A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
Spinal Muscular Atrophy
De qué trata este estudio
El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.
- Tipo de estudio
- Observacional
- Fase
- No aplica / no indicada
- Rango de edad
- 14 días - 2 años
- Sexo admitido
- Todos los sexos
- Voluntarios sanos
- No aceptados
- Participantes
- 6 Estimado
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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
The aim of this retrospective medical chart review is to describe the clinical outcomes, clinical characteristics, and demographics of patients with spinal muscular atrophy (SMA) type 1 treated with onasemnogene abeparvovec (OA) at a single clinical center in Saudi Arabia. The study will use secondary data collected from the electronic medical records of SMA type 1 patients.
¿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:
1. Patients with a genetically confirmed diagnosis of SMA type 1 who were treated with OA.
2. Availability of data on at least one visit before treatment initiation and two visits post-treatment is a must for inclusion.
3. Patients who were treated with OA starting in January 2023.
4. Patients with at least 3 months of follow-up following the treatment with OA.
Exclusion criteria:
1\. Any patient who does not fulfill any of the inclusion criteria listed above.
Población del estudio
SMA type 1 patients receiving OA at a single clinical center in Saudi Arabia.
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
OA Cohort
Patients with SMA type 1 who were treated with OA.
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Proportion of SMA Type 1 Non-Sitters Patients Achieving Independent Sitting for ≥30 Seconds at Any Visit up to 12 Months After OA Administration
Plazo: Up to 12 months
No informado en este registro.
Medidas secundarias
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
Plazo: Up to approximately 3 years
CHOP-INTEND is a validated motor function assessment for infants with SMA. The total score ranges from 0 to 64, with higher scores indicating better motor function. Scores are derived from 16 items assessing spontaneous movement, strength, and motor abilities. An increase in score reflects improvement in motor function.
Change From Baseline in CHOP-INTEND Score
Plazo: Baseline, up to approximately 3 years
CHOP-INTEND is a validated motor function assessment for infants with SMA. The total score ranges from 0 to 64, with higher scores indicating better motor function. Scores are derived from 16 items assessing spontaneous movement, strength, and motor abilities. An increase in score reflects improvement in motor function.
Hammersmith Infant Neurological Examination-Section 2 (HINE-2, Motor Milestones) Score
Plazo: Up to approximately 3 years
The HINE-2 is a motor milestone assessment evaluating developmental abilities in infants. The total score ranges from 0 to 26, with higher scores indicating greater achievement of motor milestones. The scale assesses milestones such as head control, sitting, rolling, crawling, standing, and walking. Higher scores correspond to more advanced motor development.
Change From Baseline in HINE-2 Score
Plazo: Baseline, up to approximately 3 years
The HINE-2 is a motor milestone assessment evaluating developmental abilities in infants. The total score ranges from 0 to 26, with higher scores indicating greater achievement of motor milestones. The scale assesses milestones such as head control, sitting, rolling, crawling, standing, and walking. Higher scores correspond to more advanced motor development.
Percentage of Patients who Maintain the Ability to Thrive at 12 Months After OA Treatment
Plazo: 12 months
Ability to thrive is defined as meeting the following criteria:
- Ability to tolerate thin liquids, as demonstrated by a formal swallowing assessment, with tested consistency classified as "very thin" or "thin" and results recorded as "normal swallow," "functional swallow," or "safe for swallowing";
- No requirement for nutritional support via mechanical feeding methods (e.g., feeding tube);
- Maintenance of body weight at or above the 3rd percentile for age and sex, according to World Health Organization (WHO) child growth standards, appropriate for the child's age at assessment.
Number and Percentage of Patients Without Permanent Ventilatory Support or Death After OA Administration
Plazo: Up to approximately 3 years
Event-free survival of patients without permanent ventilatory support or death after OA administration.
Permanent ventilatory support is defined as the requirement for either:
- Tracheostomy, or
- ≥16 hours per day of respiratory support (including non-invasive ventilation) for 14 or more consecutive days, in the absence of an acute reversible illness and excluding perioperative ventilation.
Number and Percentage of Patients With Adverse Events
Plazo: Up to approximately 3 years
No informado en este registro.
Number and Percentage of Patients by Demographic and Clinical Characteristics
Plazo: Baseline
Demographic and clinical characteristics include:
- Sex
- City of residence
- Preterm birth status
- Functional classification
- Developmental milestones achieved prior to treatment initiation
- SMA-related symptoms
- Comorbidities
- CHOP-INTEND Score
- HINE-2 Score
- Ambulatory status
- Swallowing assessment result
- Feeding method
- Anthropometric measures
Age
Plazo: Baseline
Age, gestational age at birth, age at symptom onset, and age at SMA type 1 diagnosis.
Duration Between Symptom Onset and Diagnosis
Plazo: Baseline
No informado en este registro.
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (0)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
No hay centros indicados. Usa el contacto oficial del estudio.
Equipo y fechas
- Patrocinador principal
- Novartis Pharmaceuticals
- Inicio del estudio
- 30 nov 2026 Estimado
- Finalización primaria
- 28 feb 2027 Estimado
- Finalización del estudio
- 28 feb 2027 Estimado
- Primera publicación
- 30 jul 2026 Real
- Última actualización
- 10 nov 2026 Real
Contactos oficiales del estudio
Título oficial del estudio
Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
Consulta a Wellness Clinical Research
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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