Clinical trials: Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study
Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is…
- Trial name
- NCT06295718
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Sahra Şirvan
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
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
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Muscular Dystrophy, Duchenne
Characterization of DupEx2 Duchenne Muscular Dystrophy
To characterize the clinical phenotype and possible predictive/prognostic factors of patients with Duchenne muscular dystrophy (DMD) due to duplication of exon 2 (Dup2).
- Trial name
- DMDDup2 (NCT06337669)
- Where
- Italy
- Volunteers
- 26 people
- Sponsor
- IRCCS San Raffaele
Becker Muscular Dystrophy
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG).
- Trial name
- NCT01539772
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 85 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Neurologic Disorder, Duchenne
Physical Therapy Treatment on Children and Adolescents With Neurological Pathologies
To evaluate the positive effects of a home-based physical therapy intervention added to the usual physical therapy programs performed in children with neurological pathologies that induce sensorimotor impairments that affect…
- Conditions
- Neurologic Disorder, Duchenne, Pediatric Disorder, Sensorimotor Disorder Nos
- Trial name
- NCT05313295
- Testing
- Home-based physiotherapy, Usual Physiotherapy
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Universidad de Almeria
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
DMD-associated Dilated Cardiomyopathy
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age
This study aims at exploring the use of empagliflozin in children and adolescents 6-18 years old with Duchenne muscular distrophy (DMD) - associated cardiomyopathy.
- Trial name
- REDMeD (NCT06643442)
- Testing
- Empagliflozin Tablets
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Sebastiano Lava
Spina Bifida, Neuromuscular Diseases
Metformin in Children With Motor Deficit
Obesity with insulin resistance in the paediatric population provides an increasing challenge. Children with neurological or neuromuscular diseases are even more prone to obesity: their locomotor impairment leads to an…
- Trial name
- NCT00720161
- Testing
- Metformin
- Where
- Belgium
- Volunteers
- 42 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
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, Cardiomyopathies
The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
Purpose This cardiac dysfunction in patients with Duchenne muscular dystrophy is associated with minor cardiac damage as indicated by elevation of plasma cardiac troponin I (cTnI).
- Trial name
- NCT00606775
- Testing
- Carvedilol
- Where
- Japan
- Volunteers
- 60 people
- Sponsor
- Suzuka Hospital
Duchenne Muscular Dystrophy
Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.
- Trial name
- NCT05249361
- Testing
- no intervention
- Where
- 2 sites in South Korea
- Volunteers
- 48 people
- Sponsor
- Samsung Medical Center
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
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Registry for Duchenne and Becker Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- FAIR-DMD (NCT07402122)
- Where
- 30 sites in Austria, Germany
- Volunteers
- 1,500 people
- Sponsor
- Dr. Andreas Ziegler
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
Duchene Muscular Dystrophy
A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy
GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin.
- Trial name
- NCT07856329
- Testing
- GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg)
- Where
- 14 sites in Belgium, France and 2 more
- Volunteers
- 77 people
- Sponsor
- Genethon