A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients
14 de septiembre de 2026 actualizado por: Novartis Pharmaceuticals
Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)
This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.
This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.
Descripción general del estudio
Estado
Aún no reclutando
Condiciones
Tipo de estudio
De observación
Inscripción (Estimado)
80
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Estudio Contacto
- Nombre: Novartis Pharmaceuticals
- Número de teléfono: +41613241111
- Correo electrónico: novartis.email@novartis.com
Copia de seguridad de contactos de estudio
- Nombre: Novartis Pharmaceuticals
Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
No
Método de muestreo
Muestra no probabilística
Población de estudio
SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.
Descripción
Inclusion criteria:
• All patients treated with onasemnogene abeparvovec.
Exclusion criteria:
• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
Cohortes e Intervenciones
Grupo / Cohorte |
|---|
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Onasemnogene Abeparvovec Intrathecal Group
SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
Periodo de tiempo: Up to approximately 4 years and 5 months
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Up to approximately 4 years and 5 months
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Incidence of Serious Adverse Events and ADRs
Periodo de tiempo: Up to approximately 4 years and 5 months
|
Incidence based on the person-year method will be calculated.
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Up to approximately 4 years and 5 months
|
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Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Periodo de tiempo: Up to approximately 4 years and 5 months
|
Incidence based on the person-year method will be calculated.
|
Up to approximately 4 years and 5 months
|
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Number of Patients by Time to Onset of Serious Adverse Events and ADRs
Periodo de tiempo: Up to approximately 4 years and 5 months
|
Up to approximately 4 years and 5 months
|
|
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Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Periodo de tiempo: Up to approximately 4 years and 5 months
|
Up to approximately 4 years and 5 months
|
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Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
Periodo de tiempo: Up to approximately 4 years and 5 months
|
Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
|
Up to approximately 4 years and 5 months
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression.
The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation).
Total scores range from 0-66.
Higher scores indicate higher levels of motor ability.
|
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
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Change From Baseline in Revised Upper Limb Module (RULM) Score
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows:
|
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
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Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA.
It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task).
CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.
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Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
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Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from:
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Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
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Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score
Periodo de tiempo: 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
The CGI-I scale is a physician-rated measure used to assess a patient's overall clinical status. The patient's overall clinical status is scored on a 7-point scale of the extent of change relative to before treatment initiation as follows:
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6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
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Number of Patients Achieving Each Developmental Milestone
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
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Developmental milestones include:
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Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
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Number of Patients Maintaining Each Developmental Milestone
Periodo de tiempo: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
|
Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Investigadores
- Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Estimado)
30 de octubre de 2026
Finalización primaria (Estimado)
31 de marzo de 2031
Finalización del estudio (Estimado)
31 de marzo de 2031
Fechas de registro del estudio
Enviado por primera vez
11 de agosto de 2026
Primero enviado que cumplió con los criterios de control de calidad
11 de agosto de 2026
Publicado por primera vez (Actual)
17 de agosto de 2026
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
17 de septiembre de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
14 de septiembre de 2026
Última verificación
1 de septiembre de 2026
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- COAV101B1401
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
NO
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