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RecruitingNCT06019637ARISERUpdated Nov 10, 2026

A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

An observational study in Spinal Muscular Atrophies, sponsored by Novartis Pharmaceuticals. Recruiting at 2 sites in Brazil. Open to participants aged 0 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-11-10.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Observational

From the registry’s dates

  • Started Nov 2023; still recruiting 2 years 11 months later.
Updated Nov 10, 2026Start date movedPrimary completion moved+3 moreGo to Updates ↓
Study type
Observational
Model
Cohort
Time perspective
Other
Enrollment
50
Ages
0 Years to 100 Years
Sex
All
01

Study summary

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Read the detailed description

This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment. This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the approved indications and may also allow for detection of new safety signals and provide further guidance on the management of safety risks associated with Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and treating physicians, as required by Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s authorization.

02

Conditions studied

  • Spinal Muscular Atrophies

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Keywords

  • Spinal Muscular Atrophies;
  • Onasemnogene Abeparvovec;
  • Brazil;
  • NIS
03

In context

Muscular Atrophy

412 studies on the registry are indexed under Muscular Atrophy; 77 are open to participants now.

This study's planned enrollment of 50 is below the median of 61 across 73 observational studies indexed under Muscular Atrophy.

Browse Muscular Atrophy studies →

Lead sponsor

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.

Browse Novartis Pharmaceuticals studies →All Novartis studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
0 Years to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

The study population will consist of approximately 50 Brazilian pediatric patients with SMA who were treated with Onasemnogene Abeparvovec (Zolgensma®) in the commercial setting, the closed MAP, or the phase IV OFELIA trial. Patients will be recruited in up to 3 centers in Brazil, over one year of recruitment.

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Subject's parent or legal guardian has provided signed eICF.
  • Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene.
  • Subject treated* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study.

Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it.

*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements.

  • Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study

Exclusion criteria

Exclusion Criteria:

  • Patients currently enrolled in any interventional clinical trial** other than the phase IV OFELIA trial will be excluded from the study.

    • Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study.

During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.

05

Study design

Observational model
Cohort
Time perspective
Other
Enrollment
50 participants (estimated)
Patient registry
No

Groups and cohorts

  • Spinal Muscular Atrophy Patients

    Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec

    Other: Onasemnogene Abeparvovec

Interventions

  • OtherOnasemnogene Abeparvovec

    Retrospective and prospective observational study. There is no treatment allocation.

    Also known as: Zolgensma

06

What researchers measure

Primary outcomes

  1. Incidence and severity of treatment-emergent SAEs

    Incidence and severity of treatment-emergent Serious AEs (SAEs)

    Time frame: Up to 5 years

Secondary outcomes

  1. Number of patients who experience at least one AESI and number of patients by AESI

    Adverse Event of Special Interest: thrombocytopenia, hepatotoxicity, thrombotic microangiopathy (TMA), dorsal root ganglia toxicity, cardiac AEs, new malignancies, new incidence of neurologic, autoimmune, and hematologic disorder

    Time frame: Up to 15 years

  2. Number of patients who have survived and have not required permanent ventilatory assistance

    Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.

    Time frame: Up to 15 years

  3. Time until death or the need for permanent ventilatory assistance

    Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.

    Time frame: Up to 15 years

  4. Number of patients who achieve each Developmental Motor Milestone

    Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.

    Time frame: Up to 15 years

  5. Number of patients who achieve each WHO Developmental Milestone within age percentiles

    Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.

    Time frame: Up to 15 years

07

Study locations

2 of 2 sites recruiting
  • Novartis Investigative Site
    Curitiba, Paraná 81520-060, Brazil
    Recruiting
  • Novartis Investigative Site
    São Paulo, São Paulo 05403-000, Brazil
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it movedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Nov 2023 (actual). Primary completion is estimated for Jan 2042, 217 months after the start.

Of 38 observational studies under Muscular Atrophy, half reached primary completion within 35 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 66. This study's planned 217 months is above that.

Since we started tracking on Sep 25, 2026, the dates moved once, on Nov 10, 2026: primary completion Sep 2038 → Jan 2042 and completion Sep 2038 → Jan 2042.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Aug 25, 2023
First posted
Aug 31, 2023
StartActual
Nov 27, 2023
Primary completionEst.
Jan 2, 2042was Sep 2038
CompletionEst.
Jan 2, 2042was Sep 2038
Last update
Nov 10, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
2 sites added, 2 sites removed
Show 2 added (2 Brazil)
  • Novartis Investigative Site · Curitiba, Brazil
  • Novartis Investigative Site · São Paulo, Brazil
Show 2 removed
  • Novartis Investigative Site · Curitiba, Brazil
  • Novartis Investigative Site · Sao Paulo, Brazil
Nov 10, 2026
Start date
Nov 22, 2023→Nov 27, 2023
Nov 10, 2026
Primary completion
Sep 30, 2038→Jan 2, 2042
Nov 10, 2026
Study completion
Sep 30, 2038→Jan 2, 2042
Nov 10, 2026
Show all 1 update
  1. Nov 10, 2026
    2 sites added, 2 sites removed
    Show 2 added (2 Brazil)
    • Novartis Investigative Site · Curitiba, Brazil
    • Novartis Investigative Site · São Paulo, Brazil
    Show 2 removed
    • Novartis Investigative Site · Curitiba, Brazil
    • Novartis Investigative Site · Sao Paulo, Brazil
    Start date Nov 22, 2023→Nov 27, 2023
    Primary completion Sep 30, 2038→Jan 2, 2042
    Study completion Sep 30, 2038→Jan 2, 2042
    + 1 other change: verification date

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 ↗

10

Registry details

Key details

Study ID
NCT06019637
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Aug 31, 2023
Start date
Nov 27, 2023
Primary completion
Jan 2, 2042 (estimated)
Completion
Jan 2, 2042 (estimated)
Last update
Nov 10, 2026

Study contacts

Novartis Pharmaceuticals
Contact
novartis.email@novartis.com
+41613241111
Novartis Pharmaceuticals
Contact
Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals
View the source record on ClinicalTrials.gov ↗

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