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NCT Number: NCT04602195

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

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.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

2 year–6 year

Sex eligibility

All sexes

Study type

Observational

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. MFM-32 responsiveness

    Time frame: 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)

Sponsors and collaborators

Lead sponsor

Hospices Civils de Lyon

Other

Registry information

Official study title

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

Acronym: NusiMFM

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Oct 26, 2020
Registry last updated
Dec 21, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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