Duchene Muscular DystrophyINNOVANNCT07378553
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
An observational study of Duchene Muscular Dystrophy and Becker Muscular Dystrophy, run by Nantes University Hospital at 1 site in France.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 60 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchene Muscular Dystrophy, Becker Muscular Dystrophy
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 3 Mar 2026to 3 Mar 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 60 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study 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.
Who may be able to join
- 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
Who cannot join
- Exclusion Criteria for patients with Duchenne and Becker Muscular Dystrophies:
- Inability to undergo static exam, missing measurement site (resection/amputation), neurocognitive impairment preventing informed consent
- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Multiparametric Ultrafast Ultrasound Evaluation of Skeletal Muscle in Duchenne and Becker Muscular Dystrophies
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.
Read the detailed description
Duchenne and Becker muscular dystrophies (DMD/BMD) are the most common forms of muscular dystrophies, progressive muscle wasting diseases leading to weakness and devastating functional impairment. DMD is characterized by predominant involvement in skeletal, including respiratory, and cardiac muscles. Diagnosis is usually established at three to four years of age. Patients typically show muscle degeneration that worsens with age, leading to wheelchair dependency usually by the age of ten, assisted ventilation before the age of twenty, and premature death in the second to fourth decade. BMD shows a similar clinical phenotype but a later onset (approximately twelve years of age), a slower disease progression, delayed or no loss of ambulation, and variable involvement of the cardiac and respiratory systems. Considerable heterogeneity has been observed within and across the DMD/BMD phenotypes, which makes the design and analysis of clinical trials and prediction of the disease progression challenging.
Recent inconsistent results in DMD clinical development programs have also raised questions about the validity of the methods used to evaluate treatment efficacy. The number of potentially effective therapeutic approaches for DMD/BMD has rapidly increased over recent years, and thus the demand for validated outcome measures to demonstrate clinically meaningful therapeutic response over time in clinical trials (e.g., one year) is higher than ever. Regulatory agencies (e.g., FDA) have explicitly been encouraging the development of imaging biomarkers that may serve as surrogate markers of how patients will respond to investigational treatments in DMD/BMD.
This study aims to validate the potential of innovative ultrasound imaging methods, developed by our research consortium over recent years, as imaging tools to monitor disease progression and serve as a surrogate outcome measure for clinical trials in muscular dystrophies. Using cutting-edge ultrasound imaging techniques, including shear wave elastography, matrix approach, 3D freehand ultrasound, and power Doppler we will quantify much needed surrogate measures of muscle damage and progressive degeneration such as intramuscular fat content, fibrosis, muscle apoptosis and structural disorganization, inflammation, hypertrophy/atrophy, and intramuscular blood flow, in the lower and upper extremity muscles. An easy-to-apply multiparametric approach will be developed to assess the most important hallmarks of muscle degeneration associated with the most common and devastating muscular dystrophies.
- Keywords
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Ultrasound Imaging, Ultrafast Ultrasound, Muscle
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Age-matched Controls | Age matched non-affected men, matched for men with Becker and Duchenne Muscular Dystrophies | |
| Boys/Men with Duchenne Muscular Dystrophy | This group will include ambulatory and non-ambulatory boys/men with Duchenne Muscular Dystrophy ranging from 5-30 years old. | |
| Boys/Men with Becker Muscular Dystrophy | This group will include ambulatory and non-ambulatory men with Becker Muscular Dystrophy ranging from 5-60 years old. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline
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
Measured: Baseline
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
Measured: Baseline
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
Measured: Baseline
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of intramuscular fat (m/s) in the lower extremity and/or upper extremity muscles across three participant groups
Measured: Baseline
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Assessment of intramuscular blood flow (%) in the lower extremity and/or upper extremity muscles across three participant groups
Measured: Baseline
Secondary outcomes (10)
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
Measured: 12 months
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
Measured: 12 months
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
Measured: 12 months
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
Measured: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of intramuscular fat (m/s) in the lower extremity and/or upper extremity muscles across three participant groups
Measured: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Assessment of intramuscular blood flow (%) in the lower extremity and/or upper extremity muscles across three participant groups
Measured: 12 months
Muscle function
Muscle strength will be evaluated by dynamometry
Measured: Baseline and changes from baseline in at 12 months
Functional outcomes
10-Meter Walk Test in DMD and BMD participants
Measured: Baseline and changes from baseline at 12 months
Functional outcomes
NorthStar Ambulatory Assessment performance in DMD and BMD participants
Measured: Baseline and changes from baseline at 12 months
Correlations between ultrasound measures, muscle function and functional outcomes
In both BMD and DMD, the correlation between each ultrasound imaging parameter [muscle stiffness (kPa), anisotropy (a.u.), viscosity (Np/mm), nonlinear elastic parameters (kPa), intramuscular fat (m/s), muscle volume (ml), and intramuscular blood flow (%)] and all functional endpoints [NorthStar Ambulatory Assessment, 10-Meter Walk Test] will be determined, as well as the ability of ultrasound measures to predict future change and loss in function.
