Clinical trials: Muscle Disorders
Duchenne Muscular Dystrophy
Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed DS5141-A-J101.
- Trial name
- NCT04433234
- Testing
- DS-5141b
- Where
- 2 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Daiichi Sankyo Co., Ltd.
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Malocclusion
An Electromyography Study Comparing the Impact of Using RMEs on Muscle Activity Among Growing Orthodontic Patients
Compare the impact of using rapid maxillary expanders on masticatory muscle activities (Masseter and Anterior Temporalis muscles) among different posterior cross-bite patient (unilateral, bilateral) as well as compare the…
- Trial name
- NCT02752347
- Testing
- Surface EMG device
- Where
- Saudi Arabia
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Riyadh Colleges of Dentistry and Pharmacy
Duchenne Muscular Dystrophy
Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration.
- Trial name
- NCT02484560
- Testing
- Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell
- Where
- Turkey (Türkiye)
- Volunteers
- 10 people
- Sponsor
- University of Gaziantep
Muscular Dystrophy, Duchenne
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in…
- Trial name
- NCT02834650
- Testing
- Cardiac MRI with contrast, Cardiac MRI without contrast, Blood Test
- Where
- 3 sites in United States
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Stanford University
Sarcopenia
Sarcopenia and Aging
Objective: Evaluate the muscular strength in the ageing process and identify the changes between the abdominal muscles, upper and louver edges.
- Trial name
- NCT00907010
- Volunteers
- 48 people
- Sponsor
- Universidade de Franca
Sarcopenia, Cirrhosis, Liver
Effect of Branch Chain Amino Acid Therapy on Sarcopenia in Children With Chronic Liver Disease.
Sarcopenia is now a well-known complication of cirrhosis and various studies, including pediatric studies, have recognized it as a poor prognostic factor.
- Trial name
- NCT05093218
- Testing
- branch chain amino acid
- Where
- India
- Volunteers
- 44 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
MELAS Syndrome, Ketogenic Dieting
Effectiveness of Ketogenic Diet in MELAS Syndrome
The goal of this clinical trial is to evaluate the effectiveness of ketogenic diet in patients with MELAS syndrome. The main questions it aims to answer are: Clarify the curative effects of ketogenic diet in the treatment of…
- Trial name
- NCT06013397
- Testing
- Ketogenic diet
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Cystic Fibrosis
Sit-to-stand Test in Cystic Fibrosis Children and Adolescents
Cystic fibrosis (CF) is a chronic hereditary respiratory disease. Exercise testing is part of CF patients regular assessment. Cardio-Pulmonary Exercise Testing (CPET) is currently considered as the gold standard to assess…
- Trial name
- NCT03069625
- Testing
- Sit-to-stand test, 6-Minute Walk Test
- Where
- 3 sites in France
- Volunteers
- 36 people
- Sponsor
- Groupe Hospitalier du Havre
Polymyositis
Epidemiological Study of Inflammatory Myopathies in a French Region
Knowledge of the descriptive epidemiology of MIs is very limited. The main objective of this study is to determine the prevalence of inflammatory myopathies in Alsace at the 1st January 2012 with "capture-recapture" design using…
- Trial name
- PREMIA (NCT02881450)
- Volunteers
- 117 people
- Sponsor
- University Hospital, Strasbourg, France
Diplegic Spastic Cerebral Palsy
Comparing the Instrument-Assisted Soft Tissue Mobilization and Neurodevelopmental Therapy for Gross Motor Functions, Muscle Tone and Spasticity in Children with Spastic Diplegic Cerebral Palsy (GMFCS Levels I and II)
Cerebral palsy is a neurological condition that affects individuals worldwide, with a global burden of 0.9%. However, prevalence rates show regional variation, with high-income countries reporting rates of 0.2% and low-income…
- Trial name
- IASTM;NDT;CP (NCT06702709)
- Testing
- Instrument Assisted Soft Tissue Mobilization, Neurodevelopmental Therapy
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- Ziauddin University
Parkinson Disease
Deep Brain Stimulation in Parkinson's Disease: Motor and Functional Outcomes
Introduction: Parkinson's Disease (PD) is a progressive neurodegenerative condition characterized by classic motor symptoms, such as bradykinesia, rigidity, resting tremor, and postural instability, which directly compromise the…
- Trial name
- NCT07567599
- Testing
- Deep Brain Stimulation
- Where
- Brazil
- Volunteers
- 40 people
- Sponsor
- Federal University of Minas Gerais
Mild Ptosis, Moderate Ptosis
A Newly Modified Technique for Levator Muscle Tucking in Blepharoptosis Surgery: An Egyptian Tertiary Center Study
Mild to moderate blepharoptosis with good levator function is usually corrected by levator muscle resection or advancement with their modifications with high success rate.
