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van CCT (UK)


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Fatigability in SMA: validity and reproducibility of tests.


- candidate number17110
- NTR NumberNTR4558
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR1-apr-2014
- Secondary IDsNL48715.041.14 
- Public TitleFatigability in SMA: validity and reproducibility of tests.
- Scientific TitleFatigability in Spinal Muscular Atrophy: validity and reproducibility of tests.
- ACRONYM
- hypothesis1) The Motor Fatigability Test is a reproducible and valid outcome measure for fatigability of the skeletal muscles in patients with SMA
2) The Respiratory Fatigability Test is a reproducible and valid outcome measure for fatigability of the respiratory muscles in patients with SMA
3) Fatigability in SMA patients is associated with neuromuscular junction dysfunction
- Healt Condition(s) or Problem(s) studiedFatigue, Spinal Muscular Atrophy (SMA)
- Inclusion criteria1) Subjects with SMA
- Genetically confirmed diagnosis of SMA type 2 or type 3 or type 4
-Ability to follow test instructions
-Parental informed consent/Inform consent
-Age 8-50 years
2) Patient control group (matched on clinical status)
-Genetically confirmed diagnosis of (neuro-) muscular disease, no signs of neuromuscular dysfunction
-Ability to follow test instructions
-Parental informed consent/ Informed consent
-Age 8-50 years
3) Healthy control group (age and gender matched)
-Sufficient understanding of Dutch
-Ability to follow test instructions
-Parental informed consent/ Informed consent
-Age 8-50 years
- Exclusion criteriaAll subjects:
Concomitant medical problems that might intervene with the outcomes of the testing
- mec approval receivedno
- multicenter trialno
- randomisedno
- groupParallel
- Type2 or more arms, non-randomized
- Studytypeobservational
- planned startdate 1-jun-2014
- planned closingdate1-jun-2016
- Target number of participants150
- InterventionsPsychometric study on validity and reproducibility of fatigability tests
- Primary outcome1) Reproducibility of fatigability tests (reliability (ICC) , measurement error (ME)
2) Construct Validity
- content validity between fatigability tests,perceived fatigue and peripheral muscle fatigue: Pearson/Spearman Correlation Coefficient
- convergent validity between fatigability tests (endurance time and delta muscle strength values) and nerve conduction study (delta compound muscle action potential) : Pearson/Spearman Correlation Coefficient
- convergent validity between fatigability tests (endurance time and delta muscle strength values), perceived fatigue (Borg scores and fatigue questionnaires) and peripheral muscle fatigue (EMG registration; Root Mean Square amplitude muscles arms/legs/respiratory)
- discriminative validity between fatigability tests SMA patients, healthy control group and patient controle group: Analysis of Variance (ANOVA)
- Secondary outcomeFeasibility parameters
- measurement completion
- acceptability
- perceived burden
- TimepointsJune 2014 November 2015: selection of subjects and obtaining informed consent
July 2014 December 2015: testing subjects
December 2014 - December 2015: Analyses
January 2015 - June 2016: final data analysis and writing of manuscripts.
- Trial web site
- statusplanned
- CONTACT FOR PUBLIC QUERIESDrs. B. Bartels
- CONTACT for SCIENTIFIC QUERIESDrs. B. Bartels
- Sponsor/Initiator University Medical Center Utrecht (UMCU), Wilhelmina Children's Hospital (WKZ)
- Funding
(Source(s) of Monetary or Material Support)
Stichting Spieren voor Spieren
- Publications
- Brief summaryThe objective of this study is to develop reproducible and valid outcome measures for fatigability in patients with Spinal Muscular Atrophy. Recently, our research group demonstrated dysfunction of the neuromuscular junction in 50% of SMA-patients. The association between neuromuscular junction dysfunction and fatigability in SMA patients remains unclear. The psychometric properties of two newly developed fatigability tests will be investigated in SMA patients and healthy and patient control groups and the association between fatigability and neuromuscular function dysfunction in SMA patients will be studied The development of appropriate outcome measures for fatigability will give more insight in fatigability and the role of the neuromuscular junction in SMA patients and will contribute to the design of pharmaceutical and physical interventions to decrease fatigability and improve physical functioning.
- Main changes (audit trail)
- RECORD1-apr-2014 - 30-mei-2014


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