Measured: Through study completion, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2026
- Study start3 Mar 2026
- Primary completion3 Mar 2029 (estimated)
- Study completion3 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchene Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Nantes University HospitalThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nantes University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- AGENCE NATIONALE DE RECHERCHE
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07378553
- Sponsor's protocol ID
- RC25_0273
- Other IDs
- 2025-A01398-41
- Acronym
- INNOVAN
- Status last verified
- Jun 2026
- First submitted
- 21 Nov 2025
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multiparametric Ultrafast Ultrasound Evaluation of Skeletal Muscle in Duchenne and Becker Muscular Dystrophies
- ClinicalTrials.gov ID
- NCT07378553
- Acronym / sponsor ID
- INNOVAN / RC25_0273
- Keywords
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Ultrasound Imaging, Ultrafast Ultrasound, Muscle
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Nantes University HospitalUniversity, hospital or other
- Collaborators
- AGENCE NATIONALE DE RECHERCHE
- Study start
- 3 Mar 2026 (actual)
- Primary completion
- 3 Mar 2029 (estimated)
- Study completion
- 3 Mar 2029 (estimated)
- First posted
- 30 Jan 2026 (actual)
- 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 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.
Detailed description
Duchenne and Becker muscular dystrophies (DMD/BMD) are the most common forms of muscular dystrophies, progressive muscle wasting diseases leading to weakness and devastating functional impairment. DMD is characterized by predominant involvement in skeletal, including respiratory, and cardiac muscles. Diagnosis is usually established at three to four years of age. Patients typically show muscle degeneration that worsens with age, leading to wheelchair dependency usually by the age of ten, assisted ventilation before the age of twenty, and premature death in the second to fourth decade. BMD shows a similar clinical phenotype but a later onset (approximately twelve years of age), a slower disease progression, delayed or no loss of ambulation, and variable involvement of the cardiac and respiratory systems. Considerable heterogeneity has been observed within and across the DMD/BMD phenotypes, which makes the design and analysis of clinical trials and prediction of the disease progression challenging.
Recent inconsistent results in DMD clinical development programs have also raised questions about the validity of the methods used to evaluate treatment efficacy. The number of potentially effective therapeutic approaches for DMD/BMD has rapidly increased over recent years, and thus the demand for validated outcome measures to demonstrate clinically meaningful therapeutic response over time in clinical trials (e.g., one year) is higher than ever. Regulatory agencies (e.g., FDA) have explicitly been encouraging the development of imaging biomarkers that may serve as surrogate markers of how patients will respond to investigational treatments in DMD/BMD.
This study aims to validate the potential of innovative ultrasound imaging methods, developed by our research consortium over recent years, as imaging tools to monitor disease progression and serve as a surrogate outcome measure for clinical trials in muscular dystrophies. Using cutting-edge ultrasound imaging techniques, including shear wave elastography, matrix approach, 3D freehand ultrasound, and power Doppler we will quantify much needed surrogate measures of muscle damage and progressive degeneration such as intramuscular fat content, fibrosis, muscle apoptosis and structural disorganization, inflammation, hypertrophy/atrophy, and intramuscular blood flow, in the lower and upper extremity muscles. An easy-to-apply multiparametric approach will be developed to assess the most important hallmarks of muscle degeneration associated with the most common and devastating muscular dystrophies.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Age-matched Controls | – | –Age matched non-affected men, matched for men with Becker and Duchenne Muscular Dystrophies |
| Boys/Men with Duchenne Muscular Dystrophy | – | –This group will include ambulatory and non-ambulatory boys/men with Duchenne Muscular Dystrophy ranging from 5-30 years old. |
| Boys/Men with Becker Muscular Dystrophy | – | –This group will include ambulatory and non-ambulatory men with Becker Muscular Dystrophy ranging from 5-60 years old. |
Who may qualify
- Ages
- 5 to 60 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- Exclusion Criteria for patients with Duchenne and Becker Muscular Dystrophies:
- Inability to undergo static exam, missing measurement site (resection/amputation), neurocognitive impairment preventing informed consent
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of muscle stiffness (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of anisotropy (a.u.) in the lower extremity and/or upper extremity muscles across three participant groups
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of viscosity (Np/mm) in the lower extremity and/or upper extremity muscles across three participant groups
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of nonlinear elastic parameters (kPa) in the lower extremity and/or upper extremity muscles across three participant groups
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of intramuscular fat (m/s) in the lower extremity and/or upper extremity muscles across three participant groups
Baseline mechanical, structural, and physiological muscle ultrasound parameters
Time frame: Baseline
Assessment of intramuscular blood flow (%) in the lower extremity and/or upper extremity muscles across three participant groups
10 secondary outcomes
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Change from baseline in mechanical, structural, and physiological muscle ultrasound parameters
Time frame: 12 months
Muscle function
Time frame: Baseline and changes from baseline in at 12 months
Functional outcomes
Time frame: Baseline and changes from baseline at 12 months
Functional outcomes
Time frame: Baseline and changes from baseline at 12 months
Correlations between ultrasound measures, muscle function and functional outcomes
Time frame: Through study completion, 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchene Muscular Dystrophy and Becker Muscular Dystrophy, run by Nantes University Hospital at 1 site 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 3 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: looking for participants now, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yann PEREON (0240083704, yann.pereon@chu-nantes.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Baseline mechanical, structural, and physiological muscle ultrasound parameters (time frame: Baseline). The study lists 6 primary outcomes in all. 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 24 Jun 2026.