Clinical trials: Becker Muscular Dystrophy
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 or Severe Becker Myopathy
IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère
Clinical, prospective and monocentric study aiming at assessing the feasibility of fibrosis detection and quantification (and of function assessment) during MRI without breath-holds in a population of adults and children with…
- Trial name
- NCT02078076
- Testing
- Magnetic Resonance Cardiac Imaging (with Gadolinium)
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Duchenne / Becker Muscular Dystrophy
Transition to Adulthood in People With Muscular Dystrophy
The study will involve boys with Duchenne Muscular Dystrophy (DMD), from adolescence to adulthood living in Italy, and their families/ caregivers.
- Trial name
- TAMDY (NCT07101185)
- Testing
- VIRTUAL REALITY
- Where
- Italy
- Volunteers
- 250 people
- Sponsor
- IRCCS Eugenio Medea
Becker Muscular Dystrophy
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
- Trial name
- ARCH (NCT05160415)
- Testing
- Sevasemten
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Edgewise Therapeutics, Inc.
Muscular Dystrophy, Duchenne Muscular Dystrophy
Follow-up Study on Female Carriers With DMD Gene Variants
Background Duchenne and Becker muscular dystrophies are X-linked recessive allelic disorders caused by mutations of the dystrophin gene on chromosome Xp21.
- Conditions
- Muscular Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Trial name
- NCT05715957
- Testing
- No intervention
- Where
- Denmark
- Volunteers
- 103 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy
Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence
Background and aim: Neuromuscular diseases encompass a range of conditions affecting muscle cells, nerves, or the interaction between the two.
- Conditions
- Becker Muscular Dystrophy, FSHD - Facioscapulohumeral Muscular Dystrophy, Hypokalemic Periodic Paralysis
- Trial name
- NCT06917430
- Testing
- No intervention
- Volunteers
- 120 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies.
The purpose of this study is to analyze the effectiveness of a 5-weeks respiratory digital intervention program in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Muscular Dystrophy
- Trial name
- DMDrespy2024 (NCT06363526)
- Testing
- Digital respiratory intervention program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy
UTSW HP [13-C] Pyruvate Injection in HCM
The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal…
- Conditions
- Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis
- Trial name
- HPHCM (NCT03057002)
- Testing
- Hyperpolarized 13C-Pyruvate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
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
Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI)
Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
The primary aim of this observational study is to investigate specific aspects of social cognition in dystrophinopathies. Body awareness, interpersonal distance and emotional processing will be measured in a sample of patients…
- Conditions
- Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI), Social Cognition
- Trial name
- NCT06874166
- Testing
- Electroencephalography (EEG), Social Cognition Tasks
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Becker Muscular Dystrophy
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
Objectives: Primary objective: to establish the histological effects of Givinostat versus placebo administered over 12 months. Secondary Objectives: To establish the macroscopic muscle effects of Givinostat versus placebo…
- Trial name
- NCT03238235
- Testing
- Givinostat
- Where
- 2 sites in Italy, Netherlands
- Volunteers
- 51 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
Longitudinal prospective observational study. This is a 24-month study with the possibility of extending the data time points. Initially baseline, then 12 and 24 months follow up studies will be completed.
- Trial name
- NCT02972580
- Testing
- Genetic characterization
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies
Taking part in community activities is essential for health and well-being. Yet, it is highly restricted for young people with Duchenne and Becker Muscular Dystrophies (DBMD), especially as they grow into adulthood.
- Trial name
- NCT06540365
- Testing
- Pathways and Resources for Engagement and Participation (PREP)
- Volunteers
- 6 people
- Sponsor
- University of Haifa
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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
Muscular Dystrophy, Duchenne and Becker Types
Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.
Duchenne's Muscular Dystrophy and Becker Dystrophy, hereafter DMD and BMD, is a serious and progressive disease that affects 1 in 3,500-6,000 males born alive.
- Trial name
- RVDuchenne (NCT03879304)
- Testing
- multimodal physiotherapy program with RV, traditional physiotherapy program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis.
- Conditions
- Muscular Dystrophies, Limb Girdle Muscular Dystrophy, Facio-Scapulo-Humeral Dystrophy, Becker Muscular Dystrophy
- Trial name
- RIMUDI (NCT06378203)
- Testing
- tele-rehabilitation system with virtual reality Home Kit - Virtual Reality Rehabilitation System (VRRS KHYMEIA)
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Eugenio Medea
Duchenne or Becker Muscular Dystrophy
Effectiveness of Aquatic Therapy in Children With Duchenne and Becker Muscular Dystrophy
The purpose of this study is ; to evaluate the effects of aquatic therapy applied in addition to conventional physical therapy on balance, functionality and quality of life in children with Duchenne and Becker muscular dystrophy.
- Trial name
- NCT06186310
- Testing
- Aquatic Therapy Group, Conventional Physical Therapy Group
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Gaziosmanpasa Research and Education Hospital
Duchenne and Becker Muscular Dystrophy, Polycytemia Vera
Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval
The study will evaluate the effect of a therapeutic dose and a supratherapeutic dose of ITF2357 on the QT/QTc interval.
- Trial name
- NCT04821063
- Testing
- ITF2357 10 mg/mL, Moxifloxacin Hydrochloride
- Where
- Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Italfarmaco