NCT07737756招募中不适用
Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Proportion of SMA Type 1 Non-Sitters Patients Achieving Independent Sitting for ≥30 Seconds at Any Visit up to 12 Months After OA Administration
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 14 Days 至 2 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with a genetically confirmed diagnosis of SMA type 1 who were treated with OA.
- •Availability of data on at least one visit before treatment initiation and two visits post-treatment is a must for inclusion.
- •Patients who were treated with OA starting in January
- •Patients with at least 3 months of follow-up following the treatment with OA.
排除标准
- •1. Any patient who does not fulfill any of the inclusion criteria listed above.
研究组 & 干预措施
OA Cohort
Patients with SMA type 1 who were treated with OA.
结局指标
主要结局
Proportion of SMA Type 1 Non-Sitters Patients Achieving Independent Sitting for ≥30 Seconds at Any Visit up to 12 Months After OA Administration
时间窗: Up to 12 months
次要结局
- Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score(Up to approximately 3 years)
- Change From Baseline in CHOP-INTEND Score(Baseline, up to approximately 3 years)
- Hammersmith Infant Neurological Examination-Section 2 (HINE-2, Motor Milestones) Score(Up to approximately 3 years)
- Change From Baseline in HINE-2 Score(Baseline, up to approximately 3 years)
- Percentage of Patients who Maintain the Ability to Thrive at 12 Months After OA Treatment(12 months)
- Number and Percentage of Patients Without Permanent Ventilatory Support or Death After OA Administration(Up to approximately 3 years)
- Number and Percentage of Patients With Adverse Events(Up to approximately 3 years)
- Number and Percentage of Patients by Demographic and Clinical Characteristics(Baseline)
- Age(Baseline)
- Duration Between Symptom Onset and Diagnosis(Baseline)
研究者
研究点 (1)
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