[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100502488":3},{"organization":4,"armGroups":7,"interventions":30,"overallOfficials":37,"centralContacts":42,"locations":47,"responsibleParty":218,"collaborators":36,"id":220,"slug":221,"hasResults":222,"nctId":223,"briefTitle":224,"officialTitle":225,"acronym":36,"eligibilityCriteria":226,"healthyVolunteers":222,"sex":227,"minAge":228,"maxAge":229,"enrollmentInfo":230,"targetDuration":36,"studyType":233,"phases":234,"briefSummary":237,"conditions":238,"keywords":242,"overallStatus":50,"whyStopped":36,"lastUpdateSubmitDate":245,"lastUpdatePostDateStruct":246,"startDateStruct":249,"completionDateStruct":251,"leadSponsor":253,"locationsCount":254},{"fullName":5,"class":6},"Vico Therapeutics B. V.","INDUSTRY",[8,14,18,22,26],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1","EXPERIMENTAL","A dose of 10 mg of the trial IMP VO659 will be administered intrathecally four times on Day 1, Day 29, Day 57 and Day 85 within the planned dosing blocks. The total duration of trial participation for each participant is up to approximately 42 weeks, consisting of a screening period of up to 6 weeks, a 13-week dosing period and a 23-week post-dosing period.",[13],"Drug: VO659",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort 2","A dose of 20 mg of the trial IMP VO659 will be administered intrathecally four times on Day 1, Day 29, Day 57 and Day 85 within the planned dosing blocks. The total duration of trial participation for each participant is up to approximately 42 weeks, consisting of a screening period of up to 6 weeks, a 13-week dosing period and a 23-week post-dosing period.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Cohort 3","A dose of 40 mg of the trial IMP VO659 will be administered intrathecally four times on Day 1, Day 29, Day 57 and Day 85 within the planned dosing blocks. The total duration of trial participation for each participant is up to approximately 42 weeks, consisting of a screening period of up to 6 weeks, a 13-week dosing period and a 23-week post-dosing period.",[13],{"label":23,"type":10,"description":24,"interventionNames":25},"Cohort 4","A dose of 20 or 40 mg of the trial IMP VO659 will be randomly assigned and will be administered intrathecally. For Dose-level Cohort 4, the dosing period consists of 3 dosing blocks for participants in the 3x20 mg treatment arm (Days -1 to 3; Days 84-87; and Days 168-171) and 2 dosing blocks for participants in the 2x40 mg treatment arm (Days -1 to 3; and Days 168-171).\n\nThe total duration of trial participation for each participant in Dose-level Cohort 4 is up to approximately 58 weeks, consisting of a screening period of up to 7 weeks, a 26-week dosing period, and a 25-week post dosing period.",[13],{"label":27,"type":10,"description":28,"interventionNames":29},"Cohort 5","A dose of 60 mg of the trial IMP VO659 will be administered intrathecally once on day 1.\n\nThe total duration of trial participation for each participant in Dose-level Cohort 5 is up to approximately 58 weeks, consisting of a screening period of up to 7 weeks, a single dosing followed by a 51-week period of non-dosing, observational visits.",[13],[31],{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":36},"DRUG","VO659","VO659 is an antisense oligonucleotide targeting CAG repeats in mRNA transcripts",[9,15,19,23,27],null,[38],{"name":39,"affiliation":40,"role":41},"Chief Medical Officer","VICO Therapeutics","STUDY_DIRECTOR",[43],{"name":39,"role":44,"phone":45,"phoneExt":36,"email":46},"CONTACT","+31 71 2036800","info@vicotx.com",[48,63,76,87,98,111,122,133,144,157,168,181,194,207],{"facility":49,"status":50,"city":51,"state":36,"zip":36,"country":52,"countryCode":53,"cosmosGeoPoint":54,"geoPoint":59,"contacts":60},"Rigshospitalet","RECRUITING","Copenhagen","Denmark","DK",{"type":55,"coordinates":56},"Point",[57,58],12.56553,55.67594,{"lat":58,"lon":57},[61],{"name":62,"role":44,"phone":36,"phoneExt":36,"email":36},"Lena Hjermind, Dr.",{"facility":64,"status":50,"city":65,"state":36,"zip":36,"country":66,"countryCode":67,"cosmosGeoPoint":68,"geoPoint":72,"contacts":73},"Centre Hospitalier Universitaire dÁngers","Angers","France","FR",{"type":55,"coordinates":69},[70,71],-0.55202,47.47156,{"lat":71,"lon":70},[74],{"name":75,"role":44,"phone":36,"phoneExt":36,"email":36},"Christophe Verny, Prof.",{"facility":77,"status":50,"city":78,"state":36,"zip":36,"country":66,"countryCode":67,"cosmosGeoPoint":79,"geoPoint":83,"contacts":84},"CHU Gui de Chauliac Montpellier- Expert Center of Neurogenetic diseases, Department of Neurology","Montpellier",{"type":55,"coordinates":80},[81,82],3.87635,43.61093,{"lat":82,"lon":81},[85],{"name":86,"role":44,"phone":36,"phoneExt":36,"email":36},"Cecilia Marelli, Dr.",{"facility":88,"status":50,"city":89,"state":36,"zip":36,"country":66,"countryCode":67,"cosmosGeoPoint":90,"geoPoint":94,"contacts":95},"Universtiry Hospitals Pitie Salpetriere - Charles foix - Paris","Paris",{"type":55,"coordinates":91},[92,93],2.3488,48.85341,{"lat":93,"lon":92},[96],{"name":97,"role":44,"phone":36,"phoneExt":36,"email":36},"Alexandra Durr, Prof. Dr.",{"facility":99,"status":50,"city":100,"state":36,"zip":36,"country":101,"countryCode":102,"cosmosGeoPoint":103,"geoPoint":107,"contacts":108},"Katholisches Klinikum Bochum","Bochum","Germany","DE",{"type":55,"coordinates":104},[105,106],7.21648,51.48165,{"lat":106,"lon":105},[109],{"name":110,"role":44,"phone":36,"phoneExt":36,"email":36},"Carsten Saft, Prof.",{"facility":112,"status":50,"city":113,"state":36,"zip":36,"country":101,"countryCode":102,"cosmosGeoPoint":114,"geoPoint":118,"contacts":119},"Deutsches Zentrum fur Neurodegenerative Erkrankungen (DZNE)","Bonn",{"type":55,"coordinates":115},[116,117],7.09549,50.73438,{"lat":117,"lon":116},[120],{"name":121,"role":44,"phone":36,"phoneExt":36,"email":36},"Jennifer Faber, Dr.",{"facility":123,"status":50,"city":124,"state":36,"zip":36,"country":101,"countryCode":102,"cosmosGeoPoint":125,"geoPoint":129,"contacts":130},"Universitatsklinikum Essen - Neurologie","Essen",{"type":55,"coordinates":126},[127,128],7.01228,51.45657,{"lat":128,"lon":127},[131],{"name":132,"role":44,"phone":36,"phoneExt":36,"email":36},"Tim Hagenacker, Prof.",{"facility":134,"status":50,"city":135,"state":36,"zip":36,"country":101,"countryCode":102,"cosmosGeoPoint":136,"geoPoint":140,"contacts":141},"Universitatsklinikum Tübingen","Tübingen",{"type":55,"coordinates":137},[138,139],9.05222,48.52266,{"lat":139,"lon":138},[142],{"name":143,"role":44,"phone":36,"phoneExt":36,"email":36},"Ludger Scholz, Prof Dr.",{"facility":145,"status":50,"city":146,"state":36,"zip":36,"country":147,"countryCode":148,"cosmosGeoPoint":149,"geoPoint":153,"contacts":154},"Meir Medical Center","Kfar Saba","Israel","IL",{"type":55,"coordinates":150},[151,152],34.90694,32.175,{"lat":152,"lon":151},[155],{"name":156,"role":44,"phone":36,"phoneExt":36,"email":36},"Nirit Lev, Dr.",{"facility":158,"status":50,"city":159,"state":36,"zip":36,"country":147,"countryCode":148,"cosmosGeoPoint":160,"geoPoint":164,"contacts":165},"Sourmansky Medical Center","Tel Aviv",{"type":55,"coordinates":161},[162,163],34.78057,32.08088,{"lat":163,"lon":162},[166],{"name":167,"role":44,"phone":36,"phoneExt":36,"email":36},"Tanya Gurevich, Prof.",{"facility":169,"status":50,"city":170,"state":36,"zip":36,"country":171,"countryCode":172,"cosmosGeoPoint":173,"geoPoint":177,"contacts":178},"Leiden University Medical Center LUMC","Leiden","Netherlands","NL",{"type":55,"coordinates":174},[175,176],4.49306,52.15833,{"lat":176,"lon":175},[179],{"name":180,"role":44,"phone":36,"phoneExt":36,"email":36},"Susanne de Bot, Dr.",{"facility":182,"status":50,"city":183,"state":36,"zip":36,"country":171,"countryCode":172,"cosmosGeoPoint":184,"geoPoint":188,"contacts":189},"Radbout University Medical Centre","Nijmegen",{"type":55,"coordinates":185},[186,187],5.85278,51.8425,{"lat":187,"lon":186},[190,192],{"name":191,"role":44,"phone":36,"phoneExt":36,"email":36},"Bart van de Warrenburg, Prof.",{"name":191,"role":193,"phone":36,"phoneExt":36,"email":36},"PRINCIPAL_INVESTIGATOR",{"facility":195,"status":50,"city":196,"state":36,"zip":36,"country":197,"countryCode":198,"cosmosGeoPoint":199,"geoPoint":203,"contacts":204},"University College London Hospitals NHS Foundation","London","United Kingdom","UK",{"type":55,"coordinates":200},[201,202],-0.12574,51.50853,{"lat":202,"lon":201},[205],{"name":206,"role":44,"phone":36,"phoneExt":36,"email":36},"Paola Giunti, Prof.",{"facility":208,"status":50,"city":209,"state":36,"zip":36,"country":197,"countryCode":198,"cosmosGeoPoint":210,"geoPoint":214,"contacts":215},"John Radcliffe Hospital","Oxford",{"type":55,"coordinates":211},[212,213],-1.25596,51.75222,{"lat":213,"lon":212},[216],{"name":217,"role":44,"phone":36,"phoneExt":36,"email":36},"Richard