Spinal Muscular Atrophy Clinical Trial
Official title:
Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders
The primary objective of the study is to explore the convergent validity of smartphone-based Konectom DOAs against in-clinic standard assessments. The secondary objectives of this study are to evaluate the test-retest reliability of smartphone-based Konectom Digital Outcome Assessments (DOAs); to determine the relationship between Konectom upper limb DOAs and conventional upper limb assessments in clinical environments; to determine the relationship between Konectom lower limb DOAs and status of ambulation in clinical environments; to evaluate group differences in smartphone-based Konectom DOAs [self-administered at home and in-clinic] between person with spinal muscular atrophy (PwSMA) and healthy subjects (HS); to evaluate the variability of Konectom DOAs self-administered in everyday environment in HS and PwSMA; to compare Konectom DOAs between in-clinic supervised administration versus self-assessments in everyday environment in HS, PwSMA groups; to evaluate the relationship of Konectom DOAs against patient-reported outcomes (PROs) in PwSMA and to evaluate the clinical safety of Konectom in PwSMA.
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 | |
| 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
|
||
| Recruiting |
NCT03300869 -
Natural History of Types 2 and 3 SMA in Taiwan
|
||
| 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
|