- Trial name
- Lidtucking (NCT04883853)
- Testing
- Mild _moderate Ptosis tucking
- Where
- Egypt
- Volunteers
- 180 people
- Sponsor
- Ain Shams University
X-Linked Myotubular Myopathy
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Trial name
- NCT07052929
- Testing
- ASP2957, Methylprednisolone, Prednisolone
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Astellas Gene Therapies
Primary Disease Fascioscapulohumeral Dystrophy (FSHD)
Routine Health Care of Patients With FSHD
On the basis of published data and our results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, investigators conducted a pilot randomized double-blind placebo…
- Trial name
- FSHD (NCT02622438)
- Testing
- Followed with an initial assessment, 12 months and followed annually corresponding to the usual follow-up of the patient.
- Where
- France
- Volunteers
- 189 people
- Sponsor
- University Hospital, Montpellier
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
Inspiratory Muscle Training
The Effect of Inspiratory Muscle Training on Respiratory Function, Balance and Functional Capacity in Trainable Mentally Handicapped Children
Background: The aim of this study was to examine the effect of inspiratory muscle training on respiratory functions, balance and functional capacity in trainable mentally disabled individuals.
- Trial name
- Inspiratory (NCT06635343)
- Testing
- CPRP, Experimental Inspiratuar Muscle Training
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Kayseri University
Relapsed or Refractory Medulloblastoma (MB), Neuroblastoma (NB), Ewing Sarcoma (ES) and Alveolar Rhabdomyosarcoma (ARMS)
A Study of TB-403 in Pediatric Subjects With Relapsed or Refractory Medulloblastoma
The purpose of this study is to assess the safety and tolerability profile of TB-403 (humanized monoclonal antibody against placental growth factor (PlGF)) in pediatric subjects with relapsed or refractory Medulloblastoma.
- Trial name
- NCT02748135
- Testing
- TB-403 20mg/kg, TB-403 50mg/kg, TB-403 100mg/kg
- Where
- 10 sites in United States
- Volunteers
- 18 people
- Sponsor
- Oncurious NV
Hodgkin Disease, Non Hodgkin Lymphoma
LMP-specific T-cells for Patients With Relapsed EBV-positive Lymphoma
This protocol is broken up into 2 portions to determine the maximum tolerated dose for treating patients with a type of lymph gland disease.
- Conditions
- Hodgkin Disease, Non Hodgkin Lymphoma, Lymphoepithelioma, Leiomyosarcoma
- Trial name
- ALCI (NCT00062868)
- Testing
- LMP1/2 CTLs (ALCI - Group A), LMP1/2 CTLs (ALCI - Group B), LMP1/2 CTLs (ALCI - Group C)
- Where
- 2 sites in United States
- Volunteers
- 74 people
- Sponsor
- Baylor College of Medicine
Limb Girdle Muscular Dystrophy
MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants
A large cohort of MRI scans from patients with pathogenic variants in the anoctamin 5 gene will be collected through an international collaboration to better describe muscle involvement.
- Trial name
- ANO5 MRI (NCT05102799)
- Testing
- No intervention
- Where
- Denmark
- Volunteers
- 200 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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