[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100627436":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":22,"locations":21,"responsibleParty":29,"collaborators":21,"id":31,"slug":32,"hasResults":33,"nctId":34,"briefTitle":35,"officialTitle":36,"acronym":21,"eligibilityCriteria":37,"healthyVolunteers":33,"sex":38,"minAge":39,"maxAge":40,"enrollmentInfo":41,"targetDuration":21,"studyType":44,"phases":45,"briefSummary":47,"conditions":48,"keywords":50,"overallStatus":61,"whyStopped":21,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":66,"completionDateStruct":68,"leadSponsor":70,"locationsCount":21},{"fullName":5,"class":6},"Novartis","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Onasemnogene Abeparvovec-brve","EXPERIMENTAL","Participants with spinal muscular atrophy, including:\n\n* independently ambulatory children\u002Fadolescents (≥ 2 years to \\\u003C 18 years);\n* independently ambulatory adults (≥ 18 years);\n* Non-ambulatory adults (≥ 18 years)",[13],"Drug: Onasemnogene Abeparvovec-brve",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.",[9],[20],"(ITVISMA®)",null,[23,28],{"name":24,"role":25,"phone":26,"phoneExt":21,"email":27},"Novartis Pharmaceuticals","CONTACT","+41613241111","novartis.email@novartis.com",{"name":24,"role":25,"phone":21,"phoneExt":21,"email":21},{"type":30,"investigatorFullName":21,"investigatorTitle":21,"investigatorAffiliation":21,"oldNameTitle":21,"oldOrganization":21},"SPONSOR","100627436","phase-4-assessing-the-real-world-safety--effectiveness-of-spinal-muscular-atrophy-participants-treated-with-intrathecal-onasemnogene-abeparvovec-brve-oav101b-itvisma-a-us-pragmatic-multicenter-study-stream-100627436",false,"NCT07448610","ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)","Onasemnogene Abeparvovec: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)","Key Inclusion Criteria:\n\nA participant will be enrolled in STREAM only if all the following conditions are met:\n\n* The participant has a genetically confirmed diagnosis of SMA (biallelic SMN1 deletion or mutation).\n* He or she is ≥ 2 years of age on the day of the intrathecal injection.\n* The treating Investigator intends to administer within the current episode of care, a single dose of ITVISMA® (1.2 × 10¹⁴ vg) in routine U.S. practice after written informed consent obtained.\n* One of the following functional categories applies at screening:\n\n  1. independently ambulatory child\u002Fadolescent 2 - \\\u003C18 years; or\n  2. independently ambulatory adult (≥ 18 years).\n  3. non-ambulatory (includes walkers with assistance) adult \\> 18 years with entry level RULM at baseline\n* At least 1 year of pre-treatment medical history, including motor-function documentation, can be retrieved from the site electronic record.\n\nKey Exclusion Criteria:\n\nA participant will be excluded if any of the following conditions apply:\n\n* Contraindication for lumbar puncture as determined by the investigator (e.g., spinal anatomy that precludes safe lumbar puncture)\n* An uncontrolled acute illness, active infection, febrile illness or acute medical condition, within 30 days prior to dosing\n* Inability to tolerate corticosteroids administered by mouth or gastrostomy tube\n* Current participation in another interventional clinical trial that would interfere with the objectives or endpoints of STREAM. Note: participation in observational cohort studies or non-interventional studies in which the participant does not receive treatment or undergo procedures which may compromise this study data integrity may be allowed following Sponsor approval\n* Planned relocation or any circumstance that is likely to prevent completion of the minimum 12-month follow-up.\n\nOther protocol inclusion, exclusion criteria may apply.","ALL","2 Years","100 Years",{"count":42,"type":43},36,"ESTIMATED","INTERVENTIONAL",[46],"PHASE4","The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S. Pragmatic Multicenter Study (STREAM).",[49],"Spinal Muscular Atrophy",[51,52,53,54,55,56,57,58,59,60],"Spinal muscular atrophy","SMA","onasemnogene abeparvovec-brve","intrathecal gene therapy","ITVISMA®","real-world evidence","sponsor-funded","real-world pragmatic trial;","motor function","safety.","NOT_YET_RECRUITING","2026-05-28",{"date":64,"type":65},"2026-05-29","ACTUAL",{"date":67,"type":43},"2026-07-01",{"date":69,"type":43},"2032-06-30",{"name":24,"class":6}]