Clinical trial NCT02235090: 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
Overall status: WITHDRAWN
Phase: NA
Conditions: Spinal Muscular Atrophy
Neuromuscular Disorders
Keywords: Spinal muscular atrophy
Neuromuscular disorders
Outcome measures
Visit-free clinical trial design
Lead sponsor: Charitable Foundation Children with Spinal Muscular Atrophy
Sponsor class: OTHER
First posted: 2014-09-09
Enrollment: 0
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.


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

Primary outcomes:
measure: Strength Changes from baseline measured by handheld myometry after spinal cord direct current stimulation of different intensity
timeFrame: Before and 0, 15, 30 minutes after spinal cord direct current stimulation
Secondary outcomes:
measure: Short time fluctuations of Hammersmith Functional Motor Scale indexes
timeFrame: Three times, three days consecutive measurement, every two months, assessed up to 6 months
Other outcomes:
Locations:
city: Kharkiv
country: Ukraine
facility: SI "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
Study officials:
affiliation: State Institution "Institute of Neurology, Psychiatry and Narcology of NAMS of Ukraine"
name: Andriy V. Shatillo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: