Clinical trials: Muscular Dystrophy
Other Diseases or Conditions
Mother-caregivers of Children With Duchenne Muscular Dystrophy
The incidence of Duchenne Muscular Dystrophy (DMD) is approximately 1 in 3.500 male newborns. During its progression there is loss of mobility, swallowing difficulties and a significant reduction in respiratory capacity.
- Trial name
- DMDK (NCT01921374)
- Testing
- sleep parameters, Hormonal profile, Inflammatory profile
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Monica Levy Andersen
Muscular Dystrophies
Wearability, Saefty and Usability Assessment for the Upper Limb Exoskeleton BRIDGE/EMPATIA
The study is a feasibility study or pilot study, that is a clinical investigation to acquire the preliminary information on a motorized exoskeleton (BRIDGE / EMPATIA exoskeleton) for the movement of the upper limb in order to…
- Trial name
- NCT03951844
- Testing
- BRIDGE/EMPATIA exoskeleton
- Where
- 2 sites in Italy
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Alessandra Pedrocchi
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.
Shoulder Pain, Neuromuscular Diseases
Understanding Control and Mechanisms of Shoulder Instability in FSHD
The aim of this study is to identify factors for shoulder instability in people with Facioscapulohumeral dystrophy (FSHD). FSHD is a non-life limiting condition with symptoms presenting in the second decade of life (Evangelista…
- Conditions
- Shoulder Pain, Neuromuscular Diseases, Shoulder Injuries, Facio-Scapulo-Humeral Dystrophy
- Trial name
- NCT05239520
- Testing
- 3D movement analysis with surface electromyography and ultrasound
- Where
- United Kingdom
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- University of Liverpool
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders
The investigators laboratory has been studying families with a history of ALS for more than 30 years and is continuing to use new ways to understand how genes may play a role in ALS, motor neuron disease and other neuromuscular…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease
- Trial name
- NCT01459302
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
Becker Muscular Dystrophy
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Monitoring patients with neuromuscular disorders is crucial but doesn't always allow for a sufficiently specific approach to all aspects of the condition.
- Trial name
- MyoBrain-BMD (NCT07740057)
- Where
- France
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Spinal Muscular Atrophy, Merosin Deficient Congenital Muscular Dystrophy
Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass
In a study from 2003 the investigators showed that adult patients with very low skeletal muscle mass (spinal muscular atrophy (SMA) type II, Duchenne muscular dystrophy, congenital muscular dystrophy) are prone to develop…
- Trial name
- NCT03970135
- Testing
- Fasting
- Where
- Denmark
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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 (DMD)
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
- Trial name
- IBISD (NCT01380964)
- Where
- France
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Genethon
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, Burden, Dependency
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
The purpose of this study is to improve the understanding of the treatment goals that a person with Duchenne Muscular Dystrophy (DMD) or the caregiver may be most interested in, based on the severity of the person's disease.
- Conditions
- Duchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management
- Trial name
- NCT03680365
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Jett Foundation, Inc.
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
Muscular Dystrophies
A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling
This is a Phase I, open-label, single-dose, 1-period study to compare EDG-5506 concentrations measured from venous and capillary blood sampling in healthy adult subjects.
- Trial name
- NCT05492734
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
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
The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy
Children with Duchenne Muscular Dystrophy (DMD) have difficulties towards the end of the ambulatory period, especially in activities that require lower extremity proximal muscle strength such as walking, climbing stairs, standing…
- Trial name
- NCT04287582
- Testing
- Electromyographic device
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Myotonic Dystrophy Type 1 (DM1)
A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients.
- Trial name
- NCT06979024
- Testing
- Triplet-primed PCR or Long-read sequencing
- Where
- China
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
Various Forms of Skeletal Muscle Dystrophy
Interaction of Muscle Dystrophy, Glucose Metabolism and Insulin Sensitivity
The aim of this project to characterize systemic and organ-specific (subcutaneous adipose tissue and skeletal muscle) metabolic responses at rest and during standardized, moderate exercise after an oral glucose load in patients…
- Trial name
- MyoMet (NCT05052073)
- Testing
- oral glucose load at rest, oral glucose load and exercise
- Where
- Germany
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Charite University, Berlin, Germany
Muscular Dystrophies, Muscular Dystrophy
Muscle Tissue Bank for Muscular Dystrophy
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
- Trial name
- NCT01950897
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Myotonic Dystrophy 1, Muscular Dystrophies
Myotonic Dystrophy Type 1 Aerobic Exercise Study
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss.
- Trial name
- DM1ex (NCT04187482)
- Testing
- Exercise training
- Where
- Canada
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- McMaster University
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege