Pediatric Spinal Muscular Atrophy (SMA) China Registry
NCT05042921 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 600
Last updated 2025-09-03
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
- Muscular Atrophy, Spinal
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Medical Director · Biogen
Eligibility
- Max Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-11-18
- Primary Completion
- 2027-09-02
- Completion
- 2027-09-02
Countries
- China
Study Locations
More Related Trials
-
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
NCT05901987 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
NCT06191354 ·Status: ACTIVE_NOT_RECRUITING ·Phase: NA
-
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
NCT04174157 ·Status: RECRUITING
-
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
NCT06562283 ·Status: RECRUITING ·Phase: NA
-
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
NCT03760029 ·Status: COMPLETED ·Phase: NA
-
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
NCT05475691 ·Status: ACTIVE_NOT_RECRUITING
-
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
NCT06532474 ·Status: RECRUITING
-
Natural History of Types 2 and 3 SMA in Taiwan
NCT03300869 ·Status: UNKNOWN
-
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
NCT06288230 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
UK SMA Patient Registry
NCT04292574 ·Status: RECRUITING
-
Natural History of SMA
NCT05755451 ·Status: RECRUITING
-
Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
NCT02044029 ·Status: COMPLETED
-
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
NCT06421831 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
NCT05839145 ·Status: NOT_YET_RECRUITING ·Phase: NA
-
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
NCT05768048 ·Status: RECRUITING
-
Infants With Spinal Muscular Atrophy Type I
NCT01547871 ·Status: TERMINATED
-
Mechanisms of Cell Death in Spinal Muscular Atrophy
NCT01754441 ·Status: COMPLETED
-
Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA
NCT05866939 ·Status: UNKNOWN
-
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
NCT06321965 ·Status: RECRUITING ·Phase: NA
-
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
NCT05335876 ·Status: RECRUITING ·Phase: PHASE3
-
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
NCT01166022 ·Status: COMPLETED ·Phase: NA
-
Amyotrophic Lateral Sclerosis Registry
NCT05892822 ·Status: UNKNOWN
-
Measuring Levels of SMN in Blood Samples of SMA Patients
NCT00061607 ·Status: COMPLETED
-
International SMA Patient Registry
NCT00466349 ·Status: COMPLETED