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Withdrawn

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02235090

Enrollment

Registered

2014-09-09

Start date

2016-10-31

Completion date

2017-10-30

Last updated

2019-02-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuromuscular Disorders, Spinal Muscular Atrophy

Keywords

Spinal muscular atrophy, Neuromuscular disorders, Outcome measures, Visit-free clinical trial design

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.

Interventions

OTHERDirect 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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

OTHER

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

5 Years to No maximum

Healthy volunteers

Yes

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

Measure	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

Measure	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

Countries

Ukraine

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026