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

Primary Purpose

Spinal Muscular Atrophy, Neuromuscular Disorders

Status

Withdrawn

Phase

Not Applicable

Locations

Ukraine

Study Type

Interventional

Intervention

Direct current stimulation of cervical spinal cord

About this trial

This is an interventional other trial for Spinal Muscular Atrophy focused on measuring Spinal muscular atrophy, Neuromuscular disorders, Outcome measures, Visit-free clinical trial design

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

Signed informed consent
5q SMA confirmed by molecular testing

Exclusion Criteria:

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

Sites / Locations

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

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Sham Comparator

Experimental

Arm Label

Zero-strength of direct current stimulation

100 microamperes direct current stimulation

1 milliampere direct current stimulation

Arm Description

Sham transdermal direct current stimulation of cervical spinal cord

Transdermal direct current stimulation of cervical spinal cord

Outcomes

Primary Outcome Measures

Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity

Secondary Outcome Measures

Short time fluctuations of Hammersmith Functional Motor Scale indexes

Full Information

NCT ID

NCT02235090

First Posted

September 3, 2014

Last Updated

February 12, 2019

Sponsor

Charitable Foundation Children with Spinal Muscular Atrophy

1. Study Identification

Unique Protocol Identification Number

NCT02235090

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2019

Overall Recruitment Status

Withdrawn

Why Stopped

Trial was not started due to lack of funding and nobody received an intervention.

Study Start Date

October 2016 (Actual)

Primary Completion Date

September 30, 2017 (Actual)

Study Completion Date

October 30, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Charitable Foundation Children with Spinal Muscular Atrophy

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Spinal Muscular Atrophy, Neuromuscular Disorders

Keywords

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

7. Study Design

Primary Purpose

Other

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Zero-strength of direct current stimulation

Arm Type

Sham Comparator

Arm Description

Sham transdermal direct current stimulation of cervical spinal cord

Arm Title

100 microamperes direct current stimulation

Arm Type

Experimental

Arm Description

Transdermal direct current stimulation of cervical spinal cord

Arm Title

1 milliampere direct current stimulation

Arm Type

Experimental

Arm Description

Transdermal direct current stimulation of cervical spinal cord

Intervention Type

Other

Intervention Name(s)

Direct current stimulation of cervical spinal cord

Intervention Description

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

Primary Outcome Measure Information:

Title

Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity

Time Frame

Before and 0, 15, 30 minutes after spinal cord direct current stimulation

Secondary Outcome Measure Information:

Title

Short time fluctuations of Hammersmith Functional Motor Scale indexes

Time Frame

Three times, three days consecutive measurement, every two months, assessed up to 6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Andriy V. Shatillo, MD, PhD

Organizational Affiliation

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

Official's Role

Principal Investigator

Facility Information:

Facility Name

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

City

Kharkiv

ZIP/Postal Code

61068

Country

Ukraine

12. IPD Sharing Statement

Learn more about this trial

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

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