Pediatric Spinal Muscular Atrophy (SMA) China Registry

Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05042921

Enrollment

600

Registered

2021-09-13

Start date

2021-11-18

Completion date

2027-09-02

Last updated

2026-09-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Muscular Atrophy, Spinal

Brief summary

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Years

Healthy volunteers

Inclusion 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/

Design outcomes

Primary

Measure	Time frame
Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)	Up to 60 months

Countries

China

Contacts

STUDY_DIRECTORMedical Director

Biogen

Outcome results