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ID
Source
Health condition
SMA
Spinal Muscular Atrophy
Fatigability
Sponsors and support
Intervention
Outcome measures
Primary outcome
1) 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 outcome
Feasibility parameters
- measurement completion
- acceptability
- perceived burden
Background summary
The 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.
Study objective
1) 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
Study design
June 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.
Intervention
Psychometric study on validity and reproducibility of fatigability tests
Lundlaan 6, 3508 AB Utrecht
B. Bartels
Utrecht
The Netherlands
+31 (0)88 75 54030
b.bartels-4@umcutrecht.nl
Lundlaan 6, 3508 AB Utrecht
B. Bartels
Utrecht
The Netherlands
+31 (0)88 75 54030
b.bartels-4@umcutrecht.nl
Inclusion criteria
1) 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 criteria
All subjects:
Concomitant medical problems that might intervene with the outcomes of the testing
Design
Recruitment
Followed up by the following (possibly more current) registration
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL4436 |
| NTR-old | NTR4558 |
| CCMO | NL48715.041.14 |
| OMON | NL-OMON44457 |