Trials / Active Not Recruiting
Active Not RecruitingNCT05042921
Pediatric Spinal Muscular Atrophy (SMA) China Registry
Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
- Status
- Active Not Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 600 (actual)
- Sponsor
- Biogen · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Timeline
- Start date
- 2021-11-18
- Primary completion
- 2027-09-02
- Completion
- 2027-09-02
- First posted
- 2021-09-13
- Last updated
- 2025-09-03
Locations
25 sites across 1 country: China
Source: ClinicalTrials.gov record NCT05042921. Inclusion in this directory is not an endorsement.