Clinical trials: DMD
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
Becker Muscular Dystrophy
Sodium Nitrate to Improve Blood Flow
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD).
- Trial name
- NCT02847975
- Testing
- Sodium nitrate
- Where
- 2 sites in United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
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
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 Distrophy (DMD)
Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common and devastating form of muscular dystrophy, caused by an X-chromosome gene mutation resulting in the absence of the protein dystrophin.
- Trial name
- NCT01823783
- Testing
- Muscle biopsy
- Where
- 7 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Becker Muscular Dystrophy, McArdle Disease
Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases
The purpose of the study is to explore the biomarker Fast Troponins response to exercise in patients with Becker muscular dystrophy, Limb-girdle muscular dystrophy and McArdle disease
- Conditions
- Becker Muscular Dystrophy, McArdle Disease, Limb-Girdle Muscular Dystrophy Type 2
- Trial name
- NCT04349566
- Where
- Denmark
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Mads Peter Godtfeldt Stemmerik
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
Executive Dysfunction, Occupational Problems
Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy
The effects of Duchenne muscular dystrophy and its treatments on executive functions and occupational performance are under-mentioned in the literature.
- Conditions
- Executive Dysfunction, Occupational Problems, Duchenne Muscular Dystrophy
- Trial name
- NCT06868784
- Testing
- Evaluations
- Where
- Turkey (Türkiye)
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
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 (DMD)
To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
This study will evaluate the pharmacokinetics plasma profile of 3 treatments: ISO 20, IBU 200 and IBU and ISO combinations (200 +20) given in single dose.
- Trial name
- ISOFEN1 (NCT01478022)
- Testing
- Isosorbide Dinitrate, Ibuprofen, Ibuprofen and Isosorbide Dinitrate combination
- Where
- Italy
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Parent Project, Italy
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
Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency)
Evaluation of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand the biochemistry of different types of Limb-Girdle Muscular Dystrophy (LGMD) and to determine appropriate outcome measures for future clinical treatment trials for LGMD.
- Conditions
- Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency), Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency), Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)
- Trial name
- NCT00893334
- Where
- 2 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
The Burden of Access in Duchenne Muscular Dystrophy in the US
This study is being conducted to determine if DMD patients / families and healthcare providers experience burdens related to access, and if so, to identify them, and to determine life impacts to the patient, if any, of these…
- Trial name
- NCT03951675
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- University of Florida
Muscular Dystrophy
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise.
- Trial name
- NCT02653833
- Testing
- Tadalafil 20 MG, beetroot juice extract
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Cedars-Sinai Medical Center
DMD, BMD
Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
Study aims to develop and to evaluate the neurophysiological and physiological response to a classical conditioning task.To better understand how Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) impacts…
- Trial name
- D-BRAIN (NCT06581887)
- Testing
- Classical conditioning task
- Where
- United Kingdom
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University College, London
Duchenne Muscular Dystrophy
Telerehabilitation in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive inherited disease that affects the muscles and causes functional limitations to varying degrees. It is vital to start physiotherapy follow-ups immediately after diagnosis.
- Trial name
- Duchenne (NCT05305976)
- Testing
- Telerehabilitation
- Where
- Turkey (Türkiye)
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Karadeniz Technical University
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
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
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