Becker Muscular DystrophyMyoBrain-BMDNCT07740057
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
An observational study of Becker Muscular Dystrophy, run by Institut de Myologie, France at 1 site in France.
Trial summary
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition. However, in the event of future treatment, it is essential for clinical and research teams to have as much information as possible about the disease, particularly… Read more about this study
- Who is it for?
- Ages 18 to 50 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Becker Muscular Dystrophy
- How many people
- 75 participants
- Where
- 1 siteFrance
- Study dates
- 15 Dec 2026to 31 Dec 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 18 to 50 years
- Men only
- Healthy volunteers accepted
- Adult
Study population: Fifty patients will be included, including 25 patients having the cerebral Dp140 isoform (Dp140+) and 25 patients lacking the cerebral Dp140 isoform (Dp140-). A same number (n=25) of age-matched healthy volunteers will be included as well.
Who may be able to join
- Genetically confirmed diagnosis of Becker muscular dystrophy
- Ambulant
- Signed ICF
Who cannot join
- Individuals presenting contraindications to MRI
- History of allergy to contrast product
- Individuals with history of neurological diseases interfering with the study
- Individuals with concomitant medical conditions that could interfere with the study outcomes
- subjects who suffer from any type of cancer (or on cancer treatment)
- obese subjects: BMI>35
Show all 10Show fewer
- subjects with cardiac (left-ventricular ejection fraction <40%) and/or respiratory insufficiency (forced vital capacity < 50%) and/or normal ECG
- Individuals who are not yet or insufficiently stabilized on psychiatric medication
- Any medical and social condition that may interfere with the study at the discretion of the medical coordinator
- Guardianship
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site
Institute of Mythology
Paris, 75651
Tanya STOJKOVIC, Md, 01 42 16 37 76, t.stojkovic@institut-myologie.org
About this study
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition. However, in the event of future treatment, it is essential for clinical and research teams to have as much information as possible about the disease, particularly regarding its physical, physiological, biological, and neurological aspects.
In this context, it was consider as vital to investigate in greater depth the links between the presence of certain dystrophin isoforms in patients and their potential impacts on cognitive, neurological, and muscular function. The results of this study could serve as a reference for future research and improve the understanding and management of this disease.
All of this data will allow for a precise evaluation of the effects of a potential treatment on the progression of the disease.
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker, MNR, Brain, Muscle, Neuropsycology, Natural history
- 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 |
|---|---|---|
| BMD patients Dp140+ | Dp140+ | |
| BMD patients Dp140- | Dp140- | |
| Healthy subjects | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Brain volume
Measurement of grey and white matter volume (in mm3) using brain MRI sequences.
Measured: 2 years
Brain cortical thickness
Measurement of cortical thickness (in mm) using brain MRI sequences.
Measured: 2 years
Brain water motion
Measurement of diffusion-based values, including apparent diffusion coefficient (in m2/s) and fractional anisotropy (unitless) using diffusion-weighted MRI sequences.
Measured: 2 years
Brain iron metabolism
Measurement of brain iron metabolism using MRI-based quantitative susceptibility mapping (measured in parts per million).
Measured: 2 years
Brain function: resting-state and following task
Measurement of resting-state brain function (default-mode-network) using functional MRI. Measurement of brain activation using fMRI following fronto-executive task (Stroop test).
Measured: 2 years
Intelligence test
Evaluation of intelligence using Wechsler Scale of Intelligence (WAIS-4).
Measured: 2 years
Psychological evaluation of depression
Psychological evaluation using Beck Depression Inventory-II (BDI-II).
Measured: 2 years
Psychological evaluation of anxiety
Psychological evaluation using State-Trait Anxiety Inventory-2 (STAI-Y2).
Measured: 2 years
Psychological evaluation of ADHD
Psychological evaluation using Connors Adult Rating Scale.
Measured: 2 years
Neuropsychological evaluation of memory for attention and cognitive processing
Neuropsychological cognitive evaluation of memory for attention and cognitive processing using Free and Cued Selective Reminding Test (FCSRT).
Measured: 2 years
Neuropsychological evaluation of visual memory and executive function
Neuropsychological cognitive evaluation of visual memory and executive function using Rey-Osterrieth Complex Figure Test (ROCF).
Measured: 2 years
Neuropsychological evaluation of visual attention and task switching
Neuropsychological cognitive evaluation of visual attention and task switching using Trail Making Test.
Measured: 2 years
Neuropsychological evaluation of inhibition
Neuropsychological cognitive evaluation of inhibition using Stroop Color and Word Test.
