Clinical trials: Muscular Dystrophy (DMD)
Duchenne Dystrophy Muscular
Biomechanical and Morphological Changes in Dystrophic Muscle
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in…
- Trial name
- MARCHE-DMD (NCT02472990)
- Where
- 10 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2I
Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
- Trial name
- NCT02165358
- Where
- Denmark
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency)
Evaluation of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand the biochemistry of different types of Limb-Girdle Muscular Dystrophy (LGMD) and to determine appropriate outcome measures for future clinical treatment trials for LGMD.
- Conditions
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
- Trial name
- NCT00893334
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Telerehabilitation-based Motor Imagery Training in Children With Duchenne Muscular Dystrophy
When the field of neurorehabilitation is examined, most of the current physiotherapy and rehabilitation approaches are based on real movements to stimulate damaged motor neural connections through neuroplasticity.
- Trial name
- NCT06109103
- Testing
- telerehabilitation-based motor imagery training, telerehabilitation-based physiotheraphy training
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Biomarker Development for Muscular Dystrophies
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Conditions
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Trial name
- NCT05019625
- Where
- 5 sites in United States
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Duchenne Muscular Dystrophy
Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy
A cross-sectional study was carried out, in which 40 boys, aged 11 to 18 years, were evaluated. The recruitment of groups was carried out at the neuromuscular disease outpatient clinic of the Federal University of São Paulo…
- Trial name
- NCT04740554
- Testing
- Duchenne Muscular Dystrophy group with Deflazacort, Duchenne Muscular Dystrophy group with Prednisone/Predisolone, Duchenne Muscular Dystrophy group without Corticosteroids therapy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
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
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), Becker's Muscular Dystrophy (BMD)
Urinary Titin Biomarker in DMD
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful…
- Trial name
- NCT07332013
- Testing
- Descending stair walk
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
Muscular Dystrophy, Duchenne Muscular Dystrophy,
Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.
This Study is single arm, single centre trial to check the safety and efficacy of Bone Marrow derived autologous cell(100 million per dose) for the patient with Duchenne Muscular Dystrophy.
- Trial name
- mdp (NCT01834066)
- Testing
- Stem Cell
- Where
- India
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
Duchenne Muscular Dystrophy
The Burden of Access in Duchenne Muscular Dystrophy in the US
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these…
- Trial name
- NCT03951675
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Florida
Muscular Dystrophies, Becker Muscular Dystrophy
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child.
- Conditions
- Muscular Dystrophies, Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
- Trial name
- biodystromirs (NCT02109692)
- Testing
- blood sample
- Where
- France
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Duchenne Muscular Dystrophy (DMD), Viscoelastic Property
Duchenne Muscular Dystrophy and the Viscoelastic Properties of Upper Limb Muscles
Muscular dystrophies are hereditary and progressive skeletal muscle diseases that cause degeneration and loss of strength in the muscles.
- Trial name
- NCT07435116
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Sanko University
Other Diseases or Conditions
Mother-caregivers of Children With Duchenne Muscular Dystrophy
The incidence of Duchenne Muscular Dystrophy (DMD) is approximately 1 in 3.500 male newborns. During its progression there is loss of mobility, swallowing difficulties and a significant reduction in respiratory capacity.
- Trial name
- DMDK (NCT01921374)
- Testing
- sleep parameters, Hormonal profile, Inflammatory profile
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Monica Levy Andersen
Muscular Dystrophy
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise.
- Trial name
- NCT02653833
- Testing
- Tadalafil 20 MG, beetroot juice extract
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.
- Trial name
- NCT06839469
- Where
- 3 sites in United States
- Volunteers
- 106 people, healthy volunteers welcome
- Sponsor
- Columbia University
Stress
Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy
Background: Children with Duchenne/Becker Muscular Dystrophy (DBMD) slowly lose muscle function. They usually die at a young age. Some mothers adapt to the demands of caring for a child with this disease better than others.
- Trial name
- NCT02194725
- Where
- United States
- Volunteers
- 228 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)