Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
study id #: NCT04602195
condition: Spinal Muscular Atrophy
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.
last updated: February 04, 2022
start date: January 19, 2021
estimated completion: March 19, 2023
last updated: December 9, 2021
size / enrollment: 60
- 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
• Eligible Sexes: all
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: 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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