Spinal Muscular Atrophy Clinical Trial
Official title:
Natural History of Types 2 and 3 Spinal Muscular Atrophy in Taiwan
The primary objective of this study is to investigate the natural history of spinal muscular atrophy (SMA) types 2 and 3 patients in Taiwan. This study will provide further insights into the clinical course SMA. Several analyses will be conducted regarding overall survival, demographic characteristics, motor function, respiratory and nutritional support, and genotype and phenotype correlation.
As with other rare diseases, individual groups of SMA have therefore opted to share patient information in the form of clinical sites to increase the overall patient cohorts on which clinical outcomes and new assisted-healthcare technologies can be assessed. Using the collaborative and retrospective study of types 2 and 3 SMA patients in Taiwan, the investigators aim to 1) characterize the correlation of genotype and phenotype, 2) correlate the onset, progression, management with disease outcome, 3) depict comorbidity and within type 2 and 3 SMA patients with different SMN2 copy number. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04851873 -
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
|
Phase 3 | |
| Completed |
NCT03223051 -
Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy
|
N/A | |
| Completed |
NCT04335942 -
Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
|
N/A | |
| Recruiting |
NCT05794139 -
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
|
Phase 2 | |
| Not yet recruiting |
NCT06300996 -
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
|
N/A | |
| Not yet recruiting |
NCT00961103 -
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
|
N/A | |
| Completed |
NCT02003937 -
Aerobic Training in Patients With Spinal Muscular Atrophy Type III
|
N/A | |
| Completed |
NCT00227266 -
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
|
Phase 2 | |
| Completed |
NCT00374075 -
Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
|
Phase 1 | |
| Enrolling by invitation |
NCT05539456 -
Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
|
||
| Recruiting |
NCT05779956 -
Personalized Medicine for SMA: a Translational Project
|
||
| Recruiting |
NCT03217578 -
Neonatal Spinal Muscular Atrophy (SMA) Screening
|
||
| Completed |
NCT01703988 -
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
|
Phase 1/Phase 2 | |
| Withdrawn |
NCT02235090 -
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
|
N/A | |
| Completed |
NCT02123186 -
Newborn Screening for Spinal Muscular Atrophy
|
N/A | |
| Completed |
NCT00756821 -
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
|
N/A | |
| Completed |
NCT00004771 -
Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease
|
Phase 2 | |
| Recruiting |
NCT05366465 -
Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
|
||
| Recruiting |
NCT06310421 -
Spinal Muscular Atrophy Neonatal Screening Program
|
||
| Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|