NCT07737756Not yet recruiting
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia
Spinal Muscular Atrophy Center-based REAL World Retrospective Medical Chart Review of Patient Treated With Onasemnogene Abeparvovec (Zolgensma®) in Saudi Arabia
Source: ClinicalTrials.gov · Last updated 15 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Study type
- Observational
- Enrollment
- 6 (estimated)
- First submitted
- 27 Jul 2026
- Start date
- 1 Nov 2026
- Primary completion date
- 1 Feb 2027
- Lead sponsor
- Novartis Pharmaceuticals
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07737756
What you commit to
- Study type: Observational
- Planned participants: 6
- From 1 Nov 2026 to the primary completion date, 1 Feb 2027
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- 1\. Any patient who does not fulfill any of the inclusion criteria listed above.
Who the study is looking for
- 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.
Age in the registry: 14 days to 2 years · Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.