Pediatric Spinal Muscular Atrophy (SMA) China Registry

Biogen

Status: Active, not recruiting

Conditions: Muscular Atrophy, Spinal

Study type: Observational

Funder types: Industry

Identifiers

NCT05042921

CN-SMG-11774

Details and patient eligibility

About

The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.

Enrollment: 600 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
• Genetically confirmed 5q-SMA.
• Age < 18 years at enrollment.

Key Exclusion Criteria:

• Unable or unwilling to provide informed consent.
• Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined inclusion/ exclusion criteria may apply.

Trial design

600 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.