Clinical trials: Muscular Dystrophy, Becker
Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA.
- Trial name
- SEE-NMD (NCT07356063)
- Testing
- Multicomponent Intervention
- Where
- Pakistan
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Duchenne / Becker Muscular Dystrophy
Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic…
- Trial name
- NCT02470962
- Testing
- Observation
- Where
- Switzerland
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
Muscular Dystrophy, Duchenne
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in…
- Trial name
- NCT02834650
- Testing
- Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test
- Where
- 3 sites in United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Stanford University
Duchenne Muscular Dystrophy
Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research…
- Trial name
- NCT00468832
- Where
- 21 sites in United States, Argentina and 6 more
- Volunteers
- 551 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
The task consists in reach as much bubbles as they can, the bubbles appear on the screen of the computer and should be reached in 10 seconds.
- Trial name
- NCT02891434
- Testing
- Acquisition on TouchScreen, Acquisition on Kinect, Acquisition on LeapMotion
- Where
- Brazil
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy, Burden, Dependency
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease.
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management
- Trial name
- NCT03680365
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Jett Foundation, Inc.
Neuromuscular Diseases in Children, Respiratory Function Impaired
Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
The clinical trial titled "Investigation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases" aims to evaluate the respiratory functions, thoracoabdominal movements, and exercise…
- Conditions
- Neuromuscular Diseases in Children, Respiratory Function Impaired, Respiratory Insufficiency, Duchenne Muscular Dystrophy
- Trial name
- NCT06756633
- Testing
- Respiratory Function Test, Structured light plethysmography, 6 Minute Walking Test
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
Becker Muscular Dystrophy
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition.
- Trial name
- MyoBrain-BMD (NCT07740057)
- Where
- France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Duchenne Muscular Dystrophy (DMD), Lower Urinary Tract Symptoms (LUTS)
Urotherapy Education for Families of Children With Duchenne Muscular Dystrophy
This study aims to investigate the effects of urotherapy education provided to families of children with Duchenne muscular dystrophy on parents' knowledge, lifestyle changes, and satisfaction with the education.
- Trial name
- NCT07821125
- Testing
- Urotherapy Education
- Where
- Turkey (Türkiye)
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Becker Muscular Dystrophy
Sodium Nitrate to Improve Blood Flow
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02847975
- Testing
- Sodium nitrate
- Where
- 2 sites in United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Duchenne Muscular Dystrophy (DMD)
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
- Trial name
- IBISD (NCT01380964)
- Where
- France
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Genethon
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials…
- Trial name
- NCT01484678
- Where
- 3 sites in United States
- Volunteers
- 550 people, healthy volunteers welcome
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 2
The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.
- Trial name
- NCT04668716
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 339 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust