[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100505484":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":34,"responsibleParty":194,"collaborators":30,"id":196,"slug":197,"hasResults":198,"nctId":199,"briefTitle":200,"officialTitle":201,"acronym":202,"eligibilityCriteria":203,"healthyVolunteers":198,"sex":204,"minAge":205,"maxAge":206,"enrollmentInfo":207,"targetDuration":30,"studyType":210,"phases":211,"briefSummary":213,"conditions":214,"keywords":30,"overallStatus":37,"whyStopped":30,"lastUpdateSubmitDate":216,"lastUpdatePostDateStruct":217,"startDateStruct":220,"completionDateStruct":222,"leadSponsor":224,"locationsCount":225},{"fullName":5,"class":6},"Hoffmann-La Roche","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Risdiplam","EXPERIMENTAL","Participants will receive risdiplam orally once daily for 72 weeks (Treatment Period). The Treatment Period will be followed by a 1-year Treatment Extension Period for a total study duration of 120 weeks (approximately 2.5 years) for each participant enrolled.",[13],"Drug: Risdiplam",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.",[9],[20],"RO7034067",[22],{"name":23,"affiliation":5,"role":24},"Clinical Trials","STUDY_DIRECTOR",[26,32],{"name":27,"role":28,"phone":29,"phoneExt":30,"email":31},"Reference Study ID Number: BN44620 https:\u002F\u002Fforpatients.roche.com\u002F No attachments to email below.","CONTACT","888-662-6728 (U.S. Only)",null,"global-roche-genentech-trials@gene.com",{"name":33,"role":28,"phone":30,"phoneExt":30,"email":30},"Fastest response: use the inquiry form. https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry",[35,49,59,69,79,90,100,109,119,129,138,149,160,169,174,183],{"facility":36,"status":37,"city":38,"state":39,"zip":40,"country":41,"countryCode":42,"cosmosGeoPoint":43,"geoPoint":48,"contacts":30},"University of Arkansas for Medical Sciences","RECRUITING","Little Rock","Arkansas","72103","United States","US",{"type":44,"coordinates":45},"Point",[46,47],-92.28959,34.74648,{"lat":47,"lon":46},{"facility":50,"status":37,"city":51,"state":52,"zip":53,"country":41,"countryCode":42,"cosmosGeoPoint":54,"geoPoint":58,"contacts":30},"Children's Hospital of Colorado","Aurora","Colorado","80045",{"type":44,"coordinates":55},[56,57],-104.83192,39.72943,{"lat":57,"lon":56},{"facility":60,"status":37,"city":61,"state":62,"zip":63,"country":41,"countryCode":42,"cosmosGeoPoint":64,"geoPoint":68,"contacts":30},"University of Florida Pediatrics","Gainesville","Florida","32610",{"type":44,"coordinates":65},[66,67],-82.32483,29.65163,{"lat":67,"lon":66},{"facility":70,"status":37,"city":71,"state":72,"zip":73,"country":41,"countryCode":42,"cosmosGeoPoint":74,"geoPoint":78,"contacts":30},"Children's Healthcare of Atlanta Center for Advanced Pediatrics","Atlanta","Georgia","30329-2309",{"type":44,"coordinates":75},[76,77],-84.38798,33.749,{"lat":77,"lon":76},{"facility":80,"status":81,"city":82,"state":83,"zip":84,"country":41,"countryCode":42,"cosmosGeoPoint":85,"geoPoint":89,"contacts":30},"Ann and Robert H. Lurie Children Hospital of Chicago","NOT_YET_RECRUITING","Chicago","Illinois","60611",{"type":44,"coordinates":86},[87,88],-87.65005,41.85003,{"lat":88,"lon":87},{"facility":91,"status":37,"city":92,"state":93,"zip":94,"country":41,"countryCode":42,"cosmosGeoPoint":95,"geoPoint":99,"contacts":30},"Helen DeVos Children's Hospital at Spectrum Health","Grand Rapids","Michigan","49503",{"type":44,"coordinates":96},[97,98],-85.66809,42.96336,{"lat":98,"lon":97},{"facility":101,"status":37,"city":102,"state":102,"zip":103,"country":41,"countryCode":42,"cosmosGeoPoint":104,"geoPoint":108,"contacts":30},"Columbia University Medical Center","New York","10032",{"type":44,"coordinates":105},[106,107],-74.00597,40.71427,{"lat":107,"lon":106},{"facility":110,"status":37,"city":111,"state":112,"zip":113,"country":41,"countryCode":42,"cosmosGeoPoint":114,"geoPoint":118,"contacts":30},"Children'S Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":44,"coordinates":115},[116,117],-75.16362,39.95238,{"lat":117,"lon":116},{"facility":120,"status":37,"city":121,"state":122,"zip":123,"country":41,"countryCode":42,"cosmosGeoPoint":124,"geoPoint":128,"contacts":30},"The