Clinical trials: Becker Muscular Dystrophy
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD.
- Trial name
- NCT07160634
- Testing
- SGT-003
- Where
- 7 sites in United States, Australia and 1 more
- Volunteers
- 80 people
- Sponsor
- Solid Biosciences Inc.
Muscular Dystrophy, Duchenne, Orthotic Devices
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.
- Conditions
- Muscular Dystrophy, Duchenne, Orthotic Devices, Upper Extremity, Neuromuscular Diseases (NMD)
- Trial name
- NCT06363357
- Testing
- Shoulder orthosis
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Seoul National University Hospital
Duchenne Muscular Dystrophy
A Registered Cohort Study on Duchenne Muscular Dystrophy
Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy.
- Trial name
- NCT04012671
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Ning Wang, MD., PhD.
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD.
- Trial name
- NCT03375164
- Testing
- delandistrogene moxeparvovec
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Becker, Muscular Dystrophy
A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children
This is a prospective, multicenter, longitudinal observational cohort study aimed at understanding the progression of Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophy, Becker, Muscular Dystrophy, Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
- Trial name
- WEST-DBMD (NCT07511920)
- Testing
- No interventions are applied; participants are followed for natural history observation only
- Where
- China
- Volunteers
- 500 people
- Sponsor
- West China Second University Hospital
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized…
- Trial name
- FOX (NCT06100887)
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 7 sites in United States
- Volunteers
- 43 people
- Sponsor
- Edgewise Therapeutics, Inc.
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
The aim of this multicentre, prospective, double blind, placebo controlled, randomized pilot study is to investigate safety and tolerance of Epigallocatechin-Gallate (EGCG, the major polyphenol in green tea) in patients with…
- Trial name
- SUNIMUD (NCT01183767)
- Testing
- Epigallocatechin-Gallate
- Where
- 3 sites in Germany
- Volunteers
- 33 people
- Sponsor
- Charite University, Berlin, Germany
Duchenne Muscular Dystrophy (DMD)
Twice Weekly Steroids and Exercise as Therapy for DMD
The study team will determine the potential of low dose twice weekly prednisone and whether exercise training can synergize to delay disease progression and improve muscle strength/physical function in boys with Duchenne muscular…
- Trial name
- NCT04322357
- Testing
- Prednisone, Exercise Training on standard steroid regimen, Exercise training on twice-weekly steroids
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed…
- Trial name
- GalaxyDMD (NCT03917719)
- Testing
- Edasalonexent
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 130 people
- Sponsor
- Catabasis Pharmaceuticals
Duchenne Muscular Dystrophy, Muscular Dystrophies
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
This pilot study aims to assess subcellular muscle structure in patients with Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) in comparison to healthy volunteers using multispectral optoacoustic tomography (MSOT).
- Conditions
- Duchenne Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy, Duchenne
- Trial name
- MSOT_DMD (NCT03490214)
- Testing
- Multispectral Optoacoustic Tomography
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
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
Aerobic Exercise in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births.
- Trial name
- NCT04173234
- Testing
- Aerobic Training, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).
- Trial name
- NCT02704325
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Sponsor
- Kevin Flanigan
Becker Muscular Dystrophy
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
- Trial name
- MESA (NCT06066580)
- Testing
- Sevasemten
- Where
- 48 sites in United States, Australia and 10 more
- Volunteers
- 260 people
- Sponsor
- Edgewise Therapeutics, Inc.
Duchenne Muscular Dystrophy
Upper Limb Assessment in Duchenne Muscular Dystrophy
The literature on outcome measures assessing upper limbs in Duchenne muscular dystrophy (DMD) is quite scanty. While there have been considerable advances for ambulant DMD boys, no prospective study has so far been devoted to…
- Trial name
- PUL in DMD (NCT02436720)
- Where
- Italy
- Volunteers
- 300 people
- Sponsor
- Catholic University of the Sacred Heart
Duchenne Muscular Dystrophy, Upper Limb Function
Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy
Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage.
- Conditions
- Duchenne Muscular Dystrophy, Upper Limb Function, Respiratory Function Test
- Trial name
- NCT03552874
- Testing
- The Performance of Upper Limb Questionnaire (PUL)
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
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
Duchenne Muscular Dystrophy (DMD)
Nutritional Status and Therapy in DMD Patients
Brief Summary The goal of this clinical trial is to evaluate the impact of a disease-specific, individualized diet on the nutritional status and functional abilities of Duchenne muscular dystrophy (DMD) patients.
- Trial name
- NCT06682585
- Testing
- Individualised dietary intervention
- Where
- Turkey (Türkiye)
- Volunteers
- 9 people
- Sponsor
- Ankara University