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

Spinal Muscular Atrophy Clinical Trial

— RetroNusiMFM  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04644393
• Other study ID #: 69HCL20_1086
• Status: Not yet recruiting
• Start date: November 2020
• Est. completion date: March 2021

Study information

• Verified date: November 2020
• Source: Hospices Civils de Lyon
• Contact: Laure LE GOFF, MD
• Phone: +33 4 72 12 95 04
• Email: laure.le-goff[@]chu-lyon.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: March 2021
• Est. primary completion date: March 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 6 Years

Eligibility

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

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Locations

Country	Name	City	State
France	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 Neurologie et réanimation pédiatriques CHU Paris IdF Ouest - Hôpital Raymond Poincaré	Garche
France	Hospices Civils de Lyon, Hôpital Femme Mère Enfant	Lyon
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	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

Sponsors (1)

Lead Sponsor	Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

Outcome

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