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

Not Applicable

Withdrawn

• Conditions: Spinal Muscular Atrophy; Neuromuscular Disorders
• Interventions: Other: Direct current stimulation of cervical spinal cord

• Registration Number: NCT02235090
• Lead Sponsor: Charitable Foundation Children with Spinal Muscular Atrophy

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-09-03
• Study First Submitted QC: 2014-09-05
• Study First Posted: 2014-09-09
• Study Start: 2016-10
• Primary Completion: 2017-09-30
• Study Completion: 2017-10-30
• Status Verified: 2019-02
• Last Update Submitted: 2019-02-12
• Last Update Posted: 2019-02-15

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Signed informed consent
• 5q SMA confirmed by molecular testing

Exclusion Criteria

• Need for ventilation
• Hypersensitivity (pain or allergic reaction) to current stimulation

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1 milliampere direct current stimulation	Direct current stimulation of cervical spinal cord	Transdermal direct current stimulation of cervical spinal cord
Zero-strength of direct current stimulation	Direct current stimulation of cervical spinal cord	Sham transdermal direct current stimulation of cervical spinal cord
100 microamperes direct current stimulation	Direct current stimulation of cervical spinal cord	Transdermal direct current stimulation of cervical spinal cord

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Short time fluctuations of Hammersmith Functional Motor Scale indexes	Three times, three days consecutive measurement, every two months, assessed up to 6 months

Trial Locations

Locations (1)

SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"; 🇺🇦 Kharkiv, Ukraine