Spinal muscular atrophy patients get a single intrathecal gene therapy dose to see how it works in real-world U.S. settings

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

STREAM is a prospective, multicenter, non-randomized cohort study in the United States assessing a single intrathecal dose of ITVISMA® in genetically confirmed SMA patients. Participants are followed in routine care, with each patient serving as their own baseline and an external matched retrospective cohort used for context. Pre-specified endpoints, standardized motor-function assessments, and a detailed analysis plan are used to describe safety and functional outcomes while addressing missing data and intercurrent events. The study emphasizes pragmatic collection of real-world evidence without randomization or placebo control. Enrollment targets SMA patients aged 2 years and older who are treated in standard clinical practice.

Why it matters

Who can join: the full rules

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 - \<18 years; or
  2. independently ambulatory adult (≥ 18 years).
  3. 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 it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Stanford Hospital
  Stanford, California, United States Recruiting
• Boston Childrens Hospital
  Boston, Massachusetts, United States Recruiting
• Columbia University Medical Center
  New York, New York, United States Recruiting
• Ohio State University Medical Center
  Columbus, Ohio, United States Recruiting
• University of Pennsylvania
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Pregnancy-time treatment of fetal SMA: a registry of mother-child outcomes

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• Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

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• Salanersen (BIIB115) for improving movement in people 15–60 with spinal muscular atrophy who are treatment‑naïve or previously on risdiplam

  This trial tests whether salanersen helps movement and is safe for people aged 15 to 60 with spinal muscular atrophy who are either new to treatment or have taken risdiplam.

  Recruiting at 3 sites Phase 3 Biogen · Palo Alto, California and 2 other locations · NCT07444476

• CuidAME: Spain's longitudinal registry for spinal muscular atrophy

  This project collects long-term clinical and treatment data from children and adults with genetically confirmed 5q spinal muscular atrophy in Spain to track outcomes over time.

  Recruiting Fundació Sant Joan de Déu · Esplugues de Llobregat · NCT07231549

• Apitegromab for infants under 2 with spinal muscular atrophy

  This trial will test whether adding apitegromab to approved SMA therapy helps infants under 2 with 5q SMA reach better motor milestones.

  Recruiting at 23 sites Phase 2 Scholar Rock, Inc. · Phoenix, Arizona and 22 other locations · NCT07047144

• Understanding fatigue in patients with spinal muscular atrophy treated with nusinersen

  This study is trying to see how nusinersen treatment affects fatigue in people with spinal muscular atrophy and how that fatigue impacts their daily activities.

  Recruiting Columbia University · New York, New York · NCT06955897

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.