Clinical trial NCT02472990: Biomechanical and Morphological Changes in Dystrophic Muscle
Official title: Determining Biomechanical and Morphological Factors That Affect Children With Duchenne Muscular Dystrophy (DMD) Who Loss of the Ability to Walk
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Duchenne Dystrophy Muscular
Keywords: DMD
morphological predictive factors
Mechanical
Lead sponsor: University Hospital, Brest
Sponsor class: OTHER
First posted: 2015-06-16
Enrollment: 50
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD
timeFrame: 24 years
Secondary outcomes:
measure: Identify the muscles wasting and their implication in the loss of strength and the walking ability
timeFrame: 24 years
measure: Biomechanical evolutionary data collected during the last 2 years of walking
timeFrame: 24 years
measure: Establish the relationship between the parameters of walking and the scrawny body morphological anomalies
timeFrame: 24 years
Other outcomes:
Locations:
city: Brest
country: France
facility: CHRU
state: Brittanny
city: Lille
country: France
facility: CHRU
state: North
city: Angers
country: France
facility: CHRU
city: Le Mans
country: France
facility: CH Le Mans
city: Lyon
country: France
facility: CHU
city: Nantes
country: France
facility: CHU de Nantes
city: Poitiers
country: France
facility: CHU
city: Rennes
country: France
facility: CHU
city: Tours
country: France
facility: CHRU
city: Vannes
country: France
facility: CHBA
Study officials:
References: