Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen

• Conditions: Spinal Muscular Atrophy

• Registration Number: NCT04644393
• 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 validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items validated for patients between 2 and 6 years old (MFM-20).

In order to show the possible use of MFM-20 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-20 and the validity of the scale in this population.

Detailed Description

Not available

Study Timeline

• Status Verified: 2020-11
• Study Start: 2020-11
• Study First Submitted: 2020-11-20
• Study First Submitted QC: 2020-11-20
• Last Update Submitted: 2020-11-20
• Study First Posted: 2020-11-25
• Last Update Posted: 2020-11-25
• Primary Completion: 2021-03
• Study Completion: 2021-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

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 at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months
• With parental assent

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
MFM-20 responsiveness	At least 6 Months after the first MFM-20 assessment, and at maximum 24 months	The MFM-20 sensitivity to change indexes considered will be the effect amplitude and the Standardized Response Mean (SRM).

Trial Locations

Locations (8)

Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence maladies neuromusculaires AOC CHU d'Angers; 🇫🇷 Angers, France

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

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

Service de Neurologie et réanimation pédiatriques CHU Paris IdF Ouest - Hôpital Raymond Poincaré; 🇫🇷 Garche, France

Apf Esean; 🇫🇷 Nantes, France

Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires CHU Paris Est - Hôpital d'Enfants Armand-Trousseau; 🇫🇷 Paris, France

Hospices Civils de Lyon, Hôpital Femme Mère Enfant; 🇫🇷 Lyon, France

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