Onasemnogene Abeparvovec: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Novartis Pharmaceuticals
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).
Description
STREAM is a prospective, multicentre cohort study that will follow U.S. participants with genetically confirmed SMA who receive a single, intrathecal dose of onasemnogene abeparvovec-brve. All eligible participants are enrolled prior to therapeutic injection. No randomisation, blinding, or placebo control is employed; instead, each participant acts as his or her own baseline comparator. This study will use an exploratory external cohort of patients matched by age, clinical characteristics, treatment history and availability of at least 1 year of pre-treatment medical history. This patient coho…
Eligibility
- Age range
- 2–100 years
- Sex
- All
- 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 -…
Interventions
- DrugOnasemnogene Abeparvovec-brve
administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.
Locations (6)
- Arkansas Children's HospitalLittle Rock, Arkansas
- Stanford HospitalStanford, California
- Boston Childrens HospitalBoston, Massachusetts
- Columbia University Medical CenterNew York, New York
- Ohio State University Medical CenterColumbus, Ohio
- University of PennsylvaniaPhiladelphia, Pennsylvania