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

• Sponsor: Charitable Foundation Children with Spinal Muscular Atrophy (Other)
• Overall Status: Withdrawn
• CT.gov ID: NCT02235090
• Collaborator: (none)
• Enrollment: 0
• Location: 1
• Arms: 3
• Actual Duration (Months): 12.9
• Patients Per Site Per Month: 0

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Spinal Muscular Atrophy; Neuromuscular Disorders	Other: Direct current stimulation of cervical spinal cord	N/A

Study Design

• Study Type: Interventional
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Other
• 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
• Actual Study Start Date: Oct 1, 2016
• Actual Primary Completion Date: Sep 30, 2017
• Actual Study Completion Date: Oct 30, 2017

Arms and Interventions

Arm	Intervention/Treatment
Sham Comparator: Zero-strength of direct current stimulation Sham transdermal direct current stimulation of cervical spinal cord	Other: 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
Experimental: 100 microamperes direct current stimulation Transdermal direct current stimulation of cervical spinal cord	Other: 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
Experimental: 1 milliampere direct current stimulation Transdermal direct current stimulation of cervical spinal cord	Other: 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

Outcome Measures

Primary Outcome Measures

1. 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

1. Short time fluctuations of Hammersmith Functional Motor Scale indexes [Three times, three days consecutive measurement, every two months, assessed up to 6 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"	Kharkiv		Ukraine	61068

Sponsors and Collaborators

• Charitable Foundation Children with Spinal Muscular Atrophy

Investigators

• Principal Investigator: Andriy V. Shatillo, MD, PhD, State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Charitable Foundation Children with Spinal Muscular Atrophy
• ClinicalTrials.gov Identifier: NCT02235090
• Other Study ID Numbers: CSMA-INPN-1
• First Posted: Sep 9, 2014
• Last Update Posted: Feb 15, 2019
• Last Verified: Feb 1, 2019

• Keywords provided by Charitable Foundation Children with Spinal Muscular Atrophy: Spinal muscular atrophy; Neuromuscular disorders; Outcome measures; Visit-free clinical trial design
• Additional relevant MeSH terms: Muscular Atrophy; Muscular Atrophy, Spinal; Neuromuscular Diseases; Atrophy

Study Results

No Results Posted as of Feb 15, 2019