Armstrong, Dr.",{"type":219,"investigatorFullName":36,"investigatorTitle":36,"investigatorAffiliation":36,"oldNameTitle":36,"oldOrganization":36},"SPONSOR","100502488","phase-1-a-safety-and-pharmacokinetics-trial-of-vo659-in-sca1-sca3-and-hd-100502488",false,"NCT05822908","A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD","A Phase 1\u002F2a, Open-label Trial to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered VO659 in Participants With Spinocerebellar Ataxia Types 1, 3 and Huntington's Disease","Main Inclusion Criteria:\n\n* Provide written informed consent (signed and dated). Patients should be assessed for their ability to give informed consent using the Evaluation to Sign Consent tool.\n* Is ≥25 and ≤60 years of age inclusive, of any gender, at the time of signing the informed consent.\n* Have SCA1, SCA3 or HD meeting one of the following criteria:\n\n  1. SCA1 and SCA3: mild to moderate disease with a Scale for Assessment and Rating of Ataxia (SARA) score of ≥3 and ≤18\n  2. HD: early manifest, Stage I disease with a Total Functional Capacity (TFC) Score of ≥11 and ≤13 and a Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Level (DCL) of 4.\n* Have genetically confirmed disease, defined by increased cytosine, adenine, and guanine (CAG) repeat length in the disease-causing allele by direct DNA testing. For each indication the requirements are:\n\n  1. SCA1: ≥41 contiguous, uninterrupted CAG repeats in the ATXN1 gene\n  2. SCA3: ≥61 repeats in the ATXN3 gene\n  3. HD: ≥40 CAG repeats in the HTT gene.\n* Please note there will be additional inclusion criteria\n\nMain Exclusion Criteria:\n\n* Have any condition that would prevent participation in trial assessments.\n* Have one or more pathogenic mutation(s) in another polyQ disease gene, i.e., ATXN2, CACNA1A, ATXN7, TBP, AR, and ATN1, plus either ATXN3 and HTT (for patients with SCA1), ATXN1 and HTT (for participants with SCA3), or ATXN1 and ATXN3 (for participants with HD), in addition to the disease-causing mutation in the ATXN1 (patients with SCA1), ATXN3 (patients with SCA3) or HTT (patients with HD) gene.\n* Have clinical diagnosis of moderate or severe chronic migraines or history of the post-lumbar-puncture headache of moderate or severe intensity requiring hospitalisation or blood patch.\n* Have a brain, spinal or systemic disorder that would interfere with the LP process, CSF circulation, or safety assessments.\n* Have history of bleeding diathesis or coagulopathy, platelet count less than the lower limit of normal unless stable and assessed by the investigator and the Medical Monitor to be not clinically significant.\n* Have uncompensated cardiovascular disorder, any past or present cardiac arrhythmia, QTcF values on screening ECG of \\>470 ms, familial history of long QT syndrome or sudden unexpected death.\n* Have a history of attempted suicide, suicidal ideation with a plan that required hospital admission and\u002For change in level of care within 12 months prior to screening.\n* Have medical, psychiatric, or other conditions that, in the judgement of the investigator, may compromise the patient's ability to understand the patient information sheet, to give informed consent, to comply with all trial requirements, or to complete the trial.\n* Prior treatment with an antisense oligonucleotide (including siRNA).\n* Pregnant or breast-feeding (lactating) women or women who plan to become pregnant or breast-feed during the trial.\n* Unable to undergo and tolerate MRI scans.\n* Please note there will be additional exclusion criteria","ALL","25 Years","60 Years",{"count":231,"type":232},68,"ESTIMATED","INTERVENTIONAL",[235,236],"PHASE1","PHASE2","The goal of this first-in-human clinical trial is to assess the safety and tolerability of four doses of a new study drug called VO659 in people with genetic disorders called spinocerebellar ataxia type 1, type 3 or Huntington's disease. Another aim is to determine the concentrations of the study drug in the cerebral spinal fluid and blood after single and multiple doses. Study drug will be administered by lumbar intrathecal bolus injections.",[239,240,241],"Spinocerebellar Ataxia Type 1","Spinocerebellar Ataxia Type 3","Huntington Disease",[243,244],"hereditary ataxia","Machado-Joseph Disease (MJD)","2025-08-20",{"date":247,"type":248},"2025-08-28","ACTUAL",{"date":250,"type":248},"2023-02-14",{"date":252,"type":232},"2028-10-15",{"name":5,"class":6},14]