Not Yet RecruitingPhase 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)

36 participantsStarted 2026-07-01

Plain-language 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).

Who can participate

Age range2 Years – 100 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

What they're measuring

Change from baseline in HFMSE for independently ambulatory participants

Timeframe: Baseline, 6, 12, 18 and 24 months

Change from baseline in RULM for non-ambulatory (including walkers with assistance) participants

Timeframe: Baseline, 6, 12, 18 and 24 months

Trial details

NCT IDNCT07448610

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2032-05-03

Contact for this trial

Novartis Pharmaceuticals

+41613241111 novartis.email@novartis.com

View on ClinicalTrials.gov More Spinal Muscular Atrophy trials