We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02235090
First Posted: September 9, 2014
Last Update Posted: September 20, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Charitable Foundation Children with Spinal Muscular Atrophy
  Purpose

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 Intervention
Spinal Muscular Atrophy Neuromuscular Disorders Other: Direct current stimulation of cervical spinal cord

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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

Resource links provided by NLM:


Further study details as provided by Charitable Foundation Children with Spinal Muscular Atrophy:

Primary Outcome Measures:
  • 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 Measures:
  • 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 ]

Estimated Enrollment: 30
Study Start Date: August 2016
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02235090


Locations
Ukraine
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"
  More Information

Additional Information:
Responsible Party: Charitable Foundation Children with Spinal Muscular Atrophy
ClinicalTrials.gov Identifier: NCT02235090     History of Changes
Other Study ID Numbers: CSMA-INPN-1
First Submitted: September 3, 2014
First Posted: September 9, 2014
Last Update Posted: September 20, 2016
Last Verified: September 2016

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:
Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Neuromuscular Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases