A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Spinal Muscular Atrophy. Basic parameters: 14 Days — 2 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
Overview
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.
Primary outcome measures
- Proportion of SMA Type 1 Non-Sitters Patients Achieving Independent Sitting for ≥30 Seconds at Any Visit up to 12 Months After OA Administration [Time frame: Up to 12 months]
Secondary outcome measures (10)
- Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score [Time frame: Up to approximately 3 years]
- Change From Baseline in CHOP-INTEND Score [Time frame: Baseline, up to approximately 3 years]
- Hammersmith Infant Neurological Examination-Section 2 (HINE-2, Motor Milestones) Score [Time frame: Up to approximately 3 years]
- Change From Baseline in HINE-2 Score [Time frame: Baseline, up to approximately 3 years]
- Percentage of Patients who Maintain the Ability to Thrive at 12 Months After OA Treatment [Time frame: 12 months]
- Number and Percentage of Patients Without Permanent Ventilatory Support or Death After OA Administration [Time frame: Up to approximately 3 years]
- Number and Percentage of Patients With Adverse Events [Time frame: Up to approximately 3 years]
- Number and Percentage of Patients by Demographic and Clinical Characteristics [Time frame: Baseline]
- Age [Time frame: Baseline]
- Duration Between Symptom Onset and Diagnosis [Time frame: Baseline]
Eligibility criteria
Inclusion criteria
- 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 2023.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07737756 · COAV101A1SA01