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)
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
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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