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

NCT ID: NCT04644393

Last Updated: 2020-11-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

40 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-11-30

Study Completion Date

2021-03-31

Brief Summary

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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

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Conditions

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Spinal Muscular Atrophy

Keywords

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Spinal Muscular Atrophy Motor Function Measure Outcome Assessment Nusinersen

Study Design

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Observational Model Type

OTHER

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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

Angers, , France

Site Status

Service de génétique médicale Pôle de Pédiatrie CHU Estaing

Clermont-Ferrand, , France

Site Status

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

Garche, , France

Site Status

Hospices Civils de Lyon, Hôpital Femme Mère Enfant

Lyon, , France

Site Status

Apf Esean

Nantes, , France

Site Status

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

Paris, , France

Site Status

Service de pédiatrie CHU de Saint-Etienne

Saint-Priest-en-Jarez, , France

Site Status

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

Toulouse, , France

Site Status

Countries

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France

Central Contacts

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Laure LE GOFF, MD

Role: CONTACT

Phone: +33 4 72 12 95 04

Email: [email protected]

Pascal Ripeprt, RCA

Role: CONTACT

Phone: +33 4 27 85 63 08

Email: [email protected]

Facility Contacts

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Julien DURIGNEUX, MD

Role: primary

Catherine SARRET, MD

Role: primary

Susana QUIJANO-ROY, MD

Role: primary

Laure LE GOFF, MD

Role: primary

Capucine de LATTRE, MD

Role: primary

Andreea SEFERIAN, MD

Role: primary

Gaelle Gousse, MD

Role: primary

Claude CANCES, MD

Role: primary

Other Identifiers

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69HCL20_1086

Identifier Type: -

Identifier Source: org_study_id