University of Texas Southwestern Medical Center at Dallas","Dallas","Texas","75390",{"type":44,"coordinates":125},[126,127],-96.80667,32.78306,{"lat":127,"lon":126},{"facility":130,"status":37,"city":131,"state":122,"zip":132,"country":41,"countryCode":42,"cosmosGeoPoint":133,"geoPoint":137,"contacts":30},"Cook Children's Jane and John Justin Neurosciences Center","Fort Worth","76104",{"type":44,"coordinates":134},[135,136],-97.32085,32.72541,{"lat":136,"lon":135},{"facility":139,"status":140,"city":141,"state":142,"zip":143,"country":41,"countryCode":42,"cosmosGeoPoint":144,"geoPoint":148,"contacts":30},"Children's Hospital of the King's Daughter","WITHDRAWN","Norfolk","Virginia","23510",{"type":44,"coordinates":145},[146,147],-76.28522,36.84681,{"lat":147,"lon":146},{"facility":150,"status":37,"city":151,"state":30,"zip":152,"country":153,"countryCode":154,"cosmosGeoPoint":155,"geoPoint":159,"contacts":30},"Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie","Berlin","13353","Germany","DE",{"type":44,"coordinates":156},[157,158],13.41053,52.52437,{"lat":158,"lon":157},{"facility":161,"status":37,"city":162,"state":30,"zip":163,"country":153,"countryCode":154,"cosmosGeoPoint":164,"geoPoint":168,"contacts":30},"UKGM Standort Gießen","Giessen","35392",{"type":44,"coordinates":165},[166,167],8.67554,50.58727,{"lat":167,"lon":166},{"facility":170,"status":37,"city":170,"state":30,"zip":171,"country":172,"countryCode":173,"cosmosGeoPoint":30,"geoPoint":30,"contacts":30},"Uniwersyteckie Centrum Kliniczne","80-952","Poland","PL",{"facility":175,"status":37,"city":176,"state":30,"zip":177,"country":172,"countryCode":173,"cosmosGeoPoint":178,"geoPoint":182,"contacts":30},"Instytut Pomnik Centrum Zdrowia Dziecka","Warsaw","04-730",{"type":44,"coordinates":179},[180,181],21.01178,52.22977,{"lat":181,"lon":180},{"facility":184,"status":37,"city":185,"state":30,"zip":186,"country":187,"countryCode":188,"cosmosGeoPoint":189,"geoPoint":193,"contacts":30},"Great Ormond Street Hospital For Children","London","WC1N 3JH","United Kingdom","UK",{"type":44,"coordinates":190},[191,192],-0.12574,51.50853,{"lat":192,"lon":191},{"type":195,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100505484","phase-4-a-study-evaluating-the-effectiveness-and-safety-of-risdiplam-administered-as-an-early-intervention-in-pediatric-participants-with-spinal-muscular-atrophy-after-gene-therapy-100505484",false,"NCT05861986","A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy","A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy","HINALEA 1","Inclusion Criteria:\n\n* \\\u003C2 years of age at the time of informed consent\n* Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene\n* Confirmed presence of two SMN2 gene copies as documented through laboratory testing\n* Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically\n* Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to enrollment\n* If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration\n* Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration\n\nExclusion Criteria:\n\n* Previous or current enrolment in investigational study prior to initiation of study treatment\n* Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information\n* Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide\n* Concomitant or previous use of an anti-myostatin agent\n* Participants requiring invasive ventilation or tracheostomy\n* Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \\[SaO2\\] \\\u003C95%) with or without ventilator support\n* Presence of feeding tube and an OrSAT score of 0\n* Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening\n* Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.","ALL","3 Months","24 Months",{"count":208,"type":209},28,"ESTIMATED","INTERVENTIONAL",[212],"PHASE4","This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \\\u003C 2 years of age genetically diagnosed with SMA.",[215],"Muscular Atrophy, Spinal","2026-08-03",{"date":218,"type":219},"2026-08-04","ACTUAL",{"date":221,"type":219},"2024-05-30",{"date":223,"type":209},"2029-03-31",{"name":5,"class":6},16]