A Phase 4 interventional study of Onasemnogene Abeparvovec-brve in Spinal Muscular Atrophy, sponsored by Novartis Pharmaceuticals. Recruiting at 6 sites in United States. Open to participants aged 2 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 4, Interventional, and Treatment
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).
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 cohort will be based on a database of retrospectively collected electronic medical records data and will be used descriptively to contextualize study outcomes. Bias is minimised through pre-specified endpoints, uniform rater training on motor-function scales, and a detailed statistical analysis plan that stipulates handling of missing data and intercurrent events in advance.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
This study's planned enrollment of 36 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Browse Muscular Atrophy, Spinal studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
A participant will be enrolled in STREAM only if all the following conditions are met:
One of the following functional categories applies at screening:
Key Exclusion Criteria:
A participant will be excluded if any of the following conditions apply:
Other protocol inclusion, exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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)
Drug: Onasemnogene Abeparvovec-brve
administered once via lumbar puncture with systemic corticosteroid prophylaxis per label.
Also known as: (ITVISMA®)
Change from baseline in HFMSE for independently ambulatory participants
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.
Time frame: Baseline, 6, 12, 18 and 24 months
Change from baseline in RULM for non-ambulatory (including walkers with assistance) participants
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.
Time frame: Baseline, 6, 12, 18 and 24 months
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence of AEs and SAEs, including changes in laboratory tests and procedure-related events qualifying and reported as AEs.
Time frame: up to 5 years
Modified SMA Functional Rating Scale (SMA-FRS) for independently ambulatory participants (≥ 18 years of age) and non-ambulatory particpants
Modified Spinal Muscular Atrophy Functional Rating Scale (SMAFRS): Ten items address questions related to eating, upper extremity dressing, lower extremity dressing, grooming, bathing, toileting, turning in bed and adjusting bed clothes, transfers, walking, and climbing stairs. This outcome measure includes effectiveness assessment across predefined participant groups based on relevant clinical and demographic factors, as specified in the protocol.
Time frame: Baseline, 6, 12, 18 and 24 months and Years 3, 4, and 5
Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) for independently ambulatory participants (<18 years of age)
The Assessment of Caregiver Experience with Neuromuscular Disease (ACEND): quantifies caregivers' perceptions of function and quality of life pertaining to time, finance and emotion. The ACEND was developed and validated to specifically assess caregiver impact experienced by raising children severely affected by neuromuscular diseases. While specifically developed for application to caregivers of patients undergoing orthopedic surgery, it has application to those with SMA. This outcome measure includes effectiveness assessment across predefined participant groups based on relevant clinical and demographic factors, as specified in the protocol.
Time frame: Baseline, Months 6, 12, 18, 24, and Years 3, 4, and 5
Subgroup outcomes: Hammersmith Functional Motor Scale Expanded (HFMSE)
Effectiveness assessment across predefined participant groups based on relevant clinical and demographic factors, as specified in the protocol. The HFMSE is a validated SMA specific assessment to give objective information on motor ability and clinical progression. Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Time frame: Baseline, 6, 12, 18 and 24 months
Subgroup outcomes: Revised Upper Limb Module (RULM)
Effectiveness assessment across predefined participant groups based on relevant clinical and demographic factors, as specified in the protocol. The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in independent ambulatory and non-independent ambulatory individuals with SMA. Total scores range from 0-37 points. Higher scores reflect higher level of motor ability.
Time frame: Baseline, 6, 12, 18 and 24 months
Frequency of hospitalizations emergency department visits, outpatient visits, respiratory interventions and nutritional support interventions
Assessment of SMA-related healthcare-resource utilisation.
Time frame: Baseline, Months 6, 12, 18, 24, and Years 3, 4, and 5
Duration of hospitalizations emergency department visits, outpatient visits, respiratory interventions and nutritional support interventions
Assessment of SMA-related healthcare-resource utilisation.
Time frame: Baseline, Months 6, 12, 18, 24, and Years 3, 4, and 5
Cobb angle
Assessment of scoliosis progression as a disease-progression marker.
Time frame: Baseline, up to 24 months
Daily hours of respiratory support (none, non-invasive ventilation, invasive ventilation)
Assessment of respiratory function as a disease-progression marker.
Time frame: Baseline, up to 24 months
Joint-range limitation measured by goniometer
Assessment of evolution of joint contractures as a disease-progression marker.
Time frame: Baseline, up to 24 months
Nutritional-support category (oral, gastrostomy feeds, total parenteral nutrition)
Assessment of nutrition status as a disease-progression marker.
Time frame: Baseline, up to 24 months
6-Minute Walk Test (6MWT) for ambulatory patients
To assess disease-progression markers. 6MWT: distance in metres; greater distance indicates better ambulatory capacity.
Time frame: Baseline, Months 6, 12, 18, and 24
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
No publications or documents are linked to this record.
Started Aug 2026 (actual). Primary completion is estimated for May 2032, 68 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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