Clinical trials: Duchenne's Muscular Dystrophy
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 (DMD)
Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD
we measure balance and quadriceps strength in Duchenne muscular dystrophy children after applying virtual reality and whole body vibration
- Trial name
- NCT07338812
- Testing
- virtual reality, whole body vibration
- Where
- 2 sites in Egypt
- Volunteers
- 50 people
- Sponsor
- Cairo University
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
Duchenne Muscular Dystrophy
DMD- Interactive Virtual Reality Study
This study will help determine if an Interactive Virtual Reality system can improve the physiotherapy of young patients with Duchenne muscular dystrophy (DMD).
- Trial name
- DMD-IVR (NCT06274983)
- Testing
- IVR platform trial
- Where
- United Kingdom
- Volunteers
- 14 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Muscular Dystrophies, Muscular Dystrophy, Duchenne
User-centred Assistive System for Arm Functions in Neuromuscular Subjects
Restore a lost function is a special experience for people affected by neuromuscular evolutive diseases. "From the patient's point of view improvement is measured by regaining lost abilities,-by being able to do something…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Muscular Dystrophy, Limb-Girdle Type 2
- Trial name
- USEFUL (NCT03127241)
- Testing
- Assistive device (Armon Ayura) test, Assistive device (Jaeco Wrex) test
- Where
- 2 sites in Italy
- Volunteers
- 38 people
- Sponsor
- Politecnico di Milano
Duchenne Muscular Dystrophy (DMD)
Usability and User Experience of a Virtual Reality Rehabilitation Game Platform in Individuals With Duchenne Muscular Dystrophy
This study aims to evaluate the usability, user experience, and tolerability of a virtual reality-based rehabilitation game platform specifically developed for individuals with Duchenne muscular dystrophy.
- Trial name
- DMD-VR-UE (NCT07565272)
- Testing
- Virtual Reality Gameplay Session
- Where
- Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Istanbul University
Duchenne Muscular Dystrophy
Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY).
- Trial name
- DMD (NCT04529707)
- Testing
- Transdiagnostic Behavioral Sleep Intervention
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- University of Pittsburgh
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT01009294
- Testing
- Ataluren, Chronic Corticosteroid Therapy
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy, Performance
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
The investigator investigated the effect of lower limb flexibility on functional performance of children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03534349
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Motor Imagery in Duchenne Muscular Dystrophy
Motor imagery (MI) can be defined as a dynamic process in which the person is mentally stimulated without performing the given motor movement.
- Trial name
- NCT05559710
- Where
- Turkey (Türkiye)
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy
The aim of this population based study is to examine, quantify and describe physical activity level in Norwegian boys with DMD, and to compare the level of physical activity level between boys with DMD and age matched healthy…
- Trial name
- NCT03947112
- Testing
- Physical activity registration
- Where
- Norway
- Volunteers
- 28 people
- Sponsor
- Haukeland University Hospital
Muscular Diseases
Non-invasive Molecular Imaging of Muscle Structure (MSOT_muscles)
This study aims to determine hemoglobin and collagen levels in muscles before and after exercise, and over time, using Multispectral Optoacoustic Tomography (MSOT).
- Trial name
- MSOT_muscles (NCT03979157)
- Testing
- Multispectral Optoacoustic Tomography
- Where
- Germany
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
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
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS)
Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias
Human induced pluripotent stem cells (hiPSCs) have driven a paradigm shift in the modeling of human disease; the ability to reprogram patient-specific cells holds the promise of an enhanced understanding of disease mechanisms and…
- Conditions
- Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- Trial name
- NCT02413450
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Duchenne Muscular Dystrophy
Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy
Whole-body vibration therapy (WBVT) is a novel, non-pharmacological intervention aimed at improving muscle strength and endurance as well as bone density.
- Trial name
- NCT01954940
- Testing
- Whole Body Vibration Therapy
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Children's Hospital of Eastern Ontario
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.
Duchenne Muscular Dystrophy
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
- Trial name
- NCT02752048
- Testing
- TAS-205
- Where
- 11 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Muscular Dystrophy, Duchenne
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded…
- Trial name
- NCT03769116
- Testing
- delandistrogene moxeparvovec
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- Sarepta Therapeutics, Inc.
Becker Muscular Dystrophy
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 175 adult participants in a cohort…
- Trial name
- NCT05291091
- Testing
- Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg
- Where
- 51 sites in United States, Australia and 10 more
- Volunteers
- 244 people
- Sponsor
- Edgewise Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.