Ireland
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Recruiting Phase 4

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)

NCT07448610 · tracked via the Priya Life Science USA tracker
Phase
Phase 4
Started
2026-08-27
Last updated
2026-10-29

Condition(s) studied

Spinal Muscular Atrophy

Investigational drug(s) / intervention(s)

Onasemnogene Abeparvovec-brve

Onasemnogene Abeparvovec-brve: administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.

Study summary

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).

Eligibility

Sex
ALL
Min age
2 Years
Max age
100 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Change from baseline in HFMSE for independently ambulatory participants — Baseline, 6, 12, 18 and 24 months
    The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
  • Change from baseline in RULM for non-ambulatory (including walkers with assistance) participants — Baseline, 6, 12, 18 and 24 months
    The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in independent ambulatory and non-ambulatory (including walkers with assistance) individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.

Trial sites (6)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas Recruiting
Stanford Hospital Stanford California Recruiting
Boston Childrens Hospital Boston Massachusetts Recruiting
Columbia University Medical Center New York New York Recruiting
Ohio State University Medical Center Columbus Ohio Recruiting
University of Pennsylvania Philadelphia Pennsylvania Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07448610 on ClinicalTrials.gov ↗ ← All trials in the USA