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)

Part of paid clinical trials in New York, New York.

Sponsor
Novartis Pharmaceuticals
Study ID
NCT07448610
Phase
PHASE4
Status
Recruiting
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Conditions

  • Spinal Muscular Atrophy

Eligibility Criteria

Sex
ALL
Age
2 Years - 100 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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).

Key Dates

First listed
Mar 4, 2026
Start date
Aug 27, 2026
Status verified
Sep 2026
Primary completion
May 3, 2032
Completion
Jun 30, 2032

Study Design

Enrollment
36 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Onasemnogene Abeparvovec-brve
    Participants with spinal muscular atrophy, including: * independently ambulatory children/adolescents (≥ 2 years to \< 18 years); * independently ambulatory adults (≥ 18 years); * Non-ambulatory adults (≥ 18 years)

Primary Outcome Measure

Change from baseline in HFMSE for independently ambulatory participants [ Time Frame: Baseline, 6, 12, 18 and 24 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia University Medical CenterNew YorkNew York10032
Claudia Chiriboga (PRINCIPAL_INVESTIGATOR)

Find similar trials in New York, NY

By research site
Columbia University Medical Center· New York, NY

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