Clinical trials: Duchenne Muscular Dystrophy (DMD)
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Wearable Technology to Assess Gait Function in SMA and DMD
The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment.
- Trial name
- NCT04193085
- Testing
- Observational
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Columbia 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
Stress
Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy
Background: Children with Duchenne/Becker Muscular Dystrophy (DBMD) slowly lose muscle function. They usually die at a young age. Some mothers adapt to the demands of caring for a child with this disease better than others.
- Trial name
- NCT02194725
- Where
- United States
- Volunteers
- 228 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Duchenne Muscular Dystrophy (DMD)
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
To perform the reliability and validity study of the Peabody Developmental Motor Scale (Peabody Developmental Motor Scales-2) in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT05990608
- Testing
- Peabody Developmental Motor Scales-2, PDMS
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
Virtual Reality in Individuals With Duchenne Muscular Dystrophy
With the growing accessibility of computer-assisted technology, one option for rehabilitation programs for individuals with Duchenne muscular dystrophy (DMD) is the use of virtual reality environments to enhance motor practice.
- Trial name
- NCT03002298
- Testing
- DMD gesture task, DMD button-press task, Control group gesture task
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
Duchenne Muscular Dystrophy
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy…
- Trial name
- ValPedsQLDMD (NCT03513367)
- Testing
- Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale, The following data of motor function
- Where
- France
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- University Hospital, Toulouse
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Becker Muscular Dystrophy
Tadalafil in Becker Muscular Dystrophy
Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with…
- Trial name
- NCT01070511
- Testing
- Tadalafil
- Where
- 2 sites in United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Duchenne Muscular Dystrophy
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
The task consists in reach as much bubbles as they can, the bubbles appear on the screen of the computer and should be reached in 10 seconds.
- Trial name
- NCT02891434
- Testing
- Acquisition on TouchScreen, Acquisition on Kinect, Acquisition on LeapMotion
- Where
- Brazil
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Muscular Dystrophy, Duchenne
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in…
- Trial name
- NCT02834650
- Testing
- Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test
- Where
- 3 sites in United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Stanford University
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2I
Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I
The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I.
- Trial name
- NCT02165358
- Where
- Denmark
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
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
PDE Inhibitors in DMD Study (Acute Dosing Study)
PDE5A inhibition, which boosts NO-cGMP signaling, will relieve functional muscle ischemia and restore normal blood flow regulation (i.e., functional sympatholysis) during exercise in boys with DMD.
- Trial name
- NCT01580501
- Testing
- Tadalafil and Sildenafil
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy
UTSW HP [13-C] Pyruvate Injection in HCM
The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal…
- Conditions
- Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis
- Trial name
- HPHCM (NCT03057002)
- Testing
- Hyperpolarized 13C-Pyruvate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Duchenne Muscular Dystrophy
A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
The purpose of this protocol is to perform Electrical Impedance Myography (EIM) testing on healthy children and children with duchenne muscular dystrophy so as to develop a new, convenient tool for the office based assessment of…
- Trial name
- NCT02340923
- Where
- 5 sites in United States
- Volunteers
- 327 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Duchenne Muscular Dystrophy
Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy
To study the cognitive function \& psychiatric aspects of patients with DMD by specific psychometry tests \& its relation to quality of life of the patients To evaluate brain structural changes using MRI and correlate them to the…
- Trial name
- NCT06732011
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Assiut University