Duchenne Dystrophy MuscularMARCHE-DMDNCT02472990
Biomechanical and Morphological Changes in Dystrophic Muscle
A study of Duchenne Dystrophy Muscular, run by University Hospital, Brest at 10 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Duchenne Dystrophy Muscular
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 10 sitesFrance
- Study dates
- Jul 2015to Jan 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
France10 sites, 10 recruiting
- Not yet recruiting
CHRU
Angers, 49933
Julien DURIGNEUX, Dr
Julien DURIGNEUX, Dr
- Recruiting
CHRU
Brest, Brittanny, 29609
Sylvain Brochard, Dr, 02 98 22 31 52, sylvain.brochard@chu-brest.fr
Sylvain Brochard, Dr
- Not yet recruiting
CH Le Mans
Le Mans, 72650
Denis Colin, Dr, 02 43 51 72 67
Denis Colin, Dr
- Not yet recruiting
CHRU
Lille, North, 59037
Jean-Marie CUISSET, Dr
Jean-Marie CUISSET
- Recruiting
CHU
Lyon, 63677
Carole Vuillerot, Dr, 07 89 22 02 92, carole.vuillerot@gmail.com
Carole Vuillerot, Dr
- Recruiting
CHU de Nantes
Nantes, 44093
Yann PEREON, 02 40 08 36 17
Yann PEREON
- Not yet recruiting
CHU
Poitiers, 86021
Karoline LODE-KOLZ, Dr, 05 49 44 44 44
Karoline LODE-KOLZ, Dr
- Not yet recruiting
CHU
Rennes, 35033
Hélène Rauscent, Dr
Hélène Rauscent, Dr
- Not yet recruiting
CHRU
Tours, 37044
Emmanuelle Lagrue, Dr
Emmanuelle Lagrue, Dr
- Not yet recruiting
CHBA
Vannes, 56017
Hubert Journel, Dr
Hubert Journel, Dr
About this study
Official title: 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.
Read the 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.
- Conditions
- Duchenne Dystrophy Muscular
- Being tested
- No drug and no placebo were used in this study
- Keywords
- DMD, morphological predictive factors, Mechanical
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No drug and no placebo were used in this studyOther
Also called Several parameters must be performed.
measures of muscular strength of legs, motor function, walk testing and analysis (3D video recording), RMI
| Study group | Role | What they receive |
|---|---|---|
| Duchenne muscular dystrophy children | Other | 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 Receives: Other: No drug and no placebo were used in this study |
| Healthy children | Other | 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 Receives: Other: No drug and no placebo were used in this study |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 24 years
Secondary outcomes (3)
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
Measured: 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.
Measured: 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
Measured: 24 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2015 (estimated)
- Study startJul 2015
- Primary completionJan 2020 (estimated)
- Study completionJan 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Dystrophy MuscularStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Brest
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02472990
- Sponsor's protocol ID
- MARCHE-DMD
- Acronym
- MARCHE-DMD
- Status last verified
- Mar 2018
- First submitted
- 10 Jun 2015
- Last updated
- 16 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determining Biomechanical and Morphological Factors That Affect Children With Duchenne Muscular Dystrophy (DMD) Who Loss of the Ability to Walk
- ClinicalTrials.gov ID
- NCT02472990
- Acronym / sponsor ID
- MARCHE-DMD / MARCHE-DMD
- Conditions
- Duchenne Dystrophy Muscular
- Keywords
- DMD, morphological predictive factors, Mechanical
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- University Hospital, BrestUniversity, hospital or other
- Study start
- Jul 2015
- Primary completion
- Jan 2020 (estimated)
- Study completion
- Jan 2021 (estimated)
- First posted
- 16 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Duchenne muscular dystrophy children | Other | Other: No drug and no placebo were used in this studyNo 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 |
| Healthy children | Other | Other: No drug and no placebo were used in this studyNo 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 |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No drug and no placebo were used in this studyAlso: Several parameters must be performed.measures of muscular strength of legs, motor function, walk testing and analysis (3D video recording), RMI | Other | Duchenne muscular dystrophy children, Healthy children |
Primary outcome
Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD
Time frame: 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
3 secondary outcomes
Identify the muscles wasting and their implication in the loss of strength and the walking ability
Time frame: 24 years
Biomechanical evolutionary data collected during the last 2 years of walking
Time frame: 24 years
Establish the relationship between the parameters of walking and the scrawny body morphological anomalies
Time frame: 24 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study of Duchenne Dystrophy Muscular, run by University Hospital, Brest at 10 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sylvain BROCHARD, Dr (02 98 22 31 52, sylvain.brochard@chu-brest.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD (time frame: 24 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2018.