Muscular Dystrophy, Duchenne Clinical Trial
Official title:
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.
Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT01865084 -
A Study of Tadalafil for Duchenne Muscular Dystrophy
|
Phase 3 | |
Completed |
NCT00243789 -
Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Completed |
NCT00033189 -
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT03703882 -
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
|
Phase 3 | |
Enrolling by invitation |
NCT04626674 -
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 1 | |
Completed |
NCT02286947 -
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT03406780 -
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT01826487 -
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
|
Phase 3 | |
Completed |
NCT02710591 -
Rimeporide in Patients With Duchenne Muscular Dystrophy
|
Phase 1 | |
Completed |
NCT01826422 -
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
|
N/A | |
Completed |
NCT00102453 -
Pentoxifylline in Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Terminated |
NCT02090959 -
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
|
Phase 3 | |
Recruiting |
NCT05833633 -
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
|
||
Completed |
NCT05209087 -
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
|
||
Completed |
NCT03789734 -
Safety Study of BLS-M22 in Healthy Volunteers
|
Phase 1 | |
Recruiting |
NCT05126758 -
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 3 | |
Completed |
NCT00016653 -
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
|
Phase 2/Phase 3 | |
Completed |
NCT03127241 -
User-centred Assistive System for Arm Functions in Neuromuscular Subjects
|
N/A | |
Completed |
NCT03490214 -
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
|
N/A | |
Completed |
NCT03179631 -
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
|
Phase 3 |