A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
- Conditions
- Spinal Muscular Atrophies
- Interventions
- Other: Onasemnogene Abeparvovec
- Registration Number
- NCT06019637
- Lead Sponsor
- Novartis Pharmaceuticals
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 50
- 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
-
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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Spinal Muscular Atrophy Patients Onasemnogene Abeparvovec Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec
- Primary Outcome Measures
Name Time Method Incidence and severity of treatment-emergent SAEs Up to 5 years Incidence and severity of treatment-emergent Serious AEs (SAEs)
- Secondary Outcome Measures
Name Time Method 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.
Trial Locations
- Locations (1)
Novartis Investigative Site
🇧🇷Sao Paulo, SP, Brazil