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

Spinal Muscular Atrophy Clinical Trial

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02235090
• Other study ID #: CSMA-INPN-1
• Status: Withdrawn
• Phase: N/A
• Start date: October 2016
• Est. completion date: October 30, 2017

Study information

• Verified date: February 2019
• Source: Charitable Foundation Children with Spinal Muscular Atrophy
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 30, 2017
• Est. primary completion date: September 30, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 5 Years and older

Eligibility

Inclusion Criteria:

• Signed informed consent

• 5q SMA confirmed by molecular testing

Exclusion Criteria:

• Need for ventilation

• Hypersensitivity (pain or allergic reaction) to current stimulation

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Neuromuscular Diseases
• Neuromuscular Disorders
• Spinal Muscular Atrophy

Intervention

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

Locations

Country	Name	City	State
Ukraine	SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"	Kharkiv

Sponsors (1)

Lead Sponsor	Collaborator
Charitable Foundation Children with Spinal Muscular Atrophy

Country where clinical trial is conducted

Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Short time fluctuations of Hammersmith Functional Motor Scale indexes		Three times, three days consecutive measurement, every two months, assessed up to 6 months