Biomechanical and Morphological Changes in Dystrophic Muscle (MARCHE-DMD)

Determining Biomechanical and Morphological Factors That Affect Children With Duchenne Muscular Dystrophy (DMD) Who Loss of the Ability to Walk

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.

Study Overview

• Status: Unknown
• Conditions: Duchenne Dystrophy Muscular
• Intervention / Treatment: Other: No drug and no placebo were used in this study

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 Type: Interventional
• Enrollment (Anticipated): 50
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Angers, France, 49933
    • Not yet recruiting
    • CHRU
    • Contact: Julien DURIGNEUX, Dr
    • Principal Investigator: Julien DURIGNEUX, Dr
  • Le Mans, France, 72650
    • Not yet recruiting
    • CH Le mans
    • Contact: Denis Colin, Dr; Phone Number: 02 43 51 72 67
    • Principal Investigator: Denis Colin, Dr
  • Lyon, France, 63677
    • Recruiting
    • CHU
    • Contact: Carole Vuillerot, Dr; Phone Number: 07 89 22 02 92; Email: carole.vuillerot@gmail.com
    • Principal Investigator: Carole Vuillerot, Dr
    • Sub-Investigator: Stéphanie Fontaine, Dr
  • Nantes, France, 44093
    • Recruiting
    • CHU de Nantes
    • Contact: Yann PEREON; Phone Number: 02 40 08 36 17
    • Principal Investigator: Yann Pereon
    • Sub-Investigator: Raphaël GROSS
  • Poitiers, France, 86021
    • Not yet recruiting
    • CHU
    • Contact: Karoline LODE-KOLZ, Dr; Phone Number: 05 49 44 44 44
    • Principal Investigator: Karoline LODE-KOLZ, Dr
  • Rennes, France, 35033
    • Not yet recruiting
    • CHU
    • Contact: Hélène Rauscent, Dr
    • Principal Investigator: Hélène Rauscent, Dr
  • Tours, France, 37044
    • Not yet recruiting
    • CHRU
    • Contact: Emmanuelle Lagrue, Dr
    • Principal Investigator: Emmanuelle Lagrue, Dr
  • Vannes, France, 56017
    • Not yet recruiting
    • CHBA
    • Contact: Hubert Journel, Dr
    • Principal Investigator: Hubert Journel, Dr
  • Brittanny
    • Brest, Brittanny, France, 29609
      • Recruiting
      • CHRU
      • Contact: Sylvain Brochard, Dr; Phone Number: 02 98 22 31 52; Email: sylvain.brochard@chu-brest.fr
      • Principal Investigator: Sylvain Brochard, Dr
      • Sub-Investigator: Sylviane Peudenier, Dr
      • Principal Investigator: Juliette ROPARS, Dr
  • North
    • Lille, North, France, 59037
      • Not yet recruiting
      • CHRU
      • Contact: Jean-Marie CUISSET, Dr
      • Principal Investigator: Jean-Marie CUISSET

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 13 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Duchenne muscular dystrophy children; 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	Other: No drug and no placebo were used in this study; measures of muscular strength of legs, motor function, walk testing and analysis (3D video recording), RMI; Other Names: Several parameters must be performed.
Other: Healthy children; 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	Other: No drug and no placebo were used in this study; measures of muscular strength of legs, motor function, walk testing and analysis (3D video recording), RMI; Other Names: Several parameters must be performed.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD	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	24 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Identify the muscles wasting and their implication in the loss of strength and the walking ability	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	24 years
Biomechanical evolutionary data collected during the last 2 years of walking	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.	24 years
Establish the relationship between the parameters of walking and the scrawny body morphological anomalies	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	24 years

Collaborators and Investigators

• Sponsor: University Hospital, Brest

Study record dates

Study Major Dates

• Study Start: July 1, 2015
• Primary Completion (Anticipated): January 1, 2020
• Study Completion (Anticipated): January 1, 2021

Study Registration Dates

• First Submitted: June 10, 2015
• First Submitted That Met QC Criteria: June 15, 2015
• First Posted (Estimate): June 16, 2015

Study Record Updates

• Last Update Posted (Actual): March 16, 2018
• Last Update Submitted That Met QC Criteria: March 15, 2018
• Last Verified: March 1, 2018

More Information

Terms related to this study

• Keywords: Mechanical; DMD; morphological predictive factors
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: MARCHE-DMD