Condition category
Musculoskeletal Diseases
Date applied
04/04/2017
Date assigned
11/04/2017
Last edited
11/04/2017
Prospective/Retrospective
Prospectively registered
Overall trial status
Ongoing
Recruitment status
No longer recruiting

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 measures

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

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

Sponsor information

Organisation

Józef Piłsudski University of Physical Education

Sponsor details

Marymoncka 34
Warsaw
00-968
Poland

Sponsor type

University/education

Website

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes