Clinical trials: Neuromuscular Disorder
Muscular Dystrophy, Duchenne, DMD
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
- Trial name
- NCT04337112
- Testing
- viltolarsen
- Sponsor
- NS Pharma, Inc.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- ACT DMD (NCT01826487)
- Testing
- Ataluren
- Where
- 55 sites in United States, Australia and 16 more
- Volunteers
- 230 people
- Sponsor
- PTC Therapeutics
Cervical Dystonia, Movement Disorders
Non-Motor Features of Cervical Dystonia (CD)
This study will examine the prevalence of four previously identified non-motor markers in a population of cervical dystonia patients, unaffected family members, and healthy volunteers in an attempt to identify a distinct…
- Conditions
- Cervical Dystonia, Movement Disorders, Focal Dystonia, Signs and Symptoms
- Trial name
- NCT03471923
- Testing
- Neurological Examination, Assessment of Non-Motor Features
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Infectious Disorder of Trachea, Respiratory Tract Infections
Bacterial Colonization in Tracheostomized Patients With Neurological or Neuromuscular Disease
Patients with neurological or neuromuscular diseases may need a long-term tracheostomy to improve their respiratory function. Bacterial flora and bacterial drug resistance in the respiratory tract have never been studied until…
- Conditions
- Infectious Disorder of Trachea, Respiratory Tract Infections, Mechanical Ventilation Complication
- Trial name
- ECOTRACH (NCT02400086)
- Where
- France
- Volunteers
- 77 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Dysphagia, Neuromuscular Diseases
Validation of the French Version of the Sydney Swallow Questionnaire in Patients With Neuromuscular Diseases
Measurements of dysphagia severity are important when making management decisions and in the objective evaluation of swallowing impairments.
- Trial name
- NCT02845362
- Testing
- Sydney Swallow Questionnaire
- Where
- 2 sites in Belgium
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Neuromuscular Block, Orthopedic Disorder of Spine
Neuromuscular Blockade for Optimising Surgical Conditions During Spinal Surgery
In the present study, the investigators compare intermediate and deep NMB i.e. the two extreme regimes of muscle paralysis (neuromuscular block; NMB), (I) patients receiving intermediate muscle paralysis (''control, conventional…
- Conditions
- Neuromuscular Block, Orthopedic Disorder of Spine, Muscle Weakness
- Trial name
- NCT02778945
- Testing
- Rocuronium 0.9 mg/kg, Rocuronium 0.6 mg/kg
- Where
- South Korea
- Volunteers
- 60 people
- Sponsor
- The Catholic University of Korea
Spinal Muscular Atrophy, Neuromuscular Disorders
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them.
- Trial name
- NCT02235090
- Testing
- Direct current stimulation of cervical spinal cord
- Where
- Ukraine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Charitable Foundation Children with Spinal Muscular Atrophy
Neurological Disorders, Neuromuscular Disorders
Genetic and Blood Biomarkers in Neurological and Neuromuscular Diseases
The purpose of this study is to identify genetic or other factors in the subjects blood that may predispose them to getting a particular disease or tell researchers how the disease will behave, for example how fast it will…
- Trial name
- Neurogenetic (NCT02780531)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- St. Louis University
Neuromuscular Transmission Disorder, Diabetes Mellitus, Type 2
Effect of Type II Diabetes Mellitus With Neuropathy on the Clinical Use of Rocuronium
International Diabetes Federation estimates that there are now 415 million adults aged 20-79 with diabetes mellitus worldwide. By 2040 this will rise to 640 million.
- Trial name
- NCT03737942
- Testing
- monitoring of neuromuscular function
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Assiut University
Extraocular Muscle Disorder, Neuromuscular Diseases
Oculomotor Training and Chinese Characters Recognition in Children With Neuromuscular Disease
The aim of this study is to investigate the effects of gaze-directed oculomotor training incorporated with web-based curriculum readings in Chinese to enhancing fixation, saccade and Chinese characters recognition in…
- Trial name
- yes (NCT03627962)
- Testing
- gaze-directed oculomotor training
- Where
- Hong Kong
- Volunteers
- 21 people
- Sponsor
- The Hong Kong Polytechnic University
Neuromuscular Disorders
Upper Limb Evaluation in Non Ambulatory Patients With Neuromuscular Disorder
The aim of this study is to evaluate different devices to quantify upper limb function (Motriplate) and strength (Myogrip, Myopinch) in non-ambulatory patients with neuromuscular disorder.
