Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules

NCT05227274 | Completed

• 1 other identifier: 69HCL21_1088
• Study Type: observational
• Target: 70
• Locations: 1 country
• Sites: 10

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.

Conditions

Neuromuscular Diseases

Keywords

Motor Function Assessment; Reliability study; Patient Participation

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

MFM-Play

Neuromuscular disease patients completed MFM using MFM-Play

Other: Items completion with or without a MFM-Play

Interventions

Items completion with or without a MFM-Play OTHER

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

MFM-Play

Eligibility Criteria

• Age: 2 Years - 60 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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)

You may not qualify if:

• 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)

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (10)

Centre de référence maladies neuromusculaires Hôpital Morvan - CHRU de Brest

Brest, 29200, France

Location

Service de Rééducation Fonctionnelle Pédiatrique - L'Escale - Hôpital Femme Mère Enfant, Hospices Civils de Lyon

Bron, 69500, France

Location

Service de Médecine Physique et de Réadaptation Pédiatrique Hôpital Couple-Enfant CHU de Grenoble

La Tronche, 38700, France

Location

Centre de référence maladies neuromusculaires Hôpital Salengro CHU Lille

Lille, 59000, France

Location

Consultation pluridisciplinaire MNM adulte Hôpital de la Croix Rousse, Groupement Hospitalier Nord HCL

Lyon, 69004, France

Location

ESEAN - APF France Handicap

Nantes, 44200, France

Location

Consultation de pédiatrie- maladie neuromusculaire Hôpital Armand Trousseau APHP

Paris, 75012, France

Location

Service de Médecine Physique et de Réadaptation Pédiatrique Hôpital Bellevue CHU Saint Etienne

Saint-Etienne, 42000, France

Location

Centre de référence MNM Adulte Saint Etienne - service neurologie CHU Hôpital Nord

Saint-Priest-en-Jarez, 42270, France

Location

Centre de référence des maladies neuromusculaires enfants Hôpital des enfants CHU de Toulouse

Toulouse, 31059, France

Location

MeSH Terms

Conditions

Neuromuscular Diseases; Patient Participation

Condition Hierarchy (Ancestors)

Nervous System Diseases; Patient Acceptance of Health Care; Treatment Adherence and Compliance; Health Behavior; Behavior

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2022
• First Posted: February 7, 2022
• Study Start: November 17, 2022
• Primary Completion: July 3, 2024
• Study Completion: July 3, 2024
• Last Updated: November 4, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (10)