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

Novartis

Status

Completed

Conditions

Spinal Muscular Atrophy

Study type

Observational

Funder types

Industry

Identifiers

NCT06178393

COAV101A12402

Details and patient eligibility

About

This global, retrospective, non-interventional, medical chart review (MCR), descriptive study collected patient-level data in regions outside the US.

The study required a repeated data collection at follow-up dates from start of treatment with nusinersen, onasemnogene abeparvovec-xioi (OA), and/or risdiplam. At the start of data collection, the study team reached out to the health care providers (HCPs) involved in treating pediatric SMA patients for participating in this study. The physicians across the participating countries conducted a retrospective MCR of pediatric patients diagnosed with SMA who were treated with at least 1of the 3 novel disease-modifying treatments (DMTs): nusinersen, OA, and/or risdiplam.

All health care encounters data i.e., emergency and inpatient admissions, surgery, and outpatient consultations of recruited patients, including their treatment with nusinersen, OA, and/or risdiplam, were abstracted to understand the treatment patterns as per routine clinical practice for SMA management globally. The first date of initial administration of 1 of the 3 target drugs was used as the "index date." Based on this, the record abstraction was performed through a retrospective MCR during the pre-index period, at index date and in the post-index period.

Enrollment

162 patients

Sex

All

Ages

Under 5 years old

Volunteers

No Healthy Volunteers

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