Clinical trials: Neuromuscular Disorder
Neuromuscular Diseases, Neurologic Disorder
Multisystem and Autonomic Complications of NMD on Long-term NIV
This exploratory observational study evaluates autonomic function using measures including heart rate variability in adults with childhood-onset neuromuscular and neurological disorders (NMD) who use non-invasive ventilation…
- Trial name
- NCT04100993
- Testing
- No intervention used
- Where
- United Kingdom
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust
Neuromuscular Diseases, Neurodevelopmental Disorders
Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability
Research Aim: This study investigates whether a 12-month treatment with hypertonic saline (salty water) can reduce antibiotic use in individuals with neuromuscular disease or cerebral palsy who frequently experience chest…
- Conditions
- Neuromuscular Diseases, Neurodevelopmental Disorders, Cerebral Palsy
- Trial name
- SPICE-UP (NCT06134401)
- Testing
- saline
- Where
- 2 sites in United Kingdom
- Volunteers
- 40 people
- Sponsor
- Imperial College London
Stroke, Dysphagia
Effects of ITBS Combined with NMES on Dysphagia After Stroke
In recent years, research on intermittent Theta Burst Stimulation (iTBS), as a special high-frequency （repetitive transcranial magnetic stimulation，rTMS） stimulation paradigm, has focused on exploring the effects in healthy…
- Conditions
- Stroke, Dysphagia, Intermittent Theta Burst Stimulation, Neuromuscular Electrical Stimulation
- Trial name
- NCT06464835
- Testing
- Transcranial magnetic stimulation
- Where
- China
- Volunteers
- 45 people
- Sponsor
- The First Affiliated Hospital of Zhengzhou University
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to <12 years).
- Trial name
- LELANTOS-2 (NCT04632940)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 52 sites in United States, Australia and 9 more
- Volunteers
- 73 people
- Sponsor
- Kyntra Bio
Myasthenia Gravis, Autoimmune Diseases of the Nervous System
Predictors and Prognostic Factors of Myasthenia Gravis Outcome
This study aims to characterize the clinical features, frequency of different subgroups of MG, and identify predictors of treatment responsiveness among different subgroups of MG.
- Conditions
- Myasthenia Gravis, Autoimmune Diseases of the Nervous System, Neuromuscular Junction Diseases, Thymoma
- Trial name
- NCT05214612
- Testing
- Drug treatment of myasthenia gravis and treatment of crisis
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Assiut University
Neuromuscular Disorders, Chest Wall Disorders
Response to NonInvasive Mechanical Ventilation According to the Breathing Pattern
The purpose of this study is to determine whether breathing pattern (specifically the inspiratory time/total respiratory cycle value) has an influence over the response to the noninvasive mechanical ventilation.
- Conditions
- Neuromuscular Disorders, Chest Wall Disorders, Obesity Hypoventilation Syndrome (OHS)
- Trial name
- NCT01991535
- Testing
- Spontaneous Ventilation Mode, Controlled Ventilation Mode, Simulator Ventilation Mode
- Where
- Spain
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Neuromuscular Disorders
Myotonia and Muscle Stiffness in NMD
The primary objective of this study is to assess stiffness, muscle tone, relaxation periods and elasticity of various muscles in patients with dystrophic or non-dystrophic myotonia.
- Trial name
- NCT04411732
- Testing
- MyotonPro, Six-minute walk test, Semiquantitative muscle ultrasound
- Where
- Germany
- Volunteers
- 133 people
- Sponsor
- LMU Klinikum
Adenovirus, Anesthesia
Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care
Understudied drugs will be administered to children per standard of care as prescribed by their treating caregiver and only biological sample collection during the time of drug administration will be involved.
- Conditions
- Adenovirus, Anesthesia, Anxiety, Anxiolysis
- Trial name
- PTN_POPS (NCT01431326)
- Testing
- The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 3,520 people
- Sponsor
- Daniel Benjamin
Anesthesia, General, Neuromuscular Transmission Disorders
Influence of Sevoflurane and Propofol on Maximum Muscular Strength, Speed of Contraction and Relaxation
Many drugs have an influence on neuromuscular transmission. In clinical practice, neuromuscular blocking agents are commonly used, but even in the absence of neuromuscular blocking agents, anesthetic drugs can influence…
- Trial name
- NCT05615025
- Testing
- Sevoflurane, Propofol
- Where
- Belgium
- Volunteers
- 48 people
- Sponsor
- Brugmann University Hospital
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy
Molecular Analysis of Patients With Neuromuscular Disease
The purpose of this study is to identify new genes responsible for neuromuscular disorders and study muscle tissue of patient with known neuromuscular disease, as well as their family members.
