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

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

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04644393

Acronym

RetroNusiMFM

Enrollment

Registered

2020-11-25

Start date

2020-11-30

Completion date

2021-03-31

Last updated

2020-11-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular Atrophy, Motor Function Measure, Outcome Assessment, Nusinersen

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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to 6 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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).

Countries

France

Contacts

Primary ContactLaure LE GOFF, MD

laure.le-goff@chu-lyon.fr+33 4 72 12 95 04

Backup ContactPascal Ripeprt, RCA

pascal.rippert@chu-lyon.fr+33 4 27 85 63 08

Outcome results