NCT04644393

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
40

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Nov 2020

Shorter than P25 for all trials

Geographic Reach
1 country

8 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2020

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

November 20, 2020

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 25, 2020

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2021

Completed
Last Updated

November 25, 2020

Status Verified

November 1, 2020

Enrollment Period

4 months

First QC Date

November 20, 2020

Last Update Submit

November 20, 2020

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular AtrophyMotor Function MeasureOutcome AssessmentNusinersen

Outcome Measures

Primary Outcomes (1)

  • 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

Eligibility Criteria

Age2 Years - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

* 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

You may qualify if:

  • 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

You may not qualify if:

  • \- 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence maladies neuromusculaires AOC CHU d'Angers

Angers, France

Location

Service de génétique médicale Pôle de Pédiatrie CHU Estaing

Clermont-Ferrand, France

Location

Service de Neurologie et réanimation pédiatriques CHU Paris IdF Ouest - Hôpital Raymond Poincaré

Garche, France

Location

Hospices Civils de Lyon, Hôpital Femme Mère Enfant

Lyon, France

Location

Apf Esean

Nantes, France

Location

Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires CHU Paris Est - Hôpital d'Enfants Armand-Trousseau

Paris, France

Location

Service de pédiatrie CHU de Saint-Etienne

Saint-Priest-en-Jarez, France

Location

Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse

Toulouse, France

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Central Study Contacts

Laure LE GOFF, MD

CONTACT

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

Pascal Ripeprt, RCA

CONTACT

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

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 20, 2020

First Posted

November 25, 2020

Study Start

November 1, 2020

Primary Completion

March 1, 2021

Study Completion

March 1, 2021

Last Updated

November 25, 2020

Record last verified: 2020-11

Locations

FR(8)