NCT04602195

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
33

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jan 2021

Typical duration for all trials

Geographic Reach
1 country

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 20, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

October 26, 2020

Completed
3 months until next milestone

Study Start

First participant enrolled

January 19, 2021

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 10, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 10, 2023

Completed
Last Updated

December 21, 2023

Status Verified

April 1, 2023

Enrollment Period

2.8 years

First QC Date

October 20, 2020

Last Update Submit

December 20, 2023

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular Atrophy, Motor Function Measure,Nusinersen

Outcome Measures

Primary Outcomes (1)

  • MFM-32 responsiveness

    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)

    1 Year after the first MFM-32 assessment

Eligibility Criteria

Age2 Years - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

* 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

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence des Maladies Neuromusculaires AOC CHU d'Angers

Angers, 49933, France

Location

Centre de Référence des maladies neuromusculaires Centre Hospitalier Régional Universitaire de Brest

Brest, 29200, France

Location

L'Escale, Service central de rééducation pédiatrique Hospices Civils de Lyon Hôpital Mere Enfant

Bron, 69677, France

Location

Service de génétique médicale Pôle de Pédiatrie CHU Estaing

Clermont-Ferrand, 63003, France

Location

Service de Neurologie et réanimation pédiatriques

Garches, 92380, France

Location

Apf Esean

Nantes, 44200, France

Location

Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires

Paris, 75571, France

Location

Service de pédiatrie CHU de Saint-Etienne

Saint-Priest-en-Jarez, 42270, France

Location

Service de Neuropédiatrie CHU de Strasbourg - Hôpital de Hautepierre

Strasbourg, 67200, France

Location

Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse

Toulouse, 31059, France

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Laure Le Goff, MD

    L'Escale, Service central de rééducation pédiatrique

    PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

First Submitted

October 20, 2020

First Posted

October 26, 2020

Study Start

January 19, 2021

Primary Completion

November 10, 2023

Study Completion

November 10, 2023

Last Updated

December 21, 2023

Record last verified: 2023-04

Locations

FR(10)