Spinal Muscular Atrophy Clinical Trial
Official title:
Assessment of Feasibility and Statistical Reliability of Functional Outcomes Measurement in Neuromuscular Patients Without Trial Site Visiting by Standard Functional Scales and by Special Autonomic Device in Double-blind, Placebo Controlled Study of Cervical Spinal Cord Transdermal Direct Current Stimulation in Patients With Spinal Muscular Atrophy
Clinical trials organization in several neuromuscular disorders (NMD) has some specific
issues. Nonambulant status and difficulties with transportation are among them. Moreover a
lot of patients with NMD have so poor condition that even short transportation is able to
worse it. Such situation forces researchers to limit a region of recruitment for clinical
trials and to exclude from trials more severe subgroup of patients, which cause additional
issues especially for rare diseases.
The purpose of this study is to prove hypothesis about possibility to reliably monitor
patient condition remotely, without trial site visiting. Visit-free study design is
potentially able to widen eligible patient population and to decrease patient dropout rate as
well as burden of numerous assessments. Meanwhile assessment frequency could be increased
enabling monitoring of short fluctuations in patients' condition.
Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above
issues are completely applicable. Direct current stimulation (DCS) of neural structures is
well studied and safe intervention, however, its effects on SMA patients' strength and
durability has not been reported for today. The investigators suppose that investigation of
DCS action in SMA patient population is an adequate model for visit-free design feasibility,
reliability and sensitivity evaluation.
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 |
NCT06300996 -
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb
|
N/A | |
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 |
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 | |
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
|