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)

Plain-language summary

This Phase 4 pragmatic, multicenter U.S. study (STREAM) examines the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®, OAV101B) in people with spinal muscular atrophy (SMA) treated in routine care. The aim is to gather real-world evidence on its safety and effectiveness. Participants must have a genetically confirmed SMA diagnosis (biallelic SMN1 deletion or mutation), be at least 2 years old on the day of the intrathecal injection, and their treating investigator must intend to give a single dose of ITVISMA®.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 4

Status

Recruiting

Sponsor

Novartis Pharmaceuticals

Intervention

Onasemnogene Abeparvovec-brve ((ITVISMA®))

Start date

8/27/2026

Estimated completion

6/30/2032

Countries

United States

Related genes

Locations

• New York, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Key Inclusion Criteria:

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:

  1. independently ambulatory child/adolescent 2 - \<18 years; or
  2. independently ambulatory adult (≥ 18 years).
  3. 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.

Key Exclusion Criteria:

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)
* 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
* 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.