[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05861999":3,"trial-entities:NCT05861999":236,"trial-summary:NCT05861999":241},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":15,"conditions":17,"keywords":19,"study_type":20,"primary_purpose":21,"phases":22,"enrollment_info":24,"interventions":27,"primary_outcomes":35,"secondary_outcomes":40,"sex":48,"minimum_age":49,"maximum_age":50,"healthy_volunteers":51,"eligibility_criteria":52,"std_ages":71,"locations":73,"central_contacts":219,"overall_officials":227,"references":231,"see_also_links":232},"NCT05861999","BN44621","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","RECRUITING","2029-03-31","2026-08","2026-08-11","2024-08-14","Hoffmann-La Roche","INDUSTRY",null,"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.",[18],"Muscular Atrophy, Spinal",[],"INTERVENTIONAL","TREATMENT",[23],"PHASE4",{"count":25,"type":26},28,"ESTIMATED",[28],{"type":29,"name":30,"description":31,"armGroupLabels":32,"otherNames":33},"DRUG","Risdiplam","Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.",[30],[34],"RO7034067",[36],{"measure":37,"description":38,"timeFrame":39},"Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment","The BSID-III is a standardized assessment commonly used to evaluate developmental functioning of infants and young children between 1 month and 42 months of age. The gross motor scale measures the movement of the limbs and torso. Items assess static positioning (e.g., sitting, standing); dynamic movement, including locomotion and coordination; balance; and motor planning. The gross motor scale consists of 72 items scored at 0 (unable to perform) or 1 (criteria for item achieved). A higher raw score indicates improvement.","Baseline, Week 72",[41,44,46],{"measure":42,"timeFrame":43},"Percentage of Participants With Adverse Events","Up to 120 weeks",{"measure":45,"timeFrame":43},"Percentage of Participants With Serious Adverse Events",{"measure":47,"timeFrame":43},"Percentage of Participants With Treatment Discontinuation Due to Adverse Events","ALL","3 Months","24 Months",false,{"inclusion":53,"exclusion":61,"raw_text":70},[54,55,56,57,58,59,60],"\\\u003C2 years of age at the time of informed consent","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","Confirmed presence of two SMN2 gene copies as documented through laboratory testing","Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically","Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment","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.","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.",[62,63,64,65,66,67,68,69],"Previous or current enrolment in investigational study prior to initiation of study treatment","Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information","Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide","Concomitant or previous use of an anti-myostatin agent","Participants requiring invasive ventilation or tracheostomy","Presence of feeding tube and an OrSAT score of 0","Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening","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.","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.",[72],"CHILD",[74,83,91,98,106,114,122,130,138,146,155,163,170,178,185,192,197,204,211],{"facility":75,"status":8,"city":76,"state":77,"zip":78,"country":79,"geoPoint":80},"University of Arkansas for Medical Sciences","Little Rock","Arkansas","72103","United States",{"lat":81,"lon":82},34.74648,-92.28959,{"facility":84,"status":8,"city":85,"state":86,"zip":87,"country":79,"geoPoint":88},"Valley Children's Hospital","Madera","California","93636",{"lat":89,"lon":90},36.96134,-120.06072,{"facility":92,"status":8,"city":93,"state":86,"zip":94,"country":79,"geoPoint":95},"Stanford Univ Medical Center","Palo Alto","94304",{"lat":96,"lon":97},37.44188,-122.14302,{"facility":99,"status":8,"city":100,"state":101,"zip":102,"country":79,"geoPoint":103},"Children's Hospital of Colorado","Aurora","Colorado","80045",{"lat":104,"lon":105},39.72943,-104.83192,{"facility":107,"status":8,"city":108,"state":109,"zip":110,"country":79,"geoPoint":111},"University of Florida Pediatrics","Gainesville","Florida","32610",{"lat":112,"lon":113},29.65163,-82.32483,{"facility":115,"status":8,"city":116,"state":117,"zip":118,"country":79,"geoPoint":119},"Children's Healthcare of Atlanta Center for Advanced Pediatrics","Atlanta","Georgia","30329-2309",{"lat":120,"lon":121},33.749,-84.38798,{"facility":123,"status":8,"city":124,"state":125,"zip":126,"country":79,"geoPoint":127},"Helen DeVos Children's Hospital at Spectrum Health","Grand Rapids","Michigan","49503",{"lat":128,"lon":129},42.96336,-85.66809,{"facility":131,"status":8,"city":132,"state":133,"zip":134,"country":79,"geoPoint":135},"Children'S Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"lat":136,"lon":137},39.95238,-75.16362,{"facility":139,"status":8,"city":140,"state":141,"zip":142,"country":79,"geoPoint":143},"University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"lat":144,"lon":145},32.78306,-96.80667,{"facility":147,"status":148,"city":149,"state":150,"zip":151,"country":79,"geoPoint":152},"Children's Hospital of the King's Daughter","WITHDRAWN","Norfolk","Virginia","23510",{"lat":153,"lon":154},36.84681,-76.28522,{"facility":156,"status":8,"city":157,"zip":158,"country":159,"geoPoint":160},"Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie","Berlin","13353","Germany",{"lat":161,"lon":162},52.52437,13.41053,{"facility":164,"status":8,"city":165,"zip":166,"country":159,"geoPoint":167},"UKGM Standort Gießen","Giessen","35392",{"lat":168,"lon":169},50.58727,8.67554,{"facility":171,"status":8,"city":172,"zip":173,"country":174,"geoPoint":175},"Soroka Medical Center","Beersheba","8410101","Israel",{"lat":176,"lon":177},31.25181,34.7913,{"facility":179,"status":8,"city":180,"zip":181,"country":174,"geoPoint":182},"Schneider Children's Medical Center of Israel","Petah Tikva","4920235",{"lat":183,"lon":184},32.08707,34.88747,{"facility":186,"status":8,"city":187,"zip":188,"country":174,"geoPoint":189},"Sourasky MC, Dana-Dwek Children's Hospital","Tel Aviv","6423906",{"lat":190,"lon":191},32.08088,34.78057,{"facility":193,"status":8,"city":194,"zip":195,"country":196},"Uniwersyteckie Centrum Kliniczne","Gda?sk","80-952","Poland",{"facility":198,"status":8,"city":199,"zip":200,"country":196,"geoPoint":201},"Instytut Pomnik Centrum Zdrowia Dziecka","Warsaw","04-730",{"lat":202,"lon":203},52.22977,21.01178,{"facility":205,"status":8,"city":206,"country":207,"geoPoint":208},"Sidra Medicine","Al Rayyan","Qatar",{"lat":209,"lon":210},25.29194,51.42444,{"facility":212,"status":8,"city":213,"zip":214,"country":215,"geoPoint":216},"Great Ormond Street Hospital For Children","London","WC1N 3JH","United Kingdom",{"lat":217,"lon":218},51.50853,-0.12574,[220,225],{"name":221,"role":222,"phone":223,"email":224},"Reference Study ID Number: BN44621 https:\u002F\u002Fforpatients.roche.com\u002F No attachments to email below.","CONTACT","888-662-6728 (U.S. Only)","global-roche-genentech-trials@gene.com",{"name":226,"role":222},"Fastest response: use the inquiry form. https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry",[228],{"name":229,"affiliation":13,"role":230},"Clinical Trials","STUDY_DIRECTOR",[],[233],{"label":234,"url":235},"Please use this form to submit your questions for a faster response: https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry. Do not include or attach any medical records when emailing or completing the form. A nurse will respond within 24 business hours.","https:\u002F\u002Fwww.gene.com\u002Fcontact-us\u002Fsubmit-medical-inquiry",{"nct_id":4,"conditions":237,"biomarkers":239},[238],"Spinal Muscular Atrophy",[240],"SMN1 Gene",{"nct_id":4,"found":242,"summary":243,"prompt_version":253},true,{"design":244,"status":245,"heading":246,"summary":247,"follow_up":248,"word_count":249,"commitments":250,"compensation":251,"drugs_mentioned":252},"This is an open-label, single-arm study, meaning everyone receives risdiplam and there is no comparison group. It plans to enroll 28 participants.","completed","Study of Risdiplam for Spinal Muscular Atrophy After Gene Therapy","This study is looking at how well and how safely a medicine called risdiplam works in young children with spinal muscular atrophy (SMA) who have already received gene therapy but are no longer improving or are getting worse. Risdiplam is given by mouth at a dose adjusted for your child's age and weight. Children between 3 and 24 months old with a confirmed diagnosis of SMA and two copies of the SMN2 gene can join. The main goal is to see how much your child's gross motor skills (like sitting and crawling) improve after 72 weeks of taking risdiplam, using a special test called the Bayley Scales of Infant and Toddler Development. The study is currently recruiting 28 participants.","Participants' gross motor skills will be measured at 72 weeks after starting risdiplam treatment.",119,"Participants will receive risdiplam orally. Their gross motor skills will be measured at the start of the study and again after 72 weeks of treatment.","Not stated in the trial record.",[30],"v2"]