[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100505485":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":34,"responsibleParty":228,"collaborators":30,"id":230,"slug":231,"hasResults":232,"nctId":233,"briefTitle":234,"officialTitle":235,"acronym":236,"eligibilityCriteria":237,"healthyVolunteers":232,"sex":238,"minAge":239,"maxAge":240,"enrollmentInfo":241,"targetDuration":30,"studyType":244,"phases":245,"briefSummary":247,"conditions":248,"keywords":30,"overallStatus":37,"whyStopped":30,"lastUpdateSubmitDate":250,"lastUpdatePostDateStruct":251,"startDateStruct":254,"completionDateStruct":256,"leadSponsor":258,"locationsCount":259},{"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: BN44621 https:\u002F\u002Fforpatients.roche.com\u002F","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. No email attachments. https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry",[35,49,59,68,78,88,98,108,118,128,138,149,158,169,178,187,198,207,217],{"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},"Valley Children's Hospital","Madera","California","93636",{"type":44,"coordinates":55},[56,57],-120.06072,36.96134,{"lat":57,"lon":56},{"facility":60,"status":37,"city":61,"state":52,"zip":62,"country":41,"countryCode":42,"cosmosGeoPoint":63,"geoPoint":67,"contacts":30},"Stanford Univ Medical Center","Palo 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Pediatrics","Atlanta","Georgia","30329-2309",{"type":44,"coordinates":94},[95,96],-84.38798,33.749,{"lat":96,"lon":95},{"facility":99,"status":37,"city":100,"state":101,"zip":102,"country":41,"countryCode":42,"cosmosGeoPoint":103,"geoPoint":107,"contacts":30},"Helen DeVos Children's Hospital at Spectrum Health","Grand Rapids","Michigan","49503",{"type":44,"coordinates":104},[105,106],-85.66809,42.96336,{"lat":106,"lon":105},{"facility":109,"status":37,"city":110,"state":111,"zip":112,"country":41,"countryCode":42,"cosmosGeoPoint":113,"geoPoint":117,"contacts":30},"Children'S Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":44,"coordinates":114},[115,116],-75.16362,39.95238,{"lat":116,"lon":115},{"facility":119,"status":37,"city":120,"state":121,"zip":122,"country":41,"countryCode":42,"cosmosGeoPoint":123,"geoPoint":127,"contacts":30},"University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"type":44,"coordinates":124},[125,126],-96.80667,32.78306,{"lat":126,"lon":125},{"facility":129,"status":37,"city":130,"state":131,"zip":132,"country":41,"countryCode":42,"cosmosGeoPoint":133,"geoPoint":137,"contacts":30},"Children's Hospital of the King's Daughter","Norfolk","Virginia","23510",{"type":44,"coordinates":134},[135,136],-76.28522,36.84681,{"lat":136,"lon":135},{"facility":139,"status":37,"city":140,"state":30,"zip":141,"country":142,"countryCode":143,"cosmosGeoPoint":144,"geoPoint":148,"contacts":30},"Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie","Berlin","13353","Germany","DE",{"type":44,"coordinates":145},[146,147],13.41053,52.52437,{"lat":147,"lon":146},{"facility":150,"status":37,"city":151,"state":30,"zip":152,"country":142,"countryCode":143,"cosmosGeoPoint":153,"geoPoint":157,"contacts":30},"UKGM Standort Gießen","Giessen","35392",{"type":44,"coordinates":154},[155,156],8.67554,50.58727,{"lat":156,"lon":155},{"facility":159,"status":37,"city":160,"state":30,"zip":161,"country":162,"countryCode":163,"cosmosGeoPoint":164,"geoPoint":168,"contacts":30},"Soroka Medical Center","Beersheba","8410101","Israel","IL",{"type":44,"coordinates":165},[166,167],34.7913,31.25181,{"lat":167,"lon":166},{"facility":170,"status":37,"city":171,"state":30,"zip":172,"country":162,"countryCode":163,"cosmosGeoPoint":173,"geoPoint":177,"contacts":30},"Schneider Children's Medical Center of Israel","Petah Tikva","4920235",{"type":44,"coordinates":174},[175,176],34.88747,32.08707,{"lat":176,"lon":175},{"facility":179,"status":37,"city":180,"state":30,"zip":181,"country":162,"countryCode":163,"cosmosGeoPoint":182,"geoPoint":186,"contacts":30},"Sourasky MC, Dana-Dwek Children's Hospital","Tel Aviv","6423906",{"type":44,"coordinates":183},[184,185],34.78057,32.08088,{"lat":185,"lon":184},{"facility":188,"status":37,"city":189,"state":30,"zip":190,"country":191,"countryCode":192,"cosmosGeoPoint":193,"geoPoint":197,"contacts":30},"Uniwersyteckie Centrum Kliniczne","Gdansk","80-952","Poland","PL",{"type":44,"coordinates":194},[195,196],18.64912,54.35227,{"lat":196,"lon":195},{"facility":199,"status":37,"city":200,"state":30,"zip":201,"country":191,"countryCode":192,"cosmosGeoPoint":202,"geoPoint":206,"contacts":30},"Instytut Pomnik Centrum Zdrowia Dziecka","Warsaw","04-730",{"type":44,"coordinates":203},[204,205],21.01178,52.22977,{"lat":205,"lon":204},{"facility":208,"status":37,"city":209,"state":30,"zip":30,"country":210,"countryCode":211,"cosmosGeoPoint":212,"geoPoint":216,"contacts":30},"Sidra Medicine","Al Rayyan","Qatar","QA",{"type":44,"coordinates":213},[214,215],51.42444,25.29194,{"lat":215,"lon":214},{"facility":218,"status":37,"city":219,"state":30,"zip":220,"country":221,"countryCode":222,"cosmosGeoPoint":223,"geoPoint":227,"contacts":30},"Great Ormond Street Hospital For Children","London","WC1N 3JH","United Kingdom","UK",{"type":44,"coordinates":224},[225,226],-0.12574,51.50853,{"lat":226,"lon":225},{"type":229,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100505485","phase-4-a-study-evaluating-the-effectiveness-and-safety-of-risdiplam-administered-in-pediatric-patients-with-spinal-muscular-atrophy-who-experienced-a-plateau-or-decline-in-function-after-gene-therapy-100505485",false,"NCT05861999","A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy","A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy","HINALEA 2","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 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* In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function\u002Fability (respiratory, motor function, other) per appropriate expectation.\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* 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":242,"type":243},28,"ESTIMATED","INTERVENTIONAL",[246],"PHASE4","This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \\\u003C2 years of age genetically diagnosed with SMA.",[249],"Muscular Atrophy, Spinal","2026-06-05",{"date":252,"type":253},"2026-06-09","ACTUAL",{"date":255,"type":253},"2024-08-14",{"date":257,"type":243},"2029-03-31",{"name":5,"class":6},19]