Clinical trials: Neuromuscular Diseases (NMD)
Neuromuscular Diseases in Children
Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough
Neuromuscular diseases (NMD) in children are severe, possibly life-threatening orphan conditions. The children are vulnerable and often subject to rapid deterioration of pulmonary function due to impaired ability to clear airway…
- Trial name
- NCT04081116
- Testing
- Symmetric settings (high pressures/fast rate), Asymmetric settings (Pi<Pe/Ti>Te), Preinclusion settings
- Where
- 4 sites in Norway
- Volunteers
- 77 people
- Sponsor
- Oslo University Hospital
Muscular Dystrophy, Duchenne, Orthotic Devices
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.
- Conditions
- Muscular Dystrophy, Duchenne, Orthotic Devices, Upper Extremity, Neuromuscular Diseases (NMD)
- Trial name
- NCT06363357
- Testing
- Shoulder orthosis
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Seoul National University Hospital
Gait Impairment, Cerebral Palsy (CP)
Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting
The primary objective of the study is to validate the safety and clinical performance of the ABLE Kids device in pediatric patients with neurological gait disorders or muscular weakness during a 10-session gait training program…
- Conditions
- Gait Impairment, Cerebral Palsy (CP), Neuromuscular Diseases (NMD), Spinal Cord Injuries (SCI)
- Trial name
- NCT07745712
- Testing
- ABLE Kids
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- ABLE Human Motion S.L.
Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis)
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark.
- Conditions
- Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis), Spinal Cord Injuries (SCI), Duchenne Muscular Dystrophy (DMD)
- Trial name
- HOMEVENT DK (NCT07467187)
- Where
- Denmark
- Volunteers
- 450 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
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
Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1)
Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders
The primary aim is to characterize the prevalence, severity and quality of musculoskeletal nociceptive pain in adult patients with neuromuscular disorders (NMD).
- Conditions
- Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- NCT04907162
- Testing
- Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory
- Where
- Germany
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- LMU Klinikum
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Hereditary Motor and Sensory Neuropathy, Charcot-Marie-Tooth
Physical Activity in Persons With Charcot-Marie-Tooth: Developing a Measurement Instrument
The project will develop knowledge about physical activity in persons with Charcot-Marie-Tooth (CMT) in Norway. We plan to explore instruments to measure physical activity level for the target-group at the community level.
- Conditions
- Hereditary Motor and Sensory Neuropathy, Charcot-Marie-Tooth, Polyneuropathies
- Trial name
- NCT04461613
- Testing
- Original IPAQ followed by a revised one., Revised IPAQ followed by an original one.
- Where
- Norway
- Volunteers
- 20 people
- Sponsor
- Oslo University Hospital
Neuromuscular Disease
Respiratory Muscle Strength in Patients With NMD
The purpose of this study is to determine if inspiratory muscle strength training (IMST) will impact maximal inspiratory pressure and pulmonary function in patients with neuromuscular disease.
- Trial name
- RMST (NCT01555905)
- Testing
- Threshold PEP or IMT device Phillips-Respironics
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of Florida
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
Neuromuscular Disease
B3 for NMD: Bench to Bedside and Back
Background: Neuromuscular diseases (NMD) represent a broad group of rare genetic and acquired disorders, affecting over 300,000 people in Canada.
- Trial name
- NCT04417023
- Testing
- B3 for NMD: Bench to Bedside and Back
- Where
- Canada
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Ottawa Hospital Research Institute
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
- Trial name
- NCT07223632
- Testing
- VCA-894A
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Vanda Pharmaceuticals
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Charcot Marie Tooth Disease (CMT), Pes Cavovarus
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study
The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.
- Conditions
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery, Exercise Training
- Trial name
- CMT-WALK (NCT07432035)
- Testing
- Functional Surgery plus Rehabilitation, Physical Exercise
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Glycogen Storage Disease Type II, Pompe Disease (Late-onset)
Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease
The goal of this multicentre, randomized and controlled cross-over trial is to evaluate the efficacy of a programme of Inspiratory Muscle Training in subjects with Late On-set Pompe Disease (LOPD).
- Trial name
- LOPD01 (NCT05951790)
- Testing
- Inspiratory Muscle Training (IMT), Inspiratory Muscle Training (IMT) + Air Stacking
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione Don Carlo Gnocchi ETS
Neuromuscular Diseases (NMD)
Northwestern Neuromuscular Disease Biorepository
The primary objective of this research is to collect clinical data and tissue samples from adult patients with neuromuscular disease cared for at the Northwestern Memorial Hospital neuromuscular clinic (Lavin Building, 19th…
- Trial name
- NCT07477509
- Where
- United States
- Volunteers
- 2,500 people
- Sponsor
- Northwestern University
Hand Injuries and Disorders, Neurological Conditions
3D Hand Orthosis Trial
In people with chronic hand conditions, hand orthoses are frequently prescribed to improve performance in activities of daily living (ADL).
- Conditions
- Hand Injuries and Disorders, Neurological Conditions, Neuromuscular Diseases (NMD), Musculoskeletal Conditions (e.g., Tendinitis, Capsulitis)
- Trial name
- NCT07633821
- Testing
- 3D-printed hand orthosis (intervention), Conventional hand orthosis (control)
- Where
- Netherlands
- Volunteers
- 100 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Obstructive Sleep Apnea, Obesity Hypoventilation Syndrome (OHS)
Evaluation of Auto-EPAP for Management of Upper Airway Obstruction During Non-Invasive Ventilation
To evaluate the safety and effectiveness of the Automatic Expiratory Positive Airway Pressure (Auto-EPAP) feature versus manual expiratory positive airway pressure (EPAP) in the Vivo 45 LS Ventilator.
- Conditions
- Obstructive Sleep Apnea, Obesity Hypoventilation Syndrome (OHS), Chronic Obstructive Pulmonary Disease (COPD), Neuromuscular Diseases (NMD)
- Trial name
- Vivo NOVA (NCT06560411)
- Testing
- Non-Invasive Ventilation
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Breas Medical, Inc.
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