Clinical trials: Muscle Disorder
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
Spinal Muscular Atrophy (SMA)
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which…
- Trial name
- MANATEE (NCT05115110)
- Testing
- RO7204239, Risdiplam
- Where
- 29 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal, Child, Only
Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy
Background and Aim: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive symmetrical weakness and atrophy of proximal muscles causing from degeneration of anterior horn cells of spinal cord.
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Trial name
- NCT05291962
- Testing
- Retrospective observational study
- Where
- Turkey (Türkiye)
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Equinus Deformity
Dynamic Evaluation of Ankle Joint and Muscle Mechanics in Children With Spastic Equinus Deformity Due to Cerebral Palsy
This research will lead to the first evaluation of intrinsic and dynamic joint and muscle mechanics of equinus in cerebral palsy. It would provide a direct cause and effect relationship between equinus and bone deformity.
- Trial name
- EQUINUS (NCT02814786)
- Testing
- MRI scanner, Gait analysis
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
The primary objective of this study is to evaluate the safety of SRP-9004.
- Trial name
- DISCOVERY (NCT06747273)
- Testing
- SRP-9004
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Rhabdomyosarcoma
Treatment of Newly Diagnosed Rhabdomyosarcoma Using Molecular Risk Stratification and Liposomal Irinotecan Based Therapy in Children With Intermediate and High Risk Disease
This is a phase II study to determine safety and efficacy of combining liposomal irinotecan with vincristine alternating with VAC in intermediate-risk patients, liposomal irinotecan with temozolomide and vincristine alternating…
- Trial name
- NCT06023641
- Testing
- Vincristine, Dactinomycin, Cyclophosphamide
- Where
- 3 sites in United States
- Volunteers
- 135 people
- Sponsor
- St. Jude Children's Research Hospital
Spinal Amyotrophy, Infantile Spinal Muscular Atrophy
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord, due to the absence of the SMN1 gene and the resulting lack of SMN…
- Conditions
- Spinal Amyotrophy, Infantile Spinal Muscular Atrophy, Juvenile Spinal Muscular Atrophy
- Trial name
- FANTASI-SMART (NCT06562283)
- Testing
- Thumb test, Grip test, Quadriceps Intermittent Fatigue test (QIF test))
- Where
- 4 sites in France
- Volunteers
- 80 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of Rituxan in Duchenne Muscular Dystrophy
1. Study population:It is applicable to male participants with genetically confirmed and clinically confirmed Duchenne muscular dystrophy (DMD), aged between 6 and 10 years. 2.
- Trial name
- NCT07475754
- Testing
- Rituxan treatment
- Volunteers
- 5 people
- Sponsor
- Peking Union Medical College Hospital
Primary Nocturnal Enuresis, Nocturnal Enuresis in Children
Effectiveness of Pelvic Floor Muscle Rehabilitation Combined With Desmopressin in Children With Primary Monosymptomatic Nocturnal Enuresis
The aim of this randomized controlled trial is to evaluate the effects of Structured Pelvic Floor Muscle Rehabilitation (SPFMR) in children with Primary Monosymptomatic Nocturnal Enuresis (PMNE) who are undergoing desmopressin…
- Trial name
- NCT06883851
- Testing
- Structured pelvic floor rehabilitation
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Bahçeşehir University
Inflammation In Skeletal Muscle
Natural History of Inflammatory Muscle Diseases
Objective: To collect information and biospecimens (such as blood, muscle, and skin samples) that will be used to research causes and treatments of inflammatory muscle diseases.
- Trial name
- NCT05738824
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Reactive Airways Dysfunction Syndrome (Disorder), Muscle Weakness
Efficacy of Inspiratory Muscle Training on Elite Swimmers
This study is a randomized controlled trial on the efficacy of inspiratory muscle training on swimming performance, airway dysfunction and perceived breathlessness in elite swimmers.
- Conditions
- Reactive Airways Dysfunction Syndrome (Disorder), Muscle Weakness, Dyspnea
- Trial name
- PEAK (NCT03062735)
- Testing
- Inspiratory Muscle Training
- Where
- Portugal
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Universidade do Porto
Neuromuscular Disease
Antioxidant Therapy in RYR1-Related Congenital Myopathy
Background: Ryanodine receptor type 1-related myopathies (RYR1-RM) are the most common non-dystrophic muscle diseases that people are born with in the U.S. They affect development, muscles, and walking.
- Trial name
- RYR1 (NCT02362425)
- Testing
- N-acetylcysteine
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- National Institute of Nursing Research (NINR)
Tumors, Brain Tumors
Everolimus for Treating Pediatric Patients With Recurrent or Refractory Tumors
Patients with recurrent or refractory solid tumors or brain tumors that are unresponsive to conventional therapy, or with no known effective therapy, will be treated.
- Conditions
- Tumors, Brain Tumors, Rhabdomyosarcoma, Sarcoma, Soft Tissue
- Trial name
- NCT00187174
- Testing
- Everolimus
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- St. Jude Children's Research Hospital
Diarrhea, Dehydration
Clinical Evaluation of Ultrasonic Hydration Monitor for Infants
Study will verify ability of infant Hydration Monitor (IHM) to assess changes of hydration status in newborns and infants by means of ultrasound velocity measurements through muscle tissues.
- Trial name
- IHM-01 (NCT01482208)
- Where
- Latvia
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Artann Laboratories
Prader-Willi Syndrome
Mitochondrial Complex I Dysfunction in PWS
Prader-Willi Syndrome (PWS) is characterized by profound infantile hypotonia, growth delay, cognitive impairment, muscle weakness and exercise intolerance.
- Trial name
- NCT03831425
- Testing
- Coenzyme Q10
- Where
- Canada
- Sponsor
- The Hospital for Sick Children
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Hypermetabolism Due to Defect in Mitochondria, Luft Disease
A Single Case Study of Hypermetabolism
In the Metabolic Disorder clinic at The Hospital for Sick Children, the investigators identified a male, proband with possible Luft's disease.
- Trial name
- NCT04805268
- Testing
- 18F FDG PET/CT
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
Duchenne Muscular Dystrophy
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT00428935
- Testing
- rAAV2.5-CMV-minidystrophin (d3990)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Nationwide Children's Hospital
Neuromuscular Diseases
Measuring Physical Activity With ActivPal
This study measures the level of physical activity in participants with neuromuscular disorders. The patient wears a small button like meter on their leg for a period of 1 week every 6 months over a period of 3 years.
- Trial name
- Activpal (NCT06084026)
- Testing
- ActivPal
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Columbia University
Temporomandibular Joint Disorders
Treatment of Temporomandibular Disorders
The aim of the present study was to carry out a pilot project comparing the effectiveness of two therapeutic approaches to temporomandibular disorders (TMDs) in adolescents.
- Trial name
- NCT01187316
- Testing
- Transcutaneous electrical nerve stimulation, Physiotherapy
- Where
- 2 sites in Brazil
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Pernambuco