Clinical trials: Muscle Disorders
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
Abdominal Obesity, Physical Activity
Physical Activity in Overweight Girls: Implications for Reversing Risk Factors for Type 2 Diabetes
In this randomized controlled trial, we will examine the effect of a 3-month exercise training (aerobic exercise versus resistance exercise) without calorie restriction on total and regional adiposity, ectopic fat in the liver…
- Trial name
- NCT01323088
- Testing
- Control, Resistance Exercise, Aerobic exercise
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Sarcopenia, COPD
Evaluate the Effect of Dietary Supplement Combined Exercise Among Patients With Sarcopenia Comorbid With Lung Disease
This trial is a prospective multi-center study. The purpose of this study is to evaluate the effect of dietary supplement combined with supervised exercised on the physical performance, body composition and lung function among…
- Conditions
- Sarcopenia, COPD, ILD
- Trial name
- NCT05926713
- Testing
- Sarcojoint®, supervised exercise
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- Orient Europharma Co., Ltd.
SMARD1, CMT2S
Gene Therapy for IGHMBP2-Related Diseases
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.
- Trial name
- NCT05152823
- Testing
- Gene Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Megan Waldrop
Temporomandibular Disorders (TMD)
Digitally Trained Applied Relaxation for Treatment of Temporomandibular Pain
Temporomandibular disorders (TMD) are common musculoskeletal pain conditions related to masticatory muscles and temporomandibular joints.
- Trial name
- RCT (NCT07129096)
- Testing
- Applied Relaxation, Occlusal splint
- Where
- Finland
- Volunteers
- 124 people
- Sponsor
- University of Oulu
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.
Cerebral Palsy, Spastic
The Effects of Balance and Respiratory Muscle Training on Functional Capacity, Balance, Respiratory Function and Respiratory Muscle Strength in Children With Cerebral Palsy
Cerebral palsy (CP) is a permanent, non-progressive disorder in which the immature brain is affected by various causes in the prenatal, perinatal or postnatal period.
- Trial name
- NCT03396289
- Testing
- Conventional physiotherapy including balance exercises, Inspiratory muscle training
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Bezmialem Vakif University
Spinal Muscular Atrophy (SMA)
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it.
- Trial name
- Sparkle (NCT07287982)
- Testing
- ARGX-119 IV
- Where
- 28 sites in United States, Australia and 2 more
- Volunteers
- 60 people
- Sponsor
- argenx
Cerebral Palsy, Neurologic Disorder
Neural Mobilization and Cerebral Palsy
Objective: To assess the effect of neural mobilization applied to the lower limbs, compared to conventional physical therapy, in children diagnosed with Cerebral Palsy.
- Trial name
- NEUROREHAB (NCT06485830)
- Testing
- Neural mobilization of the lower limb, Conventional physical therapy
- Where
- 2 sites in Spain
- Volunteers
- 40 people
- Sponsor
- University of Seville
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Muscular Dystrophy, Duchenne Type
Trunk Oriented Exercises Versus Whole-body Vibration for Duchenne Muscular Dystrophy
The abdominal muscles play an important role in stabilizing the trunk and providing postural stability. Children with Duchene muscular dystrophy have weak muscles, which may impair postural adjustments.
- Trial name
- NCT05688072
- Testing
- trunk oriented exercise, whole body vibration
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized…
- Trial name
- FOX (NCT06100887)
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 7 sites in United States
- Volunteers
- 43 people
- Sponsor
- Edgewise Therapeutics, Inc.
Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study
This cohort study (participants with CMT and control participants) has two parts (Part 1: CMT1A cohort; Part2: CMT1B, CMT2A and CMTX1 cohort) and is proposed to take place over 3 years across three sites.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease, Type X
- Trial name
- NCT03550300
- Where
- United Kingdom
- Volunteers
- 130 people
- Sponsor
- University College, London
Nemaline Myopathy, Myopathies
Natural History Study for Patients With Nemaline Myopathy in Spain
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of nemaline myopathies.
- Conditions
- Nemaline Myopathy, Myopathies, Myopathic Conditions, NEB
- Trial name
- NCT07488806
- Testing
- Muscle Ultrasound, Motor function scales, Complete physical examination
- Where
- Spain
- Volunteers
- 100 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Duchenne Muscular Dystrophy
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy
The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT07673809
- Testing
- GNR-097
- Where
- 6 sites in Belarus, Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
MYOPATHY
Stem Cell Transplantation in Idiopathic Inflammatory Myopathy Diseases
Myositis is a disease, believed to be due to immune cells, cells which normally protect the body, but are now attacking the muscles and other organ systems within body.
- Trial name
- NCT00278564
- Testing
- Hematopoietic stem cell transplantation, Cyclophosphamide, Mesna
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Northwestern University
Duchenne Muscular Dystrophy
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
This is a Phase 3, multi-center, open-label extension study in ambulant boys with DMD who have completed the 48-week treatment period of either viltolarsen or placebo in Study NS-065/NCNP-01-301.
- Trial name
- NCT04768062
- Testing
- Viltolarsen
- Where
- 31 sites in Australia, Canada and 15 more
- Volunteers
- 74 people
- Sponsor
- NS Pharma, Inc.
Abdominoplasty, Diaphragm
Gastroesophageal Reflux and Respiratory Function in Patients Undergoing Abdominoplasty
The objective of this observational study is to compare parameters of diaphragmatic mechanics, respiratory muscle function, and thoracoabdominal kinematics among women with gastroesophageal reflux disease (GERD) and a history of…
- Conditions
- Abdominoplasty, Diaphragm, Respiratory Mechanics, Gastroesophageal Reflux Disease
- Trial name
- NCT07868562
- Testing
- Assessment of anthropometric and sociodemographic data and clinical history, Assessment of Symptoms, Quality of Life, and Physical Activity, Spirometric assessment
- Where
- Brazil
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Universidade Federal de Pernambuco
Muscular Atrophy, Spinal
Natural History of SMA
This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease…
- Trial name
- iSMAR (NCT05755451)
- Where
- 5 sites in Italy
- Volunteers
- 1,200 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Spasticity, Cerebral Palsy
Oral Baclofen Pharmacokinetics and Pharmacodynamics in Children With Spasticity
Oral baclofen is used commonly to treat spasticity in children with cerebral palsy. Although for adults there is dosing,safety and efficacy information in the package insert, this is not the case for children.
- Trial name
- Best PK/PD (NCT00607542)
- Testing
- baclofen
- Where
- 11 sites in United States
- Volunteers
- 61 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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