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

NCT ID: NCT02235090

Last Updated: 2019-02-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

NA

Study Classification

INTERVENTIONAL

Study Start Date

2016-10-31

Study Completion Date

2017-10-30

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.

Detailed Description

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Conditions

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Spinal Muscular Atrophy Neuromuscular Disorders

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Zero-strength of direct current stimulation

Sham transdermal direct current stimulation of cervical spinal cord

Group Type SHAM_COMPARATOR

Direct current stimulation of cervical spinal cord

Intervention Type OTHER

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

100 microamperes direct current stimulation

Transdermal direct current stimulation of cervical spinal cord

Group Type EXPERIMENTAL

Direct current stimulation of cervical spinal cord

Intervention Type OTHER

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

1 milliampere direct current stimulation

Transdermal direct current stimulation of cervical spinal cord

Group Type EXPERIMENTAL

Direct current stimulation of cervical spinal cord

Intervention Type OTHER

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

Interventions

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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

Intervention Type OTHER

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
Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Charitable Foundation Children with Spinal Muscular Atrophy

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Andriy V. Shatillo, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"

Locations

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SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"

Kharkiv, , Ukraine

Site Status

Countries

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Ukraine

Other Identifiers

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CSMA-INPN-1

Identifier Type: -

Identifier Source: org_study_id

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