Clinical trials: DMD, open to volunteers
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 4 sites in Georgia, New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
Duchene Muscular Dystrophy
Home-Based Tele-Rehabilitation for Respiratory Function in Children With Duchenne Muscular Dystrophy
This study evaluates the feasibility and preliminary clinical effects of a home-based tele-rehabilitation program aimed at improving respiratory function in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT07629284
- Testing
- Home-Based Respiratory Telerehabilitation Program
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Samsung Medical Center
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
Duchenne Muscular Dystrophy (DMD)
Factors Influencing Physical Activity Levels in Children With Duchenne Muscular Dystrophy: An ICF-CY-Based Study
Duchenne Muscular Dystrophy (DMD) is a neuromuscular disease characterized not only by progressive muscle weakness but also by cognitive, behavioral, and psychosocial impairments.
- Trial name
- NCT07369609
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
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
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Trial name
- ENHANCE (NCT07542314)
- Testing
- ELEVIDYS, Sirolimus, Glucocorticoids
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Trial of Cell Based Therapy for DMD
This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals…
- Trial name
- NCT06692426
- Testing
- MyoPAXon, Tacrolimus
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Duchenne Muscular Dystrophy (DMD)
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD) using computer vision technology.
- Trial name
- NCT06755138
- Testing
- Observational Assessment
- Where
- South Korea
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Seoul National University Hospital
Duchenne Muscular Dystrophy, Duchenne Disease
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children
- Trial name
- NCT06290713
- Testing
- Tadalafil, Exercise Training
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
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
Duchenne Muscular Dystrophy (DMD)
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy
The aim of the BABY DUCHENNE study is to evaluate the natural history and characterize the early clinical outcomes in very young children (0-3 years) with Duchenne muscular dystrophy (DMD) identified by newborn screening programs.
- Trial name
- BABY DUCHENNE (NCT07092540)
- Where
- 4 sites in United States
- Volunteers
- 105 people
- Sponsor
- University of Rochester
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Duchenne Muscular Dystrophy (DMD)
The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder characterized by progressive muscle degeneration and frequent developmental, cognitive and behavioral impairments, occurring in one in 5000 live births of boys.
- Trial name
- DMD-DUAL-FUNC (NCT06887491)
- Testing
- Conventional physiotherapy program, Conventional physiotherapy program + Dual task program
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Lokman Hekim University
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
Child, Only
Effects of Posture and Quality of Life in Duchenne Muscular Dystrophy Children
Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder predominantly affecting boys and men, with an estimated incidence of 1 in 3,800 to 1 in 6,200 live male births.
- Trial name
- NCT07436143
- Testing
- Alexander Technique Program
- Where
- Pakistan
- Volunteers
- 33 people
- Sponsor
- Riphah International University
Duchenne Muscular Dystrophy (DMD)
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients.
- Trial name
- CHERISH (NCT07172971)
- Testing
- SGLT-2 inhibitor, SGLT2 inhibitor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Vanderbilt University Medical Center
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen…
- Trial name
- EVOLVE (NCT06606340)
- Testing
- Eteplirsen, Golodirsen, Casimersen
- Where
- 23 sites in United States
- Volunteers
- 300 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac dysfunction in male children, adolescents, and young adults with…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Cardiomyopathy
- Trial name
- NCT07515235
- Where
- Greece
- Volunteers
- 65 people
- Sponsor
- Aristotle University Of Thessaloniki