Measured: 2 years
Neuropsychological evaluation of verbal functioning
Neuropsychological cognitive evaluation of verbal functioning using Phonemic verbal fluency test.
Measured: 2 years
Neuropsychological evaluation of facial expressions.
Neuropsychological cognitive evaluation of facial expressions using Pictures of Facial Affect test.
Measured: 2 years
Neuropsychological evaluation of empathy
Neuropsychological cognitive evaluation understanding inappropriateness, speaker's intentions/motivations, speaker's belief, empathy (participant's ability to know how the character would feel), and finally story comprehension using Faux pas Recognition Test. Empathy is also evaluated using The Interpersonal Reactivity Index.
Measured: 2 years
Secondary outcomes (13)
Evaluation of muscle disease progression
Evaluation of disease progression based on quantitative assessment of MRI-based muscle fat fraction (FF, in %) and residual muscle (i.e. contractile cross-sectional area, in cm2) in lower limb (individual thigh and leg muscles).
Measured: 2 years
Evaluation of muscle disease activity (edema, inflammation, ...)
Evaluation of qMRI-biomarkers sensitive to active muscle damage or disease activity such as water T2 and water T1 mapping, expressed in milliseconds (individual thigh and leg muscles).
Measured: 2 years
Evaluation of muscle extracellular volume
Evaluation of extracellular volume (ECV) in muscle (unitless or %) will be assessed using qMRI-based T1 mapping before and after contrast agent injection (individual thigh and leg muscles).
Measured: 2 years
Evaluation of energy metabolism.
Evaluation of energy metabolism using qMRI and 31P magnetic resonance spectroscopy.
Measured: 2 years
Evaluation of intramuscular pH.
Evaluation of intramuscular pH using qMRI and 31P magnetic resonance spectroscopy.
Measured: 2 years
Evaluation of gait endurance (timed test)
Evaluation of gait endurance using 6-minute walk test (6MWT) and 10-m walking test (10mWT).
Measured: 2 years
Evaluation of exertion and dyspnea (timed test)
Evaluation of exertion and dyspnea using 30-second sit-to-stand test (30STS).
Measured: 2 years
Evaluation of TUG test performance
Evaluation of performance at time-up-and-go test (TUG).
Measured: 2 years
Motor Function assessment
Evaluation of motor function abilities using quantitative Motor Function Measurement (MFM) scale.
Measured: 2 years
Muscle strength evaluation
Strength evaluation of knee extension/flexion, dorsiflexion/plantarflexion of ankle, hand grip and fingers' pinch strength using dedicated MyoTools.
Measured: 2 years
Evaluation of quality of life
Evaluation of The "Quality of Life in genetic Neuromuscular Disease" questionnaire (QoL-gNMD).
Measured: 2 years
Health assessment
Health assessment using the Health Assessment Questionnaire (HAQ).
Measured: 2 years
Assessment of individual's activity level
Assessment of individual's activity level using Ricci et Gagnon Questionnaire.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study start15 Dec 2026 (estimated)
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker Muscular DystrophyStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institut de Myologie, France
- Sponsor type
- University, hospital or other
- Collaborators
- Paris Brain Institute (ICM), Université Paris Cité, Association Française contre les Myopathies (AFM), Paris
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07740057
- Sponsor's protocol ID
- 2026-A00317-44
- Acronym
- MyoBrain-BMD
- Status last verified
- Nov 2026
- First submitted
- 7 Jul 2026
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
- ClinicalTrials.gov ID
- NCT07740057
- Acronym / sponsor ID
- MyoBrain-BMD / 2026-A00317-44
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker, MNR, Brain, Muscle, Neuropsycology, Natural history
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Institut de Myologie, FranceUniversity, hospital or other
- Collaborators
- Paris Brain Institute (ICM), Université Paris Cité, Association Française contre les Myopathies (AFM), Paris
- Study start
- 15 Dec 2026 (estimated)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 31 Jul 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.
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition. However, in the event of future treatment, it is essential for clinical and research teams to have as much information as possible about the disease, particularly regarding its physical, physiological, biological, and neurological aspects.
In this context, it was consider as vital to investigate in greater depth the links between the presence of certain dystrophin isoforms in patients and their potential impacts on cognitive, neurological, and muscular function. The results of this study could serve as a reference for future research and improve the understanding and management of this disease.
All of this data will allow for a precise evaluation of the effects of a potential treatment on the progression of the disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BMD patients Dp140+ | – | –Dp140+ |
| BMD patients Dp140- | – | –Dp140- |
| Healthy subjects | – | – |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
- Study population
- Fifty patients will be included, including 25 patients having the cerebral Dp140 isoform (Dp140+) and 25 patients lacking the cerebral Dp140 isoform (Dp140-). A same number (n=25) of age-matched healthy volunteers will be included as well.
