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.
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