- Conditions
- Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
- Trial name
- NCT00390104
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Healthy Volunteer, Muscular Dystrophies
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
This is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506.
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT05730842
- Testing
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Sleep Disorder; Breathing-Related, Central Apnea
Transcutaneous CO2 Monitoring at Home for Children With Neuromuscular Disease
There is an unmet demand for the evaluation of nocturnal hypoventilation in children with NMD. An ambulatory screening tool that can reliably facilitate timely diagnosis and treatment in these children would be invaluable.
- Conditions
- Sleep Disorder; Breathing-Related, Central Apnea, Neuromuscular Diseases, Obstructive Sleep Apnea
- Trial name
- NCT03478566
- Testing
- Transcutaneous CO2 monitoring
- Where
- Canada
- Volunteers
- 39 people
- Sponsor
- The Hospital for Sick Children
Disuse Atrophy (Muscle) of Lower Extremities, Muscle Function
The Impact of Bed Rest, Aging and NMES on Skeletal Muscle
Loss of muscle mass is common phenotypic trait of muscular disuse and ageing. The loss of muscle mass affects, among others, the ability to maintain homeostasis of glucose metabolism and the energy reservoir in catabolic…
- Conditions
- Disuse Atrophy (Muscle) of Lower Extremities, Muscle Function, Neuromuscular Electrical Stimulation, Myofibrillar Protein Synthesis
- Trial name
- NCT05617222
- Testing
- Bed rest, Neuromuscular electrical stimulation
- Where
- Denmark
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Bispebjerg Hospital
Spinal Muscular Atrophy, Quality of Life
Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
The aim of the investigator's study was to investigate translating the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old and using it in clinics reliably and validity with a Turkish version of the PedsQL Generic Core…
- Conditions
- Spinal Muscular Atrophy, Quality of Life, Neuromuscular Diseases, Pediatric Disorder
- Trial name
- NCT05539456
- Where
- Turkey (Türkiye)
- Volunteers
- 55 people
- Sponsor
- Istanbul Medipol University Hospital
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Trial name
- NCT04201418
- Testing
- Patisiran
- Where
- 27 sites in United States
- Volunteers
- 67 people
- Sponsor
- Alnylam Pharmaceuticals
Autoimmune Diseases, Cardiovascular Disorders
Mesenchymal Stem Cells for the Treatment of Various Chronic and Acute Conditions
This multi-arm, multi-site study investigates the safety, tolerability, and efficacy of stem cell therapy for the treatment of various acute and chronic conditions.
- Conditions
- Autoimmune Diseases, Cardiovascular Disorders, Diabetes Complications, Integumentary Disease
- Trial name
- NCT04684602
- Testing
- PrimePro™/ PrimeMSK™
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Thomas Advanced Medical LLC
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle…
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Trial name
- CMDPROS (NCT01403402)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Cure CMD
Nocturnal Hypoventilation, Neuromuscular Disease
Autotitrating Versus Standard Non-invasive Ventilation (NIV) in Newly Diagnosed Patients
The aim of the study is to compare the efficacy and tolerance of autotitrating non-invasive ventilation (NIV) versus standard NIV in patients with newly diagnosed nocturnal hypoventilation who have never experienced nocturnal…
- Conditions
- Nocturnal Hypoventilation, Neuromuscular Disease, Chest Wall Disorder
- Trial name
- NCT00901485
- Testing
- AutoVPAP, VPAPIIIST-A
- Where
- United Kingdom
- Volunteers
- 23 people
- Sponsor
- ResMed
Stroke, Acute, TBI
Tracheostomy With Bedside Simultaneous Gastrostomy Vs Usual Care Tracheostomy And Delayed Gastrostomy Placement
There is currently no prospective study analyzing the effect of tracheostomy with bedside simultaneous gastrostomy versus tracheostomy with delayed gastrostomy placement (TSG versus TDG) on the outcomes of neurocritically-ill…
- Conditions
- Stroke, Acute, TBI, Neuromuscular Diseases, Status Epilepticus
- Trial name
- BEGASTON (NCT04758052)
- Testing
- Timing of placement
- Where
- United States
- Sponsor
- University of Mississippi Medical Center
Respiratory Failure, Neuromuscular Disease
Autotitrating Versus Standard Non-invasive Ventilation (NIV) in Acute Exacerbation of Respiratory Failure
The aim of the study is to compare the efficacy and tolerance of autotitrating non-invasive ventilation (NIV) versus standard NIV in patients admitted to hospital with acute exacerbation of chronic respiratory failure.
- Conditions
- Respiratory Failure, Neuromuscular Disease, Chest Wall Disorder, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT01174316
- Testing
- AutoVPAP™, VPAPIIIST-A™
- Where
- United Kingdom
- Sponsor
- ResMed