Spinal Muscular Atrophy Clinical Trial
Official title:
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is caused by the homozygous loss of the Survival Motor Neuron (SMN) 1 gene, which leads to degeneration of spinal alpha-motor neurons and muscle atrophy. Three treatments have been approved for SMA but the available data show interpatient variability in therapy response and, to date, individual factors such as age or SMN2 copies,cannot fully explain this variance. The aim of this project is: - collect clinical data and patient-reported outcome measures (PROM) from patients treated with nusinersen, risdiplam, onasemnogene abeparvovec, - identify novel biomarkers and RNA molecular signature profiling, - develop a predictive algorithm using artificial intelligence (AI) methodologies based on machine learning (ML), able to integrate clinical outcomes, patients' characteristics, and specific biomarkers. This effort will help to better stratify the SMA patients and to predict their therapeutic outcome, thus to address patients towards personalized therapies.
n/a
| 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 | |
| Completed |
NCT02003937 -
Aerobic Training in Patients With Spinal Muscular Atrophy Type III
|
N/A | |
| Not yet recruiting |
NCT00961103 -
Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
|
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 |
NCT03300869 -
Natural History of Types 2 and 3 SMA in Taiwan
|
||
| 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
|