Responsiveness and Validation Study of MFM-20 in SMA Pati... | NCT04644393

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

NCT04644393

Sponsor

Hospices Civils de Lyon

Status

Unknown status

Last Update Posted

Nov 25, 2020Actual

Enrollment

40Estimated

Phase

Not provided

Conditions

Spinal Muscular Atrophy

Interventions

Not provided

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2020

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2020Estimated

Primary Completion Date

Mar 2021Estimated

Completion Date

Mar 2021Estimated

First Submitted Date

Nov 20, 2020

First Submission Date that Met QC Criteria

Nov 20, 2020

First Posted Date

Nov 25, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 20, 2020

Last Update Posted Date

Nov 25, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

40Estimated

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

6 Years

Standard Ages

Child

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Laure LE GOFF, MD	Contact	+33 4 72 12 95 04		laure.le-goff@chu-lyon.fr
Pascal Ripeprt, RCA	Contact	+33 4 27 85 63 08		pascal.rippert@chu-lyon.fr

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence maladies neuromusculaires AOC CHU d'Angers		Angers			France

Condition Browse Module

MeSH Terms

ID	Term
D009134	Muscular Atrophy, Spinal

Ancestor Terms

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease

Browse Leaves

Not provided

Browse Branches

Not provided