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NusiMFM: Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen

Sponsor
Hospices Civils de Lyon (Other)
Overall Status
Recruiting
CT.gov ID
NCT04602195
Collaborator
(none)
60
Enrollment
10
Locations
25.9
Anticipated Duration (Months)
6
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items originally validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items originally validated for patients between 2 and 6 years old (MFM-20).

In order to prove the possible use of MFM-32 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.

Condition or Disease Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    60 participants
    Observational Model:
    Other
    Time Perspective:
    Prospective
    Official Title:
    Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen: NusiMFM
    Actual Study Start Date :
    Jan 19, 2021
    Anticipated Primary Completion Date :
    Mar 19, 2023
    Anticipated Study Completion Date :
    Mar 19, 2023

    Outcome Measures

    Primary Outcome Measures

    1. MFM-32 responsiveness [1 Year after the first MFM-32 assessment]

      Responsiveness of MFM-32 in comparison to PGIC. The responsiveness sensitivity to change indexes considered will be the effect size (ES) amplitude and the Standardized Response Mean (SRM)

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 6 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed

    • Aged 2 to 6 years old

    • Treated by Nusinersen for at least two months

    • With parental assent

    Exclusion Criteria:

    • Patients with associated cognitive impairment making impossible evaluation of motor function

    • Patients participating to a clinical study with a potential effect on their motor function.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence des Maladies Neuromusculaires AOC CHU d'Angers Angers France 49933
    2 Centre de Référence des maladies neuromusculaires Centre Hospitalier Régional Universitaire de Brest Brest France 29200
    3 L'Escale, Service central de rééducation pédiatrique Hospices Civils de Lyon Hôpital Mere Enfant Bron France 69677
    4 Service de génétique médicale Pôle de Pédiatrie CHU Estaing Clermont-Ferrand France 63003
    5 Service de Neurologie et réanimation pédiatriques Garches France 92380
    6 Apf Esean Nantes France 44200
    7 Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires Paris France 75571
    8 Service de pédiatrie CHU de Saint-Etienne Saint-Priest-en-Jarez France 42270
    9 Service de Neuropédiatrie CHU de Strasbourg - Hôpital de Hautepierre Strasbourg France 67200
    10 Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse Toulouse France 31059

    Sponsors and Collaborators

    • Hospices Civils de Lyon

    Investigators

    • Principal Investigator: Laure Le Goff, MD, L'Escale, Service central de rééducation pédiatrique

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Hospices Civils de Lyon
    ClinicalTrials.gov Identifier:
    NCT04602195
    Other Study ID Numbers:
    • 69HCL19_0875
    First Posted:
    Oct 26, 2020
    Last Update Posted:
    Dec 9, 2021
    Last Verified:
    Feb 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Hospices Civils de Lyon
    Spinal Muscular Atrophy, Motor Function Measure,Nusinersen
    Additional relevant MeSH terms:
    Muscular Atrophy
    Muscular Atrophy, Spinal
    Atrophy

    Study Results

    No Results Posted as of Dec 9, 2021