- Trial name
- ULENAP (NCT00993161)
- Testing
- Motriplate
- Where
- 4 sites in France
- Volunteers
- 174 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
Respiratory Failure, Neuromuscular Disorder
Regional Distribution of Ventilation to Assess Respiratory Muscle Dysfunction
The aim of this study is to better discriminate respiratory muscle dysfunction by comparing the measurements of thoracoabdominal motion obtained by an optoelectronic recording and the conventional tests of respiratory muscle…
- Conditions
- Respiratory Failure, Neuromuscular Disorder, Diaphragmatic Paralysis
- Trial name
- DREM (NCT01569217)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Brain Injuries, Dementia
ACT-ICU Study: Activity and Cognitive Therapy in the Intensive Care Unit
Intensive care unit (ICU) hospitalization saves lives but often does so at a high personal cost to ICU survivors who frequently experience significant cognitive impairment and an array of physical and functional disabilities that…
- Conditions
- Brain Injuries, Dementia, Myopathies, Muscle Disorders
- Trial name
- ACT-ICU (NCT01270269)
- Testing
- Behavioral: Physical and functional rehabilitation, Behavioral: Cognitive, physical, & functional rehabilitation
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- Vanderbilt University
Colorectal Disorders, Observation of Neuromuscular Block
Effectiveness of Transverse Abdominus Plane Catheter Blocks to Patient-controlled Analgesia in Laparoscopic Colon Resections
The control of postoperative pain has become a major issue in surgery awareness and it is considered an important measurement of patient satisfaction. Improvements in pain relief, including stopping pain before it starts (i.e.
- Trial name
- NCT01592630
- Testing
- 0.2% ropivacaine, Saline
- Where
- 2 sites in United States
- Sponsor
- Stamford Hospital
Neuromuscular Disorders
Screening Questionnaire for Respiratory Muscle Weakness and Sleep-disordered Breathing in Neuromuscular Disorders
It is the aim of this project to develop and validate a German language screening questionnaire for symptoms of respiratory muscle weakness and sleep-disordered breathing (SDB) in patients with neuromuscular disorders.
- Trial name
- NCT02833168
- Testing
- patient-filled questionnaires, spiromanometry
- Where
- 2 sites in Germany
- Volunteers
- 90 people
- Sponsor
- Universität Münster
Neuromuscular Disorder, Respiratory Failure
Optimizing Communication During Ventilation
Mechanical ventilation is one of the only treatment that has improved survival of patients with neuromuscular respiratory failure. As disease progresses, some patients may require longer ventilation period.
- Trial name
- PHONOVNI (NCT01819090)
- Testing
- Elysee 150 ventilator, Elysee 150 ventilator with a ventilation switch control
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Respiration Disorders, Neuromuscular Disease
Evaluation of Vital Capacity
Respiratory failure is the most common cause of death in neuromuscular diseases. The aim of this research is to evaluate the thoracopulmonary recruitment by comparison of inspiratory vital capacity (VC ins)and…
- Trial name
- CVassist (NCT02022072)
- Testing
- measures of assisted vital capacity by a mechanical insufflation/exsufflation (Cough Assist®)
- Where
- France
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Adult Patients With Neuromuscular Disorders Using Wheelchair
Wheelchair Positioning and Pain in Neuromuscular Patients
A retrospective study with the patients having a positioning consultation at hospital, between 2009 and 2014. In every neuromuscular disorder, the investigators describe the population seen in consultation, the aim of…
- Trial name
- NCT02141724
- Testing
- Positioning consultation
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Paraparesis, Tetraparesis
Prospective Cohort Follow-up of French Patients With Profound and Multiple Disabilities: Healthcare Pathways and Quality of Life Among Patients and Their Families
The studies conducted so far concerning the medical and paramedical cares provided to patients with profound and multiple disabilities (PMD) often show important limitations: samples are too small or very heterogeneous, generally…
- Conditions
- Paraparesis, Tetraparesis, Hemiparesis, Ataxia
- Trial name
- Eval-PLH (NCT02400528)
- Testing
- quality of life questionnaires
- Where
- France
- Volunteers
- 1,200 people
- Sponsor
- Assistance Publique Hopitaux De Marseille