Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

Plain-language summary

This study evaluates the real-world use of novel treatments such as nusinersen, onasemnogene abeparvovec and risdiplam in children with spinal muscular atrophy (SMA), through a retrospective chart review at sites outside the United States. It reviewed records of children under 5 years of age with a genetically confirmed SMA diagnosis. The study was conducted as a non-phase observational study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Novartis Pharmaceuticals

Start date

5/5/2022

Estimated completion

12/30/2022

Countries

United States

Related genes

Locations

• Bannockburn, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion criteria:

* Genetically confirmed diagnosis of SMA.
* Aged less than 5 years at index date.
* Initiated treatment with nusinersen or onasemnogene abeparvovec-xioi or risdiplam or any combination of them during the identification period.
* Availability of medical information in chart for at least 1 visit prior to initiation of treatment with the target treatment(s).

Exclusion criteria:

None