NCT07448610 · Novartis Pharmaceuticals
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)
What this study is about
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 conducted at multiple hospitals Study (STREAM).
View original scientific description
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).
Interventions
DRUG
Onasemnogene Abeparvovec-brve
administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.
Primary outcome measures
Change from baseline in HFMSE for independently ambulatory participants
Time frame: 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
Time frame: 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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:
- 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
- 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.
Where
- New York, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Sep 18, 2026 · Source of record for eligibility and locations