Spinal Muscular Atrophies
Conditions
Keywords
Spinal Muscular Atrophies;, Onasemnogene Abeparvovec;, Brazil;, NIS
Brief summary
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
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.
Interventions
Retrospective and prospective observational study. There is no treatment allocation.
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
0 Years to 100 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of treatment-emergent SAEs | Up to 5 years | Incidence and severity of treatment-emergent Serious AEs (SAEs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients who experience at least one AESI and number of patients by AESI | 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 |
| Number of patients who have survived and have not required permanent ventilatory assistance | 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. |
| Time until death or the need for permanent ventilatory assistance | 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. |
| Number of patients who achieve each Developmental Motor Milestone | 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. |
| Number of patients who achieve each WHO Developmental Milestone within age percentiles | 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. |
Countries
Brazil
Contacts
Primary ContactNovartis Pharmaceuticals
Backup ContactNovartis Pharmaceuticals
Outcome results
None listed