Adult Spinal Muscular Atrophy (SMA) China Registry

Biogen

Status: Active, not recruiting

Conditions: Muscular Atrophy, Spinal

Study type: Observational

Funder types: Industry

Identifiers

NCT05618379

CN-SMG-11772

Details and patient eligibility

About

The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).

Enrollment: 200 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

-Genetically confirmed 5q-SMA.

Key Exclusion Criteria:

-Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

200 participants in 2 patient groups

DMT Treated Participants

Description:

Participants with SMA who received prior treatment with DMTs including nusinersen will be followed prospectively for up to 60 months and the available data is collected retrospectively.

Untreated Participants

Description:

Participants with SMA who received no treatment will be followed prospectively for up to 60 months.