Clinical trials: Myopathies
Cerebral Palsy, Muscle
How Are the Muscles Affected in Cerebral Palsy? A Study of Muscle Biopsies Taken During Orthopaedic Surgery.
Cerebral palsy (CP) is a motor disorder caused by an injury to the immature brain. Even though the brain damage does not change, children with CP will have progressively weaker, shorter and stiffer muscles that will lead to…
- Conditions
- Cerebral Palsy, Muscle, Muscle Contracture, Muscle Spasticity
- Trial name
- CPTDBiopsy (NCT05506228)
- Testing
- No intervention
- Where
- Sweden
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Eva Ponten
Muscular Dystrophy, Duchenne
Characterization of DupEx2 Duchenne Muscular Dystrophy
To characterize the clinical phenotype and possible predictive/prognostic factors of patients with Duchenne muscular dystrophy (DMD) due to duplication of exon 2 (Dup2).
- Trial name
- DMDDup2 (NCT06337669)
- Where
- Italy
- Volunteers
- 26 people
- Sponsor
- IRCCS San Raffaele
Becker Muscular Dystrophy
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG).
- Trial name
- NCT01539772
- Where
- 16 sites in United States, Canada and 2 more
- Volunteers
- 85 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Neurologic Disorder, Duchenne
Physical Therapy Treatment on Children and Adolescents With Neurological Pathologies
To evaluate the positive effects of a home-based physical therapy intervention added to the usual physical therapy programs performed in children with neurological pathologies that induce sensorimotor impairments that affect…
- Conditions
- Neurologic Disorder, Duchenne, Pediatric Disorder, Sensorimotor Disorder Nos
- Trial name
- NCT05313295
- Testing
- Home-based physiotherapy, Usual Physiotherapy
- Where
- Spain
- Volunteers
- 30 people
- Sponsor
- Universidad de Almeria
Duchenne Muscular Dystrophy
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study
Dushenne muscular dystrophy (DMD), which is characterized by the deficiency of dystrophin protein, prevents the muscle from maintaining its normal activity, causing progressive damage to the heart, respiratory muscles and…
- Trial name
- NCT07172737
- Testing
- Respiratory Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Izmir Katip Celebi University
Systemic Lupus Erythematosus (SLE), Diffuse Systemic Sclerosis
YTS109 in Pediatric Relapsed/Refractory Autoimmune Diseases
This exploratory, single-arm, open-label study will evaluate the safety and preliminary efficacy of YTS109 cell therapy in pediatric patients with relapsed/refractory autoimmune diseases, including systemic lupus erythematosus…
- Conditions
- Systemic Lupus Erythematosus (SLE), Diffuse Systemic Sclerosis, Idiopathic Inflammatory Myopathies (IIMs), ANCA Associated Vasculitis (AAV)
- Trial name
- NCT07439029
- Testing
- YTS109 cell
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Fudan University
Myopathy, Neuropathy
Identification of Tongue Involvement in Late-Onset Pompe Disease
This purpose of this study is to determine if tongue strength and tongue ultrasound measurements differentiates patients with untreated late-onset Pompe Disease (LOPD) from patients with acquires/hereditary myopathies or…
- Conditions
- Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
- Trial name
- NCT02765828
- Testing
- Observational study
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Duke University
Choreatic Disorder
Heart and Skeletal Muscle Problems in Neuroacanthocytosis
The purpose of this study is to learn about heart and skeletal muscle problems related to neuroacanthocytosis (also called Mcleod's syndrome and Levine-Critchley disease).
