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

study id #: NCT04602195

condition: Spinal Muscular Atrophy

status: Recruiting

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.

results: https://clinicaltrials.gov/ct2/show/results/NCT04602195

last updated: February 04, 2022

start date: January 19, 2021

estimated completion: March 19, 2023

last updated: December 9, 2021

size / enrollment: 60

primary outcomes:

• 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
  

inclusion criteria:

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.

sponsor: Hospices Civils de Lyon

contacts: Laure Le Goff, MD, 4 72 12 95 04, laure.le-goff@chu-lyon.fr

investigators: Laure Le Goff, MD,L'Escale, Service Central De Rééducation Pédiatrique

trial center locations: France

• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Juliette ROPARS, Dr, 0298223657, juliette.ropars@chu-brest.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Vincent LAUGEL, Pr, 03 88 12 77 43, vincent.laugel@chru-strasbourg.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Andreea SEFERIAN, MD, 1 44 73 65 37, a.seferian@institut-myologie.org
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Susana QUIJANO-ROY, Pr, 1 47 41 78 90, susana.quijano-y@aphp.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Capucine De LATTRE, MD, 2 40 34 76 74, c.delattre@esean.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Julien DURIGNEUX, MD, 2 41 35 44 45, judurigneux@chu-angers.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Le Goff Laure, MD, 4 72 12 95 04, laure.le-goff@chu-lyon.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Catherine SARRET, MD, 4 73 75 06 53, csarret@chu-clermontferrand.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Gaelle GOUSSE, MD, 4 77 82 87 29, gaelle.gousse@chu-st-etienne.fr
  
  
• Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
  Claude CANCES, MD, 5 34 55 85 77, cances.c@chu-toulouse.fr