NCT02235090

Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting

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

• Status: Withdrawn
• Phase: N/A
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Charitable Foundation Children with Spinal Muscular Atrophy · Academic / Other
• Sex: All
• Age: 5 Years
• Healthy volunteers: Accepted

Summary

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.

Conditions

• Spinal Muscular Atrophy
• Neuromuscular Disorders

Interventions

Type	Name	Description
OTHER	Direct current stimulation of cervical spinal cord	10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord

Timeline

• Start date: 2016-10-01
• Primary completion: 2017-09-30
• Completion: 2017-10-30
• First posted: 2014-09-09
• Last updated: 2019-02-15

Locations

1 site across 1 country: Ukraine