A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
NCT07474311 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 213
Last updated 2026-03-20
Summary
The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
Conditions
- Muscular Atrophy, Spinal
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Novartis Pharmaceuticals · Novartis Pharmaceuticals
Eligibility
- Max Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2011-11-22
- Primary Completion
- 2025-03-13
- Completion
- 2025-03-13
Countries
- Czechia
Study Locations
More Related Trials
-
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
NCT06321965 ·Status: RECRUITING ·Phase: NA
-
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
NCT04851873 ·Status: COMPLETED ·Phase: PHASE3
-
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
NCT05475691 ·Status: ACTIVE_NOT_RECRUITING
-
Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study
NCT05839145 ·Status: NOT_YET_RECRUITING ·Phase: NA
-
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
NCT02193074 ·Status: TERMINATED ·Phase: PHASE3
-
Metabolomics of Children With SMA
NCT04587492 ·Status: COMPLETED
-
Infants With Spinal Muscular Atrophy Type I
NCT01547871 ·Status: TERMINATED
-
Pediatric Spinal Muscular Atrophy (SMA) China Registry
NCT05042921 ·Status: ACTIVE_NOT_RECRUITING
-
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
NCT07070999 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Mechanisms of Cell Death in Spinal Muscular Atrophy
NCT01754441 ·Status: COMPLETED
-
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
NCT03400852 ·Status: TERMINATED ·Phase: PHASE2
-
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
NCT06747884 ·Status: RECRUITING
-
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
NCT02644668 ·Status: COMPLETED ·Phase: PHASE2
-
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
NCT05768048 ·Status: RECRUITING
-
A Study of NIDO-361 in Patients With SBMA
NCT06411912 ·Status: COMPLETED ·Phase: PHASE2
-
Defining Endpoints in Becker Muscular Dystrophy
NCT05257473 ·Status: ACTIVE_NOT_RECRUITING
-
A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
NCT00568698 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
NCT07265232 ·Status: RECRUITING ·Phase: PHASE3
-
A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD
NCT01437345 ·Status: COMPLETED
-
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
NCT05794139 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2
-
Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
NCT03554343 ·Status: COMPLETED
-
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
NCT03339830 ·Status: COMPLETED
-
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
NCT03381729 ·Status: TERMINATED ·Phase: PHASE1
-
Spinal Muscular Atrophy Neonatal Screening Program
NCT06310421 ·Status: RECRUITING
-
A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy
NCT07475754 ·Status: NOT_YET_RECRUITING ·Phase: NA