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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Spinal Muscular Atrophy, Neuromuscular Disorders

Intervention / Treatment
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* Other: Direct current stimulation of cervical spinal cord


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent 5q SMA confirmed by molecular testing Exclusion Criteria: Need for ventilation Hypersensitivity (pain or allergic reaction) to current stimulation

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Sham Comparator: Zero-strength of direct current stimulation<br>Sham transdermal direct current stimulation of cervical spinal cord | Other: Direct current stimulation of cervical spinal cord<br>* 10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord<br>|
| Experimental: 100 microamperes direct current stimulation<br>Transdermal direct current stimulation of cervical spinal cord | Other: Direct current stimulation of cervical spinal cord<br>* 10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord<br>|
| Experimental: 1 milliampere direct current stimulation<br>Transdermal direct current stimulation of cervical spinal cord | Other: Direct current stimulation of cervical spinal cord<br>* 10 minutes direct current stimulation of 0, 100 microamperes, 1 milliampere strengths applied through dermal electrodes to cervical spinal cord<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity |  | Before and 0, 15, 30 minutes after spinal cord direct current stimulation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Short time fluctuations of Hammersmith Functional Motor Scale indexes |  | Three times, three days consecutive measurement, every two months, assessed up to 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Spinal muscular atrophy, Neuromuscular disorders, Outcome measures, Visit-free clinical trial design