Clinical trials: Neuromuscular Diseases (NMD)
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), Pneumothorax
Incidence and Risk Factors of Pneumothorax in Patients With Neuromuscular Diseases
Neuromuscular diseases can cause weakness of the respiratory muscles, leading to respiratory pump failure and respiratory insufficiency.
- Trial name
- MYOPNEUMO (NCT07909018)
- Testing
- Cough assistance device and/or thoracic mobilization and/or mechanical ventilation
- Where
- France
- Volunteers
- 31,202 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy
The goal of this clinical trial is to learn if providing a multicomponent intervention improves the confidence of caregivers of children with DMD or SMA.
- Trial name
- SEE-NMD (NCT07356063)
- Testing
- Multicomponent Intervention
- Where
- Pakistan
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
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
Duchenne Muscular Dystrophy (DMD), Chronic Pain
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
1. Brief Summary This prospective observational study aims to evaluate the 6-month longitudinal changes in chronic pain, pain characteristics, coping strategies, and functional performance in children and adolescents aged 7 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Chronic Pain, Neuromuscular Diseases (NMD)
- Trial name
- NCT07892196
- Testing
- Routine Clinical Follow-up and Pain Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Antalya Training and Research Hospital
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Optimum Insufflation Capacity in NMD
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Chronic Respiratory Insufficiency
- Trial name
- NCT01981915
- Testing
- IPPB, LIAM
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Universität Duisburg-Essen
Neuromuscular Diseases
Oscillation and Lung Expansion (OLE) Therapy for Treatment of Neuromuscular Disease (NMD) Patients
The study will be a non-randomized open label pilot study comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.
- Trial name
- NCT05366010
- Testing
- Oscillation and Lung Expansion (OLE) therapy
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Baxter Healthcare Corporation
Direct Acting Anticoagulant Adverse Reaction, Needle Injury
Ultrasound Evaluation of Hematoma Risk After Needle EMG in Patient on DOAC Therapy
Approximately 30 minutes after needle EMG, patients who are taking direct oral anticoagulants (DOACs) will undergo an ultrasound examination to evaluate for the presence of possible intramuscular hematomas at the muscles where…
- Conditions
- Direct Acting Anticoagulant Adverse Reaction, Needle Injury, EMG, Peripheral Neuropathies
- Trial name
- U-HAND (NCT07166302)
- Where
- 2 sites in Czechia
- Volunteers
- 70 people
- Sponsor
- Masaryk University
Home-based Programs, EzyGain
EMMVIES : Walking Training at Home in VIrtual Reality for Children With Chronic Illnesses Impacting Physical Health
Over the past 30 years, the prevalence of congenital or acquired chronic diseases in children has risen, affecting between 10 and 30% of them, or at least 100,000 children in France.
- Conditions
- Home-based Programs, EzyGain, AMY Treadmill, Cerebral Palsy
- Trial name
- EMMVIES (NCT07021638)
- Testing
- EMMVIES program
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Angers
SMA, DMD
Swiss Registry for Neuromuscular Disorders
The Swiss Patient Registry for DMD/BMD and SMA was launched in 2008 in order to give Swiss patients access to new therapies. It was founded with the financial support of several patient organizations and research foundations.
- Conditions
- SMA, DMD, BMD, IMD
- Trial name
- Swiss-Reg-NMD (NCT05102916)
- Where
- 19 sites in Switzerland
- Volunteers
- 2,000 people
- Sponsor
- University of Bern
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
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
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
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
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
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Neuromuscular Diseases, Children, Only
Mechanical Cough Augmentation in Children With NMD
The study investigates the prevalence and use of mechanical insufflation - exsufflation (MI-E) in children with Neuromuscular disorders (NMD) in Norway.
- Conditions
- Neuromuscular Diseases, Children, Only, Airway Clearance Impairment, Cough
- Trial name
- NCT03831568
- Where
- Norway
- Volunteers
- 72 people
- Sponsor
- Oslo University Hospital
Neuromuscular Diseases, Neurological Diseases or Conditions
Perceptions of the Clinical and Psychosocial Impact of Covid-19 in Patients With NMD
This study aims to evaluate perceived clinical and psychosocial experiences of patients and their families during the Covid-19 pandemic.
- Trial name
- CLASSIC (NCT04581577)
- Testing
- Semi-structured telephone questionnaire
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust
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
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
The Duchenne Registry
The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
- Trial name
- NCT02069756
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- The Duchenne Registry