Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function

Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age18-50
SponsorInstitut de Myologie, France

About this trial

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.

Eligibility criteria

Qualifiers

Genetically confirmed diagnosis of Becker muscular dystrophy

Ambulant

Signed ICF

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

75 Participants
are grouped into 3 trial groups

Sponsors and collaborators

Institut de Myologie, France

Lead sponsor

Paris Brain Institute (ICM)

Collaborator

Université Paris Cité

Collaborator

Association Française contre les Myopathies (AFM), Paris

Collaborator