Clinical trials: Duchenne's Muscular Dystrophy
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) 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.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne
Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy
This study was planned to investigate the parental influence on physical activity (PA) level and participation in ambulatory children with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05209087
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
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 (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
Duchenne Muscular Dystrophy
An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen).
- Trial name
- NCT04708314
- Testing
- Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Rare Disease Research, LLC
Duchenne Muscular Dystrophy
Prognostic Factors Affecting Duchenne Muscular Dystrophy
Determination of prognostic factors affecting ambulation of duchenne muscular dystrophy
- Trial name
- NCT03372655
- Volunteers
- 82 people
- Sponsor
- Assiut University
Muscular Dystrophy, Duchenne
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 (vesleteplirsen) in participants with Duchenne muscular dystrophy (DMD) who participated in…
- Trial name
- NCT03675126
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Develop a New Group Version of the Becker-DeGroot-Marsckek (BDM) Auction to Measure Willingness to Pay of Compound Members for Shared Hardware., Develop a New Survey Instrument to Measure Behavioural Determinants of Hand Washing and Water Treatment Like Disgust and Shame or Social Pressure.
Disgust and Shame Based Safe Water and Handwashing Promotion
An estimated 2.2 million children under the age of 5 years die from diarrheal disease each year. Most of the burden of diarrheal disease is thought to be preventable with improvements in sanitation, water quality, and hygiene.
- Conditions
- Develop a New Group Version of the Becker-DeGroot-Marsckek (BDM) Auction to Measure Willingness to Pay of Compound Members for Shared Hardware., Develop a New Survey Instrument to Measure Behavioural Determinants of Hand Washing and Water Treatment Like Disgust and Shame or Social Pressure., Identify New Methods for Measuring Hand Washing and Water Treatment Behaviour., Compare the Effectiveness of the Disgust and Shame Based Interventions With Standard Public Health Interventions.
- Trial name
- NCT02037243
- Testing
- Standard Public Health Intervention with Water Treatment, Standard Public Health Intervention with Water Treatment & Hand Washing
- Where
- Bangladesh
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- International Centre for Diarrhoeal Disease Research, Bangladesh
Duchenne Muscular Dystrophy, Upper Extremity Problem
Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy
Purpose: This study aimed to evaluate the construct validity and reliability of the Turkish version of the Upper Limb Short Questionnaire (ULSQ) in Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05274555
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 41 people
- Sponsor
- Dokuz Eylul University
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the…
- Trial name
- NCT01772043
- Where
- 9 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy (DMD)
Can External Vagus Nerve Stimulation Reduce Systemic Levels of Inflammatory Mediators in Duchenne Muscular Dystrophy Patients?
The intended investigation is a pilot study to evaluate the safety and efficacy of a novel transcutaneous auricular vagus nerve stimulator system, termed TRAVAGUS ONE, to reduce systemic levels of inflammatory mediators in…
- Trial name
- Travagus One (NCT07543302)
- Testing
- Transcutaneous auricular vagus nerve stimulation
- Where
- Sweden
- Volunteers
- 20 people
- Sponsor
- taVNS AB
Duchenne Muscular Dystrophy
Dual Task in Duchenne Muscular Dystrophy
This study was planned to determine the effects of the dual-task performance of children with DMD with motor dysfunction and varying degrees of cognitive impairment compared to their healthy peers, to compare the dual-task…
- Trial name
- NCT05575648
- Testing
- 10 meter walk test
- Where
- Turkey (Türkiye)
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy (DMD)
DMD Voice: Qualitative Interviews With Patients and Caregivers
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.
- Trial name
- NCT06925269
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Fear of Falling, Duchenne Muscular Dystrophy (DMD)
Fear of Falling in Muscular Dystrophy
Primary objectives WP1: Evaluate the prevalence of FOF in the study population and how this varies over time. Evaluate whether there are relationships between the variables investigated (clinical, motor, cognitive, psychological)…
- Conditions
- Fear of Falling, Duchenne Muscular Dystrophy (DMD), Muscular Dystrophies, Rehabilitation
- Trial name
- NCT07129954
- Testing
- Motor rehabilitation, Motor Rehabilitation and Cognitive Behavioral Therapy
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Universita di Verona
Becker Muscular Dystrophy
Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients
This study is done to evaluate whether treatment with the drug sildenafil (Revatio®) can improve muscular, cardiac, cerebrovascular or cognitive function in patients with Beckers muscular dystrophy when compared to placebo…
- Trial name
- NCT01350154
- Testing
- Sildenafil
- Where
- Denmark
- Volunteers
- 17 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.