolled. Participants in Regimens 1 and 2 received multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses (i.e. whether a given dose was ION717 or placebo) was blinded. The trial sites listed below are actively recruiting eligible participants for Regimen 3. Regimen 3 is open label.",[40,47,50],{"label":41,"type":42,"description":43,"interventionNames":44},"ION717 + Placebo, Regimen 1","EXPERIMENTAL","Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period.",[45,46],"Drug: ION717","Drug: Placebo",{"label":48,"type":42,"description":43,"interventionNames":49},"ION717 + Placebo, Regimen 2",[45,46],{"label":51,"type":42,"description":52,"interventionNames":53},"ION717, Regimen 3","Participants will receive multiple doses of ION717 during the 30-week treatment period and the 142-week open-label extension period.",[45],[55,60],{"type":56,"name":57,"description":58,"armGroupLabels":59,"otherNames":11},"DRUG","ION717","ION717 will be administered by IT injection.",[41,48,51],{"type":56,"name":61,"description":62,"armGroupLabels":63,"otherNames":11},"Placebo","Placebo-matching ION717 will be administered by IT injection.",[41,48],[65],{"name":5,"role":66,"phone":67,"phoneExt":11,"email":68},"CONTACT","(844) 892-5665","PrionDisease@clinicaltrialmedia.com",[70,84,93,103,114,127,138,149,160,171,178,188,198],{"facility":71,"status":72,"city":73,"state":74,"zip":75,"country":76,"countryCode":77,"cosmosGeoPoint":78,"geoPoint":83,"contacts":11},"Massachusetts General Hospital","RECRUITING","Boston","Massachusetts","02114","United States","US",{"type":79,"coordinates":80},"Point",[81,82],-71.05977,42.35843,{"lat":82,"lon":81},{"facility":85,"status":72,"city":86,"state":86,"zip":87,"country":76,"countryCode":77,"cosmosGeoPoint":88,"geoPoint":92,"contacts":11},"NYU Langone Health","New York","10016",{"type":79,"coordinates":89},[90,91],-74.00597,40.71427,{"lat":91,"lon":90},{"facility":94,"status":72,"city":95,"state":96,"zip":97,"country":76,"countryCode":77,"cosmosGeoPoint":98,"geoPoint":102,"contacts":11},"University Hospitals Cleveland Medical Center","Cleveland","Ohio","44106",{"type":79,"coordinates":99},[100,101],-81.69541,41.4995,{"lat":101,"lon":100},{"facility":104,"status":72,"city":105,"state":11,"zip":106,"country":107,"countryCode":108,"cosmosGeoPoint":109,"geoPoint":113,"contacts":11},"Royal Melbourne Hospital","Parkville","3050","Australia","AU",{"type":79,"coordinates":110},[111,112],144.95,-37.78333,{"lat":112,"lon":111},{"facility":115,"status":116,"city":117,"state":118,"zip":119,"country":120,"countryCode":121,"cosmosGeoPoint":122,"geoPoint":126,"contacts":11},"McGill University Health Centre","ACTIVE_NOT_RECRUITING","Montreal","Quebec","H3A 2B4","Canada","CA",{"type":79,"coordinates":123},[124,125],-73.58781,45.50884,{"lat":125,"lon":124},{"facility":128,"status":72,"city":129,"state":11,"zip":130,"country":131,"countryCode":132,"cosmosGeoPoint":133,"geoPoint":137,"contacts":11},"Hôpital Universitaire Pitié Salpêtrière","Paris","75013","France","FR",{"type":79,"coordinates":134},[135,136],2.3488,48.85341,{"lat":136,"lon":135},{"facility":139,"status":72,"city":140,"state":11,"zip":141,"country":142,"countryCode":143,"cosmosGeoPoint":144,"geoPoint":148,"contacts":11},"University Medical Center Göttingen","Göttingen","37075","Germany","DE",{"type":79,"coordinates":145},[146,147],9.93228,51.53443,{"lat":147,"lon":146},{"facility":150,"status":72,"city":151,"state":11,"zip":152,"country":153,"countryCode":154,"cosmosGeoPoint":155,"geoPoint":159,"contacts":11},"Tel Aviv Sourasky Medical Center","Tel Aviv","6423906","Israel","IL",{"type":79,"coordinates":156},[157,158],34.78057,32.08088,{"lat":158,"lon":157},{"facility":161,"status":72,"city":162,"state":11,"zip":163,"country":164,"countryCode":165,"cosmosGeoPoint":166,"geoPoint":170,"contacts":11},"Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta","Milan","20133","Italy","IT",{"type":79,"coordinates":167},[168,169],9.18951,45.46427,{"lat":169,"lon":168},{"facility":172,"status":116,"city":173,"state":174,"zip":175,"country":176,"countryCode":177,"cosmosGeoPoint":11,"geoPoint":11,"contacts":11},"Mihara Memorial Hospital","Gunma","Isesaki-shi","372-0006","Japan","JP",{"facility":179,"status":72,"city":180,"state":181,"zip":182,"country":176,"countryCode":177,"cosmosGeoPoint":183,"geoPoint":187,"contacts":11},"National Center of Neurology and Psychiatry","Tokyo","Kodaira-shi","187-8551",{"type":79,"coordinates":184},[185,186],139.69171,35.6895,{"lat":186,"lon":185},{"facility":189,"status":72,"city":190,"state":191,"zip":192,"country":176,"countryCode":177,"cosmosGeoPoint":193,"geoPoint":197,"contacts":11},"Neuromuscular Center Yoshimizu Hospital","Yamaguchi","Shimonoseki-shi","751-0826",{"type":79,"coordinates":194},[195,196],131.46667,34.18333,{"lat":196,"lon":195},{"facility":199,"status":72,"city":200,"state":11,"zip":201,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":11},"Hospital Clinic De Barcelona","Barcelona","08036","Spain","ES",{"type":79,"coordinates":205},[206,207],2.15899,41.38879,{"lat":207,"lon":206},{"type":210,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100527915",false,"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.","ALL","18 Years",{"count":220,"type":221},76,"ESTIMATED","INTERVENTIONAL",[224,225],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.",[228],"Prion Disease","2026-04-23",{"date":231,"type":232},"2026-04-24","ACTUAL",{"date":234,"type":232},"2024-01-04",{"date":236,"type":221},"2030-06",{"name":5,"class":6},13]