Neuromuscular Diseases
Conditions
Keywords
Motor Function Assessment, Reliability study, Patient Participation
Brief summary
Neuromuscular diseases include more than 200 rare disorders affecting muscles or the nervous system. Functional scales assessing motor function are tools allowing to measure the evolution of motor impairment of patients with a neuromuscular disease. They are frequently used as main outcome measures in clinical trials which are currently in full development thanks to advances in genetic and drug researches. Among the available scales, the Motor Function Measure (MFM), a scale consisting of 32 items, had shown good metrological properties in terms of validity, reliability and sensitivity to change, regardless of the diagnosis and extent of motor impairment. By exploring the potential of digital technologies applied to MFM, investigators created a completion module composed of animations with different playful and informative scenarios displayed on a digital tablet. The main purpose of this project is to conduct a multicentre study to assess reliabilities of the MFM completed using the MFM-Play. Expected benefits of this project are to improve the experience and the participation of the children to who is often imposed multiple assessments during the numerous follow-up visits of clinical trials, while maintaining good metrological properties of the MFM.
Interventions
OTHERItems completion with or without a MFM-Play
Patients will completed 3 MFM during the follow-up, two times using the MFM-Play, and one without the MFM-Play Delay between each MFM completion will be 1 month ± 15 days
Sponsors
Hospices Civils de Lyon
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 60 Years
Inclusion criteria
* Patients with a neuromuscular disease confirmed by the clinic, electroneuromyography (ENMG), biology, muscular biopsy, or genetic * aged ≥ 2 yo and ≤ 60 yo * having been informed and not objecting to participating in the study (information and non-opposition of parents/holders of parental authority for minor patients)
Exclusion criteria
* participating in other interventional research with an exclusion period that is still ongoing or that may interfere with the results of this study * having had surgery or illness impacting their motor function within the last 6 months * having understanding and/or behavioral deficiencies preventing rigorous assessment * Patient placed under legal protection (this includes guardianship, curatorship and safeguard of justice)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Intraclass correlation coefficient (ICC) for MFM total score and D1-D3 subscores | 3 months | ICC for MFM total score and D1-D3 subscores between the two MFM Play assessments performed by different therapists ICCs for MFM total score and D1-D3 subscores between MFM Play and conventional MFM |
Countries
France
Outcome results
None listed