A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Spinal Muscular Atrophies
De qué trata este estudio
El registro indica que este estudio recluta participantes. La disponibilidad puede variar por centro.
- Tipo de estudio
- Observacional
- Fase
- No aplica / no indicada
- Rango de edad
- 0 años - 100 años
- Sexo admitido
- Todos los sexos
- Voluntarios sanos
- No aceptados
- Participantes
- 50 Estimado
Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.
Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.
Acerca de la investigación
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Leer la descripción completa
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.
¿Quién podría participar?
La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.
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.
Población del estudio
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.
Entiende la evaluación inicialTratamiento, grupos y participación
El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.
- Asignación
- No informado
- Enmascaramiento
- No informado
Spinal Muscular Atrophy Patients
Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec
Other: Onasemnogene Abeparvovec
Onasemnogene Abeparvovec OTHER
Retrospective and prospective observational study. There is no treatment allocation.
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Incidence and severity of treatment-emergent SAEs
Plazo: Up to 5 years
Incidence and severity of treatment-emergent Serious AEs (SAEs)
Medidas secundarias
Number of patients who experience at least one AESI and number of patients by AESI
Plazo: 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
Plazo: 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
Plazo: 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
Plazo: 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
Plazo: 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.
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (2)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
2 centros
Novartis Investigative SiteCuritiba, Paraná, BrazilReclutando
Novartis Investigative SiteSão Paulo, São Paulo, BrazilReclutando
Equipo y fechas
- Patrocinador principal
- Novartis Pharmaceuticals
- Inicio del estudio
- 27 nov 2023 Real
- Finalización primaria
- 2 ene 2042 Estimado
- Finalización del estudio
- 2 ene 2042 Estimado
- Primera publicación
- 31 ago 2023 Real
- Última actualización
- 10 nov 2026 Real
Contactos oficiales del estudio
Título oficial del estudio
A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
Consulta a Wellness Clinical Research
Tu mensaje va a WCR, incluso si otra organización realiza este estudio. WCR puede orientarte, pero podría no realizar este estudio, tener acceso a su equipo ni poder inscribirte.
Fuente: API v2 de ClinicalTrials.gov. Patrocinadores e investigadores envían la información. La NLM no aprueba la seguridad ni la ciencia de todos los estudios registrados. Aviso del registro.
Consultado: 12 nov 2026 · Última actualización del registro: 10 nov 2026