Key inclusion criteria (3 listed)
- Genetically confirmed diagnosis of Becker muscular dystrophy
- Ambulant
- Signed ICF
Key exclusion criteria (10 listed)
- Individuals presenting contraindications to MRI
- History of allergy to contrast product
- Individuals with history of neurological diseases interfering with the study
- Individuals with concomitant medical conditions that could interfere with the study outcomes
- Individuals who are not yet or insufficiently stabilized on psychiatric medication
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
Brain volume
Time frame: 2 years
Measurement of grey and white matter volume (in mm3) using brain MRI sequences.
Brain cortical thickness
Time frame: 2 years
Measurement of cortical thickness (in mm) using brain MRI sequences.
Brain water motion
Time frame: 2 years
Measurement of diffusion-based values, including apparent diffusion coefficient (in m2/s) and fractional anisotropy (unitless) using diffusion-weighted MRI sequences.
Brain iron metabolism
Time frame: 2 years
Measurement of brain iron metabolism using MRI-based quantitative susceptibility mapping (measured in parts per million).
Brain function: resting-state and following task
Time frame: 2 years
Measurement of resting-state brain function (default-mode-network) using functional MRI. Measurement of brain activation using fMRI following fronto-executive task (Stroop test).
Intelligence test
Time frame: 2 years
Evaluation of intelligence using Wechsler Scale of Intelligence (WAIS-4).
Psychological evaluation of depression
Time frame: 2 years
Psychological evaluation using Beck Depression Inventory-II (BDI-II).
Psychological evaluation of anxiety
Time frame: 2 years
Psychological evaluation using State-Trait Anxiety Inventory-2 (STAI-Y2).
Psychological evaluation of ADHD
Time frame: 2 years
Psychological evaluation using Connors Adult Rating Scale.
Neuropsychological evaluation of memory for attention and cognitive processing
Time frame: 2 years
Neuropsychological cognitive evaluation of memory for attention and cognitive processing using Free and Cued Selective Reminding Test (FCSRT).
Neuropsychological evaluation of visual memory and executive function
Time frame: 2 years
Neuropsychological cognitive evaluation of visual memory and executive function using Rey-Osterrieth Complex Figure Test (ROCF).
Neuropsychological evaluation of visual attention and task switching
Time frame: 2 years
Neuropsychological cognitive evaluation of visual attention and task switching using Trail Making Test.
Neuropsychological evaluation of inhibition
Time frame: 2 years
Neuropsychological cognitive evaluation of inhibition using Stroop Color and Word Test.
Neuropsychological evaluation of verbal functioning
Time frame: 2 years
Neuropsychological cognitive evaluation of verbal functioning using Phonemic verbal fluency test.
Neuropsychological evaluation of facial expressions.
Time frame: 2 years
Neuropsychological cognitive evaluation of facial expressions using Pictures of Facial Affect test.
Neuropsychological evaluation of empathy
Time frame: 2 years
Neuropsychological cognitive evaluation understanding inappropriateness, speaker's intentions/motivations, speaker's belief, empathy (participant's ability to know how the character would feel), and finally story comprehension using Faux pas Recognition Test. Empathy is also evaluated using The Interpersonal Reactivity Index.
13 secondary outcomes
Evaluation of muscle disease progression
Time frame: 2 years
Evaluation of muscle disease activity (edema, inflammation, ...)
Time frame: 2 years
Evaluation of muscle extracellular volume
Time frame: 2 years
Evaluation of energy metabolism.
Time frame: 2 years
Evaluation of intramuscular pH.
Time frame: 2 years
Evaluation of gait endurance (timed test)
Time frame: 2 years
Evaluation of exertion and dyspnea (timed test)
Time frame: 2 years
Evaluation of TUG test performance
Time frame: 2 years
Motor Function assessment
Time frame: 2 years
Muscle strength evaluation
Time frame: 2 years
Evaluation of quality of life
Time frame: 2 years
Health assessment
Time frame: 2 years
Assessment of individual's activity level
Time frame: 2 years
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 3 Nov 2026.
- Individual participant dataSharing planned: No
Related trials for Becker Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
RecruitingObservational study
Duchenne Electronic Health Record Study
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
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 Becker Muscular Dystrophy, run by Institut de Myologie, France 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 10 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: opening soon, as of the sponsor's last update on 3 Nov 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 Harmen REYNGOUDT, PhD (01 42 16 58 91, h.reyngoudt@institut-myologie.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Brain volume (time frame: 2 years). The study lists 16 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 3 Nov 2026.