- Trial name
- NCT00007228
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Cerebral Palsy
Individual Patient Expanded Access IND of Hope Biosciences Autologous Adipose-derived Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
This is an Individual Patient Expanded Access Protocol of Autologous HB-adMSCs for the Treatment of Cerebral Palsy (CP) with the primary goal of treating 1 individual with CP who has exhausted all treatment options, his condition…
- Trial name
- NCT04029896
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Spasticity, Post-Stroke
JCP Study of Investigation of Patient Background Characteristics
To collect the background characteristics of juvenile cerebral palsy patients aged 2-year or older prescribed BOTOX for the treatment of equines foot due to lower limb spasticity, compare them with those of patients whose deaths…
- Trial name
- NCT01332474
- Testing
- BOTOX
- Volunteers
- 326 people
- Sponsor
- GlaxoSmithKline
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Dystonic Cerebral Palsy
Intrathecal Baclofen and Pediatric Dystonia
The goal of this study is to better understand outcomes in children with dystonic cerebral palsy (CP) receiving intrathecal baclofen (ITB), ventral dorsal rhizotomy (VDR), or deep brain stimulation (DBS) as part of standard…
- Trial name
- PREDICT-ITB (NCT06606574)
- Testing
- ITB Titration Protocol, Ventral Dorsal Rhizotomy, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Baylor College of Medicine
Limb-Girdle Muscular Dystrophy, Type 2E
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Trial name
- NCT03652259
- Testing
- SRP-9003
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Temporomandibular Disorders
Effectiveness of a Counseling Program for Temporomandibular Disorders (TMD) Treatment
This study aims to evaluate the effectiveness of counseling on pain, function and well-fare outcomes for the management of patients with temporomandibular disorder (TMD).
- Trial name
- NCT02380105
- Testing
- Counseling, Interocclusal Appliances
- Volunteers
- 50 people
- Sponsor
- Universidade Federal do Rio Grande do Norte
Spina Bifida, Neuromuscular Diseases
Metformin in Children With Motor Deficit
Obesity with insulin resistance in the paediatric population provides an increasing challenge. Children with neurological or neuromuscular diseases are even more prone to obesity: their locomotor impairment leads to an…
- Trial name
- NCT00720161
- Testing
- Metformin
- Where
- Belgium
- Volunteers
- 42 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Cerebral Palsy, Spasticity
Power Wheelchair Joystick Use in Spastic Cerebral Palsy
There are over 750,000 individuals in the U.S. with Cerebral Palsy (CP). Up to 46% of adults with CP report limited mobility in their communities.
- Trial name
- NCT00337688
- Testing
- isometric joystick
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Back Pain, Low Back Pain
Low Back and Neck Pain in Nursing Students at The University of The West Indies
The purpose of this study is to identify the prevalence of neck and low back pain in nursing students enrolled at the University of the West Indies, Mona; to assess their knowledge, attitude and practice of good spine health and…
- Conditions
- Back Pain, Low Back Pain, Musculoskeletal Pain, Neck Pain
- Trial name
- NCT07167888
- Testing
- Clinical Control Group (Group A), Spine Exercise Program (Group B)
- Where
- Jamaica
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- The University of The West Indies
Hemiparesis, Paraparesis
Immediate Effects On Gait and Spasticity After The Use Of Passive Kinesiotherapy Equipment In The Lower Limbs Of Neurological Patients
Undergo assessment of muscle tone (Ashworth Scale), gait (BAIOBIT) and risk of falling (Timed Up and Go) before using the device; Use the device on the spastic lower limb(s) for 30 minutes; Go through the same assessment carried…
- Conditions
- Hemiparesis, Paraparesis, Tetraparesis, Cerebral Palsy
- Trial name
- NCT06696040
- Testing
- Passive mobilization using a device
- Where
- Brazil
- Volunteers
- 80 people
- Sponsor
- Fernanda Trubian
Centronuclear Myopathy
Multispectral Optoacoustic Tomography for Advanced Imaging of Centronuclear Myopathy
Twenty patients with centronuclear myopathy and twenty age- and sex-matched, muscle-healthy controls will undergo diagnostic examination.
- Trial name
- MOSAIC (NCT07021820)
- Testing
- MSOT
- Where
- Germany
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Muscular Dystrophies, Becker Muscular Dystrophy
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child.
- Conditions
- Muscular Dystrophies, Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
- Trial name
- biodystromirs (NCT02109692)
- Testing
- blood sample
- Where
- France
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
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