Real-world safety and effectiveness of intrathecal onasemnogene abeparvovec in spinal muscular atrophy
Studies how well onasemnogene abeparvovec-brve works and how safe it is in spinal muscular atrophy.
Plain-language title generated from the registry record
Registry title: 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)
Key facts
- Where
- Boston, Columbus, Little Rock, New York, Philadelphia, and Stanford, United StatesSee the 6 sites
- Phase
- Phase 4
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 100 years old?
Source: NCT07448610, who can take part
Where in United States
- Boston Childrens HospitalBoston, United StatesTaking people
- Ohio State University Medical CenterColumbus, United StatesTaking people
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- Columbia University Medical CenterNew York, United StatesTaking people
- University of PennsylvaniaPhiladelphia, United StatesTaking people
- Stanford HospitalStanford, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT07448610
- Record updated
- 29 October 2026
- Original description
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).Read full original descriptionShow less
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)
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).
Inclusion: A participant will be enrolled in STREAM only if all the following conditions are met:; The participant has a genetically confirmed diagnosis of SMA (biallelic SMN1 deletion or mutation); He or she is ≥ 2 years of age on the day of the intrathecal injection; 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; One of the following functional categories applies at screening:; independently ambulatory child/adolescent 2 - <18 years; or; independently ambulatory adult (≥ 18 years); non-ambulatory (includes walkers with assistance) adult > 18 years with entry level RULM at baseline; At least 1 year of pre-treatment medical history, including motor-function documentation, can be retrieved from the site electronic record.
Exclusion: An uncontrolled acute illness, active infection, febrile illness or acute medical condition, within 30 days prior to dosing; Inability to tolerate corticosteroids administered by mouth or gastrostomy tube; A participant will be excluded if any of the following conditions apply:; Contraindication for lumbar puncture as determined by the investigator (e.g., spinal anatomy that precludes safe lumbar puncture); 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; Planned relocation or any circumstance that is likely to prevent completion of the minimum 12-month follow-up; Other protocol inclusion, exclusion criteria may apply.