Clinical trials: Congenital Myopathy
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
Facioscapulohumeral Muscular Dystrophy
ADVANCED FSHD-COM: New Clinical Outcome Measures to Evaluate Non-ambulant FSHD Patients, a Pilot Study
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common adult muscular dystrophy with an estimated prevalence range of 2-7 per 100,000.
- Trial name
- ADVANCED (NCT05453461)
- Testing
- Validation of new COMs for non ambulant FSHD patients
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Cardiomyopathy, Arrhythmia
Genetics of Cardiovascular and Neuromuscular Disease
We are studying the genetics of human cardiovascular and neuromuscular disease. There are many different genetic regions that have been associated with the development of cardiomyopathy.
- Conditions
- Cardiomyopathy, Arrhythmia, Muscular Dystrophy
- Trial name
- NCT00138931
- Testing
- Blood draw (genetic testing)
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- University of Chicago
Duchenne Muscular Dystrophy, Gait Disorders in Children
Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy
The aim of this study was to develop a gait assessment instrument for Duchenne Muscular Dystrophy patients (DMD-GAS), and investigate its validity and reliability.The scale was developed considering the expert opinions which…
- Conditions
- Duchenne Muscular Dystrophy, Gait Disorders in Children, Gait Disorders, Neurologic
- Trial name
- NCT05244395
- Testing
- Duchenne Muscular Dystrophy Gait Assessment Scale
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Hacettepe University
Rheumatic Diseases, Adult Onset Still Disease
Rheumatology Patient Registry and Biorepository
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
- Conditions
- Rheumatic Diseases, Adult Onset Still Disease, Ankylosing Spondylitis, Psoriatic Arthritis
- Trial name
- NCT04402086
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Yale University
Duchenne Muscular Dystrophy (DMD)
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Background and Rationale Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the dystrophin gene, resulting in progressive muscle weakness and multiple complications, including respiratory and cardiac…
- Trial name
- GUP23005 (NCT07890883)
- Where
- 15 sites in Italy
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Istituto Giannina Gaslini
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Myotonia Congenita, Paramyotonia Congenita
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1.
- Conditions
- Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
- Trial name
- NCT02251457
- Testing
- Ranolazine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Ohio State University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
This is an investigation of the efficacy and safety of CRD007 in Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers.
- Trial name
- NCT01540604
- Testing
- CRD007
- Where
- Sweden
- Sponsor
- RSPR Pharma AB
Arthritis, Rheumatoid Arthritis
Delivery of Enhance Fitness Trial
Arthritis is a common condition in the United States, and a leading cause of pain and disability. Physical exercise is recommended for managing arthritis, but access to evidence-based exercise programs is limited, particularly in…
- Conditions
- Arthritis, Rheumatoid Arthritis, Fibromyalgia, Gout
- Trial name
- DEFT (NCT05275348)
- Testing
- Tele-Enhance Fitness, In-person Enhance Fitness
- Where
- United States
- Volunteers
- 310 people
- Sponsor
- University of Washington
Limb Girdle Muscular Dystrophy
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy.
- Trial name
- NCT02050776
- Testing
- Autologous bone marrow mononuclear cell transplantation
- Where
- India
- Sponsor
- Neurogen Brain and Spine Institute
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Muscular Dystrophy, Duchenne
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD.
- Trial name
- PolarisDMD (NCT03703882)
- Testing
- Edasalonexent
- Where
- 40 sites in United States, Australia and 6 more
- Volunteers
- 131 people
- Sponsor
- Catabasis Pharmaceuticals
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
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients that will involve direct intramuscular injection to the extensor digitorum brevis muscle (EDB).
- Trial name
- NCT02704325
- Testing
- rAAVrh74.MCK.GALGT2
- Where
- United States
- Sponsor
- Kevin Flanigan
Duchenne Muscular Dystrophy
Open-label Extension of the HOPE-2 Trial
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up.
- Trial name
- HOPE-2-OLE (NCT04428476)
- Testing
- Deramiocel (CAP-1002)
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Capricor Inc.
Steinert's Disease
Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would…
- Trial name
- NCT02930408
- Testing
- electrocardiogram, Echocardiography
- Volunteers
- 70 people
- Sponsor
- Poitiers University Hospital
Duchenne Muscular Dystrophy
Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, arises from mutations in the dystrophin gene. Antisense therapy with the use of antisense oligonucleotides (AON) has the potential to restore effectively…
- Trial name
- NCT00159250
- Testing
- AVI-4658 (PMO)
- Where
- United Kingdom
- Volunteers
- 7 people
- Sponsor
- Imperial College London
Chronic Pain, Chronic Pain Syndrome
Outcomes Mandate National Integration With Cannabis as Medicine
This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.
- Conditions
- Chronic Pain, Chronic Pain Syndrome, Chronic Pain Due to Injury, Chronic Pain Due to Trauma
- Trial name
- OMNI-Can (NCT03944447)
- Testing
- Cannabis, Medical, RYAH-Medtech Inhaler
- Where
- 17 sites in United States
- Volunteers
- 200,000 people, healthy volunteers welcome
- Sponsor
- OMNI Medical Services, LLC
Venous Thromboembolism, Pulmonary Embolism
Venous Thromboembolism in DM1
The risk for venous thromboembolism (VTE) in DM1 and in other inherited myopathies, which can lead to chronic immobilization, are unknown.
- Conditions
- Venous Thromboembolism, Pulmonary Embolism, Deep Vein Thrombosis, Myopathy
- Trial name
- DM1-VTE (NCT03141749)
- Volunteers
- 2,810 people
- Sponsor
- Institut de Myologie, France
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