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

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ClinicalTrials.gov Identifier: NCT06019637
Recruitment Status : Recruiting
First Posted : August 31, 2023
Last Update Posted : December 15, 2023
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment
Spinal Muscular Atrophies Other: Onasemnogene Abeparvovec

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.
Study Design
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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
Actual Study Start Date : November 22, 2023
Estimated Primary Completion Date : August 2, 2038
Estimated Study Completion Date : August 2, 2038

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Spinal Muscular Atrophy Patients
Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec
 Other: Onasemnogene Abeparvovec
Retrospective and prospective observational study. There is no treatment allocation.
Other Name: Zolgensma


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of treatment-emergent SAEs [ Time Frame: Up to 5 years ]
    Incidence and severity of treatment-emergent Serious AEs (SAEs)


Secondary Outcome Measures :
  1. Number of patients who experience at least one AESI and number of patients by AESI [ Time Frame: Up to 15 years ]
    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

  2. Number of patients who have survived and have not required permanent ventilatory assistance [ Time Frame: Up to 15 years ]
    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.

  3. Time until death or the need for permanent ventilatory assistance [ Time Frame: Up to 15 years ]
    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.

  4. Number of patients who achieve each Developmental Motor Milestone [ Time Frame: Up to 15 years ]
    Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.

  5. Number of patients who achieve each WHO Developmental Milestone within age percentiles [ Time Frame: Up to 15 years ]
    Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   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.
Criteria

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:

  • 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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06019637


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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Brazil
Novartis Investigative Site Recruiting
Curitiba, PR, Brazil, 81520-060
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT06019637    
Other Study ID Numbers: COAV101ABR01
First Posted: August 31, 2023    Key Record Dates
Last Update Posted: December 15, 2023
Last Verified: December 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Spinal Muscular Atrophies;
Onasemnogene Abeparvovec;
Brazil;
NIS
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
 Nervous System Diseases
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases