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

Completed

• Conditions: Spinal Muscular Atrophy

• Registration Number: NCT04602195
• Lead Sponsor: Hospices Civils de Lyon

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-10-20
• Study First Submitted QC: 2020-10-20
• Study First Posted: 2020-10-26
• Study Start: 2021-01-19
• Status Verified: 2023-04
• Primary Completion: 2023-11-10
• Study Completion: 2023-11-10
• Last Update Submitted: 2023-12-20
• Last Update Posted: 2023-12-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

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

Read More

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.

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (10)

Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence des Maladies Neuromusculaires AOC CHU d'Angers; 🇫🇷 Angers, France

Centre de Référence des maladies neuromusculaires Centre Hospitalier Régional Universitaire de Brest; 🇫🇷 Brest, France

Service de Neurologie et réanimation pédiatriques; 🇫🇷 Garches, France

L'Escale, Service central de rééducation pédiatrique Hospices Civils de Lyon Hôpital Mere Enfant; 🇫🇷 Bron, France

Service de génétique médicale Pôle de Pédiatrie CHU Estaing; 🇫🇷 Clermont-Ferrand, France

Apf Esean; 🇫🇷 Nantes, France

Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires; 🇫🇷 Paris, France

Service de Neuropédiatrie CHU de Strasbourg - Hôpital de Hautepierre; 🇫🇷 Strasbourg, France

Service de pédiatrie CHU de Saint-Etienne; 🇫🇷 Saint-Priest-en-Jarez, France

Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse; 🇫🇷 Toulouse, France