[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100505485":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":34,"responsibleParty":224,"collaborators":30,"id":226,"slug":227,"hasResults":228,"nctId":229,"briefTitle":230,"officialTitle":231,"acronym":232,"eligibilityCriteria":233,"healthyVolunteers":228,"sex":234,"minAge":235,"maxAge":236,"enrollmentInfo":237,"targetDuration":30,"studyType":240,"phases":241,"briefSummary":243,"conditions":244,"keywords":30,"overallStatus":37,"whyStopped":30,"lastUpdateSubmitDate":246,"lastUpdatePostDateStruct":247,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":255},{"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 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",[35,49,59,68,78,88,98,108,118,128,139,150,159,170,179,188,194,203,213],{"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 Alto","94304",{"type":44,"coordinates":64},[65,66],-122.14302,37.44188,{"lat":66,"lon":65},{"facility":69,"status":37,"city":70,"state":71,"zip":72,"country":41,"countryCode":42,"cosmosGeoPoint":73,"geoPoint":77,"contacts":30},"Children's Hospital of Colorado","Aurora","Colorado","80045",{"type":44,"coordinates":74},[75,76],-104.83192,39.72943,{"lat":76,"lon":75},{"facility":79,"status":37,"city":80,"state":81,"zip":82,"country":41,"countryCode":42,"cosmosGeoPoint":83,"geoPoint":87,"contacts":30},"University of Florida Pediatrics","Gainesville","Florida","32610",{"type":44,"coordinates":84},[85,86],-82.32483,29.65163,{"lat":86,"lon":85},{"facility":89,"status":37,"city":90,"state":91,"zip":92,"country":41,"countryCode":42,"cosmosGeoPoint":93,"geoPoint":97,"contacts":30},"Children's Healthcare of Atlanta Center for Advanced 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 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Daughter","WITHDRAWN","Norfolk","Virginia","23510",{"type":44,"coordinates":135},[136,137],-76.28522,36.84681,{"lat":137,"lon":136},{"facility":140,"status":37,"city":141,"state":30,"zip":142,"country":143,"countryCode":144,"cosmosGeoPoint":145,"geoPoint":149,"contacts":30},"Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie","Berlin","13353","Germany","DE",{"type":44,"coordinates":146},[147,148],13.41053,52.52437,{"lat":148,"lon":147},{"facility":151,"status":37,"city":152,"state":30,"zip":153,"country":143,"countryCode":144,"cosmosGeoPoint":154,"geoPoint":158,"contacts":30},"UKGM Standort Gießen","Giessen","35392",{"type":44,"coordinates":155},[156,157],8.67554,50.58727,{"lat":157,"lon":156},{"facility":160,"status":37,"city":161,"state":30,"zip":162,"country":163,"countryCode":164,"cosmosGeoPoint":165,"geoPoint":169,"contacts":30},"Soroka Medical Center","Beersheba","8410101","Israel","IL",{"type":44,"coordinates":166},[167,168],34.7913,31.25181,{"lat":168,"lon":167},{"facility":171,"status":37,"city":172,"state":30,"zip":173,"country":163,"countryCode":164,"cosmosGeoPoint":174,"geoPoint":178,"contacts":30},"Schneider Children's Medical Center of Israel","Petah Tikva","4920235",{"type":44,"coordinates":175},[176,177],34.88747,32.08707,{"lat":177,"lon":176},{"facility":180,"status":37,"city":181,"state":30,"zip":182,"country":163,"countryCode":164,"cosmosGeoPoint":183,"geoPoint":187,"contacts":30},"Sourasky MC, Dana-Dwek Children's Hospital","Tel Aviv","6423906",{"type":44,"coordinates":184},[185,186],34.78057,32.08088,{"lat":186,"lon":185},{"facility":189,"status":37,"city":190,"state":30,"zip":191,"country":192,"countryCode":193,"cosmosGeoPoint":30,"geoPoint":30,"contacts":30},"Uniwersyteckie Centrum Kliniczne","Gda?sk","80-952","Poland","PL",{"facility":195,"status":37,"city":196,"state":30,"zip":197,"country":192,"countryCode":193,"cosmosGeoPoint":198,"geoPoint":202,"contacts":30},"Instytut Pomnik Centrum Zdrowia Dziecka","Warsaw","04-730",{"type":44,"coordinates":199},[200,201],21.01178,52.22977,{"lat":201,"lon":200},{"facility":204,"status":37,"city":205,"state":30,"zip":30,"country":206,"countryCode":207,"cosmosGeoPoint":208,"geoPoint":212,"contacts":30},"Sidra Medicine","Al Rayyan","Qatar","QA",{"type":44,"coordinates":209},[210,211],51.42444,25.29194,{"lat":211,"lon":210},{"facility":214,"status":37,"city":215,"state":30,"zip":216,"country":217,"countryCode":218,"cosmosGeoPoint":219,"geoPoint":223,"contacts":30},"Great Ormond Street Hospital For Children","London","WC1N 3JH","United Kingdom","UK",{"type":44,"coordinates":220},[221,222],-0.12574,51.50853,{"lat":222,"lon":221},{"type":225,"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":238,"type":239},28,"ESTIMATED","INTERVENTIONAL",[242],"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.",[245],"Muscular Atrophy, Spinal","2026-08-10",{"date":248,"type":249},"2026-08-11","ACTUAL",{"date":251,"type":249},"2024-08-14",{"date":253,"type":239},"2029-03-31",{"name":5,"class":6},19]