[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100505484":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":32,"responsibleParty":191,"collaborators":30,"id":193,"slug":194,"hasResults":195,"nctId":196,"briefTitle":197,"officialTitle":198,"acronym":199,"eligibilityCriteria":200,"healthyVolunteers":195,"sex":201,"minAge":202,"maxAge":203,"enrollmentInfo":204,"targetDuration":30,"studyType":207,"phases":208,"briefSummary":210,"conditions":211,"keywords":30,"overallStatus":35,"whyStopped":30,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":217,"completionDateStruct":219,"leadSponsor":221,"locationsCount":222},{"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],{"name":27,"role":28,"phone":29,"phoneExt":30,"email":31},"Reference Study ID Number: BN44620 https:\u002F\u002Fforpatients.roche.com\u002F","CONTACT","888-662-6728 (U.S. Only)",null,"global-roche-genentech-trials@gene.com",[33,47,57,67,77,88,98,107,117,127,136,146,157,166,171,180],{"facility":34,"status":35,"city":36,"state":37,"zip":38,"country":39,"countryCode":40,"cosmosGeoPoint":41,"geoPoint":46,"contacts":30},"University of Arkansas for Medical Sciences","RECRUITING","Little Rock","Arkansas","72103","United States","US",{"type":42,"coordinates":43},"Point",[44,45],-92.28959,34.74648,{"lat":45,"lon":44},{"facility":48,"status":35,"city":49,"state":50,"zip":51,"country":39,"countryCode":40,"cosmosGeoPoint":52,"geoPoint":56,"contacts":30},"Children's Hospital of Colorado","Aurora","Colorado","80045",{"type":42,"coordinates":53},[54,55],-104.83192,39.72943,{"lat":55,"lon":54},{"facility":58,"status":35,"city":59,"state":60,"zip":61,"country":39,"countryCode":40,"cosmosGeoPoint":62,"geoPoint":66,"contacts":30},"University of Florida Pediatrics","Gainesville","Florida","32610",{"type":42,"coordinates":63},[64,65],-82.32483,29.65163,{"lat":65,"lon":64},{"facility":68,"status":35,"city":69,"state":70,"zip":71,"country":39,"countryCode":40,"cosmosGeoPoint":72,"geoPoint":76,"contacts":30},"Children's Healthcare of Atlanta Center for Advanced Pediatrics","Atlanta","Georgia","30329-2309",{"type":42,"coordinates":73},[74,75],-84.38798,33.749,{"lat":75,"lon":74},{"facility":78,"status":79,"city":80,"state":81,"zip":82,"country":39,"countryCode":40,"cosmosGeoPoint":83,"geoPoint":87,"contacts":30},"Ann and Robert H. Lurie Children Hospital of Chicago","NOT_YET_RECRUITING","Chicago","Illinois","60611",{"type":42,"coordinates":84},[85,86],-87.65005,41.85003,{"lat":86,"lon":85},{"facility":89,"status":35,"city":90,"state":91,"zip":92,"country":39,"countryCode":40,"cosmosGeoPoint":93,"geoPoint":97,"contacts":30},"Helen DeVos Children's Hospital at Spectrum Health","Grand Rapids","Michigan","49503",{"type":42,"coordinates":94},[95,96],-85.66809,42.96336,{"lat":96,"lon":95},{"facility":99,"status":35,"city":100,"state":100,"zip":101,"country":39,"countryCode":40,"cosmosGeoPoint":102,"geoPoint":106,"contacts":30},"Columbia University Medical Center","New York","10032",{"type":42,"coordinates":103},[104,105],-74.00597,40.71427,{"lat":105,"lon":104},{"facility":108,"status":35,"city":109,"state":110,"zip":111,"country":39,"countryCode":40,"cosmosGeoPoint":112,"geoPoint":116,"contacts":30},"Children'S Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":42,"coordinates":113},[114,115],-75.16362,39.95238,{"lat":115,"lon":114},{"facility":118,"status":35,"city":119,"state":120,"zip":121,"country":39,"countryCode":40,"cosmosGeoPoint":122,"geoPoint":126,"contacts":30},"The University of Texas Southwestern Medical Center at Dallas","Dallas","Texas","75390",{"type":42,"coordinates":123},[124,125],-96.80667,32.78306,{"lat":125,"lon":124},{"facility":128,"status":35,"city":129,"state":120,"zip":130,"country":39,"countryCode":40,"cosmosGeoPoint":131,"geoPoint":135,"contacts":30},"Cook Children's Jane and John Justin Neurosciences Center","Fort Worth","76104",{"type":42,"coordinates":132},[133,134],-97.32085,32.72541,{"lat":134,"lon":133},{"facility":137,"status":35,"city":138,"state":139,"zip":140,"country":39,"countryCode":40,"cosmosGeoPoint":141,"geoPoint":145,"contacts":30},"Children's Hospital of the King's Daughter","Norfolk","Virginia","23510",{"type":42,"coordinates":142},[143,144],-76.28522,36.84681,{"lat":144,"lon":143},{"facility":147,"status":35,"city":148,"state":30,"zip":149,"country":150,"countryCode":151,"cosmosGeoPoint":152,"geoPoint":156,"contacts":30},"Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie","Berlin","13353","Germany","DE",{"type":42,"coordinates":153},[154,155],13.41053,52.52437,{"lat":155,"lon":154},{"facility":158,"status":35,"city":159,"state":30,"zip":160,"country":150,"countryCode":151,"cosmosGeoPoint":161,"geoPoint":165,"contacts":30},"UKGM Standort Gießen","Giessen","35392",{"type":42,"coordinates":162},[163,164],8.67554,50.58727,{"lat":164,"lon":163},{"facility":167,"status":35,"city":167,"state":30,"zip":168,"country":169,"countryCode":170,"cosmosGeoPoint":30,"geoPoint":30,"contacts":30},"Uniwersyteckie Centrum Kliniczne","80-952","Poland","PL",{"facility":172,"status":35,"city":173,"state":30,"zip":174,"country":169,"countryCode":170,"cosmosGeoPoint":175,"geoPoint":179,"contacts":30},"Instytut Pomnik Centrum Zdrowia Dziecka","Warsaw","04-730",{"type":42,"coordinates":176},[177,178],21.01178,52.22977,{"lat":178,"lon":177},{"facility":181,"status":35,"city":182,"state":30,"zip":183,"country":184,"countryCode":185,"cosmosGeoPoint":186,"geoPoint":190,"contacts":30},"Great Ormond Street Hospital For Children","London","WC1N 3JH","United Kingdom","UK",{"type":42,"coordinates":187},[188,189],-0.12574,51.50853,{"lat":189,"lon":188},{"type":192,"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":205,"type":206},28,"ESTIMATED","INTERVENTIONAL",[209],"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.",[212],"Muscular Atrophy, Spinal","2026-05-01",{"date":215,"type":216},"2026-05-04","ACTUAL",{"date":218,"type":216},"2024-05-30",{"date":220,"type":206},"2029-03-31",{"name":5,"class":6},16]