Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age5-60
SponsorNantes University Hospital
About this trial
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in Duchenne and Becker Muscular Dystrophies.
The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with Duchenne and Becker Dystrophies with muscles of healthy age-matched individuals of the same age and monitor disease progression in those with muscular dystrophies over a 12-month year period.
The ultrafast ultrasound technology used in this study allows the simultaneous assessment of muscle structure, mechanics and physiology, including stiffness, anisotropy, viscosity, intramuscular fat, muscle volume, and microvascular perfusion. The amount of muscle alteration measured will be related to performance in daily activities, such as walking and muscle strength, in order to identify sensitive and objective markers of disease progression.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Inclusion Criteria for patients with Duchenne Muscular Dystrophy: Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with Duchenne Muscular Dystrophy based on absence of dystrophin expression.
Inclusion Criteria for patients with Becker Muscular Dystrophy: Ambulatory and non-ambulatory males (ages 5-60 at baseline testing) previously diagnosed with Becker Muscular Dystrophy based on genetically confirmed, reduced or dysfunctional dystrophin.
Inclusion Criteria for Aged-matched controls: Ambulatory males (ages 5-60 years) without disease or injury to the lower and/or upper extremities
Disqualifiers
Exclusion Criteria for Age-matched controls: Any condition affecting muscle metabolism/function, neuromuscular disease, or injury to the lower and/or upper extremities in the past 5 years
Trial population
Study Population This bicentric study will include 60 participants: 18 male patients with Duchenne Muscular Dystrophy (DMD), aged 5 to 30 years (ambulant and non-ambulant), 18 male patients with Becker Muscular Dystrophy (BMD), aged 5 to 60 years, and 24 healthy male subjects, aged 5 to 60 years, without neuromuscular disease or limb injury. All participants must have French social insurance coverage and provide informed consent (from parents or legal representatives for minors). Patients will be recruited mainly from the CHU de Nantes, the Institut de Myologie (Paris), and through the French Muscular Dystrophy Association (AFM-Téléthon). Healthy volunteers will be recruited from Nantes University staff, students, and their children. The inclusion of minors is necessary because Duchenne and Becker muscular dystrophies are X-linked genetic disorders diagnosed in childhood, and disease severity increases with age.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
60 Participants
are grouped into 3 trial groupsGroup A: Age-matched Controls
Group B: Boys/Men with Duchenne Muscular Dystrophy
Group C: Boys/Men with Becker Muscular Dystrophy
Trial outcomes
Primary outcomes
1
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of muscle stiffness (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
Baseline2
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of anisotropy (a.u.) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
Baseline3
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of viscosity (Np/mm) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
Baseline4
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of nonlinear elastic parameters (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
BaselineSecondary outcomes
1
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of muscle stiffness (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
12 months2
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of anisotropy (a.u.) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
12 months3
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of viscosity (Np/mm) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
12 months4
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of nonlinear elastic parameters (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Time frame
12 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Nantes University Hospital
Lead sponsor
AGENCE NATIONALE DE RECHERCHE
Collaborator
Source ClinicalTrials.gov