Clinical trials: Duchenne Muscular Dystrophy (DMD)
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to muscle weakness, breathing difficulties, heart disease, and early death.
- Trial name
- NCT07642635
- Testing
- Semaglutide (Rybelsus®)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University Medical Center
Muscular Dystrophy, Duchenne
Study of Genotype and Phenotype Characterization in Duchenne Muscular Dystrophy With Small Mutations
Improved standards of care and the regular early use of glucocorticoid treatment have changed the natural history of Duchenne muscular dystrophy (DMD), affecting both survival and time of loss of functional milestones.
- Trial name
- NCT05833633
- Testing
- MUSCLE MAGNETIC RESONANCE IMAGING (MRI)
- Where
- Italy
- Volunteers
- 17 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Duchene Muscular Dystrophy
How Can Different Forms of Strengthening Exercises Affect the Muscular Tissue and Its Chemical Markers in Children With Duchenne Muscular Dystrophy
The treatment of patients diagnosed with DMD needs to be multidisciplinary, judicious, and always focused on the patient's well-being. Physiotherapy and occupational therapy are very often standard care.
- Trial name
- NCT07581743
- Testing
- strengthening exercises, Low-Intensity Exercise Protocol, traditional physical therapy program
- Where
- Egypt
- Volunteers
- 45 people
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the…
- Trial name
- NCT01772043
- Where
- 9 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Executive Dysfunction, Occupational Problems
Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy
The effects of Duchenne muscular dystrophy and its treatments on executive functions and occupational performance are under-mentioned in the literature.
- Conditions
- Executive Dysfunction, Occupational Problems, Duchenne Muscular Dystrophy
- Trial name
- NCT06868784
- Testing
- Evaluations
- Where
- Turkey (Türkiye)
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Muscular Dystrophy, Duchenne
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
This study was planned to investigate the parental influence on physical activity (PA) level and participation in ambulatory children with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05209087
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the…
- Trial name
- NCT03433807
- Testing
- Idebenone
- Where
- 12 sites in United States
- Sponsor
- Santhera Pharmaceuticals
Duchenne Muscular Dystrophy
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Duchenne's Muscular Dystrophy, Scoliosis
Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy
Nearly all patients with Duchenne's Muscular Dystrophy (DMD) have scoliosis. Posterior instrumented spinal fusion, which is a surgery to correct scoliosis, has been shown to improve quality of life and satisfaction of both…
- Trial name
- NCT00207857
- Where
- United States
- Sponsor
- Children's Healthcare of Atlanta
Neuromuscular Diseases
Muscle Oxygenation in Effort in Neuromuscular Diseases
Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.
- Trial name
- OXYNEMU (NCT02789059)
- Testing
- muscle oxygenation
- Where
- 3 sites in France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
Duchenne Muscular Dystrophy
Telerehabilitation in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive inherited disease that affects the muscles and causes functional limitations to varying degrees. It is vital to start physiotherapy follow-ups immediately after diagnosis.
- Trial name
- Duchenne (NCT05305976)
- Testing
- Telerehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Duchenne Muscular Dystrophy (DMD)
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and…
- Trial name
- NCT02036463
- Testing
- Prednisone
- Where
- United States
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Duchenne Muscular Dystrophy
Stacking Exercises Aid the Decline in FVC and Sick Time
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths…
- Trial name
- STEADFAST (NCT01999075)
- Testing
- Lung Volume Recruitment (LVR), Conventional Treatment
- Where
- 9 sites in Canada
- Volunteers
- 70 people
- Sponsor
- Children's Hospital of Eastern Ontario
Duchenne Muscular Dystrophy
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester
Duchenne Muscular Dystrophy
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or…
- Trial name
- NCT04179409
- Testing
- Amondys 45, Exondys 51, Vyondys 53
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy, Performance
Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy
Investigator investigated that regression of hamstring flexibility and performance in children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03589612
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Hacettepe University