Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules (MFM-Play)

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.

Study Overview

• Status: Completed
• Conditions: Neuromuscular Diseases
• Intervention / Treatment: Other: Items completion with or without a MFM-Play

• Study Type: Observational
• Enrollment (Actual): 70

Contacts and Locations

Study Locations

• France
  • Brest, France, 29200
    • Centre de référence maladies neuromusculaires Hôpital Morvan - CHRU de Brest
  • Bron, France, 69500
    • Service de Rééducation Fonctionnelle Pédiatrique - L'Escale - Hôpital Femme Mère Enfant, Hospices Civils de Lyon
  • La Tronche, France, 38700
    • Service de Médecine Physique et de Réadaptation Pédiatrique Hôpital Couple-Enfant CHU de Grenoble
  • Lille, France, 59000
    • Centre de référence maladies neuromusculaires Hôpital Salengro CHU Lille
  • Lyon, France, 69004
    • Consultation pluridisciplinaire MNM adulte Hôpital de la Croix Rousse, Groupement Hospitalier Nord HCL
  • Nantes, France, 44200
    • ESEAN - APF France Handicap
  • Paris, France, 75012
    • Consultation de pédiatrie- maladie neuromusculaire Hôpital Armand Trousseau APHP
  • Saint-Priest-en-Jarez, France, 42270
    • Centre de référence MNM Adulte Saint Etienne - service neurologie CHU Hôpital Nord
  • Saint-Étienne, France, 42000
    • Service de Médecine Physique et de Réadaptation Pédiatrique Hôpital Bellevue CHU Saint Etienne
  • Toulouse, France, 31059
    • Centre de référence des maladies neuromusculaires enfants Hôpital des enfants CHU de Toulouse

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Patients with a follow-up in a French neuromuscular reference center

Description

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)

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
MFM-Play; Neuromuscular disease patients completed MFM using MFM-Play	Other: Items 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of item scoring between 2 MFM completion.	Level of agreement between MFM item scores reported by physiotherapists during 2 different MFM completion using the MFM-Play. Level of agreement between MFM item scores reported by a same physiotherapist during a MFM completion using the MFM-Play and a MFM completed conventionally.	3 months

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon

Study record dates

Study Major Dates

• Study Start (Actual): November 17, 2022
• Primary Completion (Actual): July 3, 2024
• Study Completion (Actual): July 3, 2024

Study Registration Dates

• First Submitted: January 26, 2022
• First Submitted That Met QC Criteria: January 26, 2022
• First Posted (Actual): February 7, 2022

Study Record Updates

• Last Update Posted (Estimated): November 4, 2024
• Last Update Submitted That Met QC Criteria: October 31, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: Patient Participation; Motor Function Assessment; Reliability study
• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: 69HCL21_1088

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No