Onasemnogene Abeparvovec
OtherRetrospective and prospective observational study. There is no treatment allocation.
Other names: Zolgensma
Recruiting
NCT Number: NCT06019637
A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Recruiting
Interested in participating?
Request InfoKey information
Conditions
Age range
0 year–100 year
Sex eligibility
All sexes
Study type
Observational
Primary location
Novartis Investigative Site, Curitiba, Paraná, Brazil
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About this study
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.
Who can participate
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
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.
Treatment and study plan
Primary outcomes
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Incidence and severity of treatment-emergent SAEs
Time frame: Up to 5 years
Incidence and severity of treatment-emergent Serious AEs (SAEs)
Secondary outcomes
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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
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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.
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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.
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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.
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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.
Study contacts
Contact information is provided by the study sponsor or research team.
Sponsors and collaborators
Lead sponsor
Novartis Pharmaceuticals
Industry
Registry information
Official study 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
Acronym: ARISER
Important dates
- Study start
- 2023
- Primary completion
- 2038
- Study completion
- 2038
- First posted
- Aug 31, 2023
- Registry last updated
- Jan 15, 2025
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.