Biomechanical and Morphological Changes in Dystrophic Muscle

Not Applicable

• Conditions: Duchenne Dystrophy Muscular
• Interventions: Other: No drug and no placebo were used in this study

• Registration Number: NCT02472990
• Lead Sponsor: University Hospital, Brest

Brief Summary

The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in reeducative, pharmacological or surgical therapeutic targets.

Detailed Description

The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in reeducative, pharmacological or surgical therapeutic targets. We suppose that there are muscular characteristics and predictive parameters of the loss of walk ability. The identification of these potential therapeutic targets would improve the surveillance and the clinical care but would also guide future clinical and fundamental trials too.

Study Timeline

• Study First Submitted: 2015-06-10
• Study First Submitted QC: 2015-06-15
• Study First Posted: 2015-06-16
• Study Start: 2015-07
• Status Verified: 2018-03
• Last Update Submitted: 2018-03-15
• Last Update Posted: 2018-03-16
• Primary Completion: 2020-01
• Study Completion: 2021-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Young man ou woman (5 to 17 years old) with Duchenne Muscular Dystrophy (confirmed by immunohistochimy on the muscular biopsy and/or mutation in the dystrophin confirmed by molecular biology)
• Time more than 7 secondes to test of 10 m and/or distance less than 330 m to walk test of 6 minutes. These values are recent markers to include children with a strong risk of loss of walking ability in 2 years.
• Parental inform sign consent and / or child inform consent

Exclusion Criteria

• Recent orthopaedic surgery of lower limbs (6 months)
• Other chronic disease associated, which have an impact on the walking
• Cognitive Deficiency or behavior disorders limiting the understanding of the study
• Children who can benefit ATU (translarna ® or other) during the study
• All MRI contradications : pacemaker or neurosensory stimulator or implantable defibrillator, neurosurgical valves, cochlear implant or ferromagnetic implants near nervous structures, brace, metallic prostheses, not cooperative or agitated patients, patient claustrophobic, pregnant woman.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Healthy children	No drug and no placebo were used in this study	No drug and no placebo were used in this study. For 2h30 (time) * Measurement of leg strength using a dynamometer * Measurement of Motor Function * Walk test 6 minutes * Walk test 10 meters * Walk analysis: 3D recording of walking * Muscle MRI
Duchenne muscular dystrophy children	No drug and no placebo were used in this study	No drug and no placebo were used in this study. For 2h30 (time) * Measurement of leg strength using a dynamometer * Measurement of Motor Function * Walk test 6 minutes * Walk test 10 meters * Walk analysis: 3D recording of walking * Muscle MRI

Primary Outcome Measures

Name	Time	Method
Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD	24 years	Determine the biomechanical factors (muscular atrophy, muscular strength, muscular shrinkage) and morphological (greasy infiltration, contractile portion, muscular geometry) predictive of the loss of the walk ability of the children with DMD

Secondary Outcome Measures

Name	Time	Method
Identify the muscles wasting and their implication in the loss of strength and the walking ability	24 years	Identify the muscles wasting and their implication in the loss of strength and walking. This will be made possible thanks to the predictive analysis and the evolutionary analysis of the MRI and strength before and after loss of walking ability
Biomechanical evolutionary data collected during the last 2 years of walking	24 years	Biomechanical evolutionary data collected during the last 2 years of walking by the repetition of the analysis of the walking until the loss of the walking ability.
Establish the relationship between the parameters of walking and the scrawny body morphological anomalies	24 years	Establish the relationship between the parameters of walking and the scrawny body morphological anomalies (muscular Atrophy, greasy infiltration, contractile muscular portion, three-dimensional morphological parameters) by doing correlations analysis

Trial Locations

Locations (5)

CHBA; 🇫🇷 Vannes, France

CHRU; 🇫🇷 Tours, France

CH Le Mans; 🇫🇷 Le Mans, France

CHU; 🇫🇷 Rennes, France

CHU de Nantes; 🇫🇷 Nantes, France