Plain English Summary
Background and study aims
Spinal muscular atrophy (SMA) is a rare inherited condition that causes muscle weakness and progressive (worsening over time) loss of movement. It is caused by deterioration in the nerve cells connecting the brain and spinal cord to the body’s muscles. As the link between the nerves and muscles breaks down, patients gradually lose their ability to move and their muscles waste away (atrophy). The aim of this study is to assess the strength of muscles in the trunk (torso) for rotational activity (twisting) and the level of independent functioning in children and youth with SMA.
Who can participate?
Patients aged 5-18 years who have been diagnosed with SMA and helathy children and youths of the same age.
What does the study involve?
All participants attend a single study visit at which the strength of their trunk (torso) is measured using a handheld measurement device while they are lying on their side. This is done at six points on the body. Following this, participants are asked to complete questionnaires about their daily activities and any limitations they may be facing as well as background information.
What are the possible benefits and risks of participating?
There are no direct benefits or risks involved with participating.
Where is the study run from?
Center of Functional Rehabilitation ORTHOS (Poland)
When is the study starting and how long is it expected to run for?
December 2016 to December 2017
Who is funding the study?
Józef Piłsudski University of Physical Education (Poland)
Who is the main contact?
Dr Agnieszka Stępień
Trial website
Contact information
Type
Scientific
Primary contact
Dr Agnieszka Stępień
ORCID ID
http://orcid.org/0000-0003-3905-8229
Contact details
Józef Piłsudski University of Physical Education
Marymoncka 34
Warsaw
00-968
Poland
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
1/2017
Study information
Scientific title
Strength of trunk rotational muscles and limitations in everyday functioning in children and youth with spinal muscular atrophy
Acronym
SMA
Study hypothesis
1. Strength of trunk muscles responsible for rotating trunk or pelvis in SMA children and youth is lower than in healthy peers
2. There is a correlation between the strength values of these muscles and limitations in everyday functioning
Ethics approval
The Senate Research Ethics Committee at Józef Piłsudski University of Physical Education in Warsaw, Poland, 07/02/2017, ref: SKE 01-03/2017
Study design
Single-centre case-control study
Primary study design
Observational
Secondary study design
Case-control study
Trial setting
Other
Trial type
Prevention
Patient information sheet
No participant information sheet available
Condition
Spinal muscular atrophy
Intervention
All participants will have a one-time measurement of muscle strength taken in a side-lying position using a handheld MICROFET measurement system. The device will be placed consecutively in three points on the front side of the body (i.e. forehead; trunk – in the shoulder joint area; trunk – in the anterior superior iliac spine area) and later in three points at the back of the body (i.e. posterior middle part of the skull; posterior part of the trunk in the shoulder joint area; pelvis – below iliac crest). 5-10 seconds will be devoted to one measurement during maximal isometric tension. Two series of measurements on each side will be performed in each of the aforementioned points. A higher value will be included in the analysis.
Participants will be also asked to complete a questionnaire including questions regarding limitations during everyday activities. Additional information will be collected on age, gender, accompanying illnesses.
Intervention type
Other
Phase
Drug names
Primary outcome measure
1. Strength of muscles responsible for trunk rotation is assessed using a handheld MICROFET, in which the value of strength expressed in pounds or Newtons in a pre-set time unit, on the study visit
2. Functioning limitations are measured using a questionnaire designed for the purpose of this study, including questions regarding limitations during everyday activities prepared on the basis of ICF on the study visit
Secondary outcome measures
No secondary outcome measures
Overall trial start date
01/12/2016
Overall trial end date
31/12/2017
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
Patients:
1. Spinal muscular atrophy (SMA) diagnosed through clinical and genetic examinations
2. Aged 5-18 years
Controls:
1. Healthy
2. Aged 5-18 years
3. Do not practice sport
Participant type
Mixed
Age group
Child
Gender
Both
Target number of participants
minimum 30
Participant exclusion criteria
Patients:
1. No confirmation of SMA in genetic tests
2. Children under 5
3. Children and youth not able to cooperate during an examination
Controls:
1. Age under 5 or over 18
2. Children not able to cooperate during an examination.
Recruitment start date
15/04/2017
Recruitment end date
30/09/2017
Locations
Countries of recruitment
Poland
Trial participating centre
Center of Functional Rehabilitation ORTHOS
Modzelewskiego 37
Warsaw
02-679
Poland
Funders
Funder type
University/education
Funder name
Józef Piłsudski University of Physical Education
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
The results will be published in an article in a neurologic, physical therapy or rehabilitation journal.
IPD Sharing plan:
The datasets generated during and/or analysed during the current study are/will be available upon request from Agnieszka Stępień (orthosas@wp.pl)
Intention to publish date
31/12/2018
Participant level data
Available on request
Basic results (scientific)
Publication list