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Showing 1-20 of 29 trials
NCT05747924
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT05200702
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a powered knee-hip dermoskeleton (MyoSuit, MyoSwiss, Zurich, Switzerland) in patients with neuromuscular disorders, ii) Elaborate recommendations regarding usability criteria for safe and efficient use the device in patients with neuromuscular disorders (e.g. type and severity of patient's functional deficits), iii) generate necessary data to foresee a future study involving a home use of the device and assessment of long-term benefits.
NCT07250737
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
NCT01568658
Background: \- Some nerve and muscle disorders that start early in life (before age 25), like some forms of muscular dystrophy, can run in families. However, the genetic causes of these disorders are not known. Also, doctors do not fully understand how symptoms of these disorders change over time. Researchers want to learn more about genetic nerve and muscle disorders that start in childhood by studying affected people and their family members, as well as healthy volunteers. Objectives: \- To better understand nerve and muscle disorders that start early in life and run in families. Eligibility: * Individuals at least 4 weeks old with childhood-onset muscular and nerve disorders, including those who have a later onset of a disorder that typically has childhood onset. * Affected and unaffected family members of the individuals with muscular and nerve disorders. * Healthy volunteers at least 4 weeks old with no nerve or muscle disorders. Design: * Participants will be screened with a physical exam and medical history. Genetic information will be collected from blood, saliva, cheek swab, or skin samples. Urine samples may also be collected. * Healthy volunteers and unaffected family members will have imaging studies of the muscles. These studies will include magnetic resonance imaging (MRI) and ultrasound scans. Results will be compared with those from the affected participants. * All participants with nerve and muscle disorders will have multiple tests, including the following: * Imaging studies of the muscles, including ultrasound and MRI scans. * Imaging studies of the bones, such as x-rays and DEXA scans. * Heart and lung function tests. * Eye exams. * Nerve and muscle electrical activity tests and biopsies. * Video and photo image collection of affected muscles. * Speech, language, and swallowing evaluation. * Lumbar puncture to collect spinal fluid for study. * Tests of movement, attention, thinking, and coordination. * Participants with nerve and muscle disorders will return to the Clinical Center every year. They will repeat the tests and studies at these visits.
NCT05724173
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with their thoughts.
NCT06094205
The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to speak fluently to communicate via a computer just by trying to speak.
NCT07138963
The aim of this study is to correlate the phenotype and genotype among a sample of Egyptian patients with Congenital myopathies and Congenital muscular dystrophies.
NCT07129954
Primary objectives WP1: Evaluate the prevalence of FOF in the study population and how this varies over time. Evaluate whether there are relationships between the variables investigated (clinical, motor, cognitive, psychological) and the presence of FOF. WP2: To evaluate, among those who presented disabling FOF, the effects of two different therapeutic approaches: motor rehabilitation vs. motor rehabilitation plus cognitive-behavioral psychotherapy. Secondary objectives WP1: To evaluate whether different profiles defined by specific clinical, motor, cognitive, psychological, and personological characteristics can be characterized among patients with dystrophy and FOF and how these impact functionality, activity, participation, and quality of life. WP2: Evaluate the effects of cognitive-behavioral therapy (CBT) and a motor treatment on cognitive and psychological aspects, the frequency of falls, and the functional validity.
NCT03698149
Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurological disorders.
NCT03406780
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.
NCT06547216
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT02921321
Muscular Dystrophy can affect the skeletal muscles and also the heart and breathing muscles, causing significant morbidity and mortality. As patients are now living longer, treatment of muscular dystrophies involves drugs that help improve heart function. However, better types of heart imaging studies are needed to understand how these treatments work. Researchers want to improve heart imaging to identify earlier indicators of heart dysfunction in muscular dystrophy patients and how these are changed by medical treatment. The new imaging indicators will also help identify candidates for entry into future clinical trials.
NCT05890833
The combination of short quantitatively assessing muscular function and balance in combination with short clinical scores, can be a new valid approach to evaluate the patient risk of fall and help to create a quick checkup test to prescribe an appropriate assistive device. The primary goal of this project is to provide a short battery of clinical assessments used to determine risk of falling for patients with neuromuscular diseases (NMD) based on correlation between clinical assessments between two groups of NMD patients and scales used to assess risk of falling for patients.
NCT05881122
This study investigated the effects of a 2-part dietary consultation on adherence to an anti-inflammatory diet in individuals with neuromuscular disability. The effects on self-efficacy for adhering to the diet as well as neuropathic pain and depression one month post-consult were also determined.
NCT05409079
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home environments. Functional outcomes will include documenting active range of motion and the ability to perform activities of daily living (ADLs) using the standardized Canadian Occupational Performance Measure (COPM) and the Role Evaluation of Activities of Life (REAL) assessments. Secondary objectives are to assess the safety record and report on adverse events (AEs) and parameters related to device usage, including device usage time and the time required to don/doff the device. Secondary objectives also include characterization of user upper limb performance based on etiology.
NCT03986671
This is a pilot study to examine the diagnostic utility of a novel transmembrane surface sensor, and compare signals obtained with the transmembrane sensor to conventional needle EMG signals from healthy volunteers to those with documented neurologic pharyngeal muscle dysfunction (ALS and muscular dystrophy) and to those with severe OSA.
NCT01783509
The purpose of this study is to understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination, assessment of patient (or parent) understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all parts of your blood). By tracking this information, we hope to be able to understand more about the diagnosis, progression and natural history of this disorder.
NCT01950897
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
NCT04187482
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss. As a result, individuals suffering from DM1 become very inactive and lose mobility resulting in a lower quality of life. This study will investigate the effect of a 12-week moderate intensity exercise protocol on skeletal muscle function and cellular benefits in DM1 patients.
NCT04154098
Shoulder instability due to muscle weakness is a common problem in disorders of the upper extremities. During arm motion, the scapula acts as a dynamic base for the humeral head. To safely move the shoulder with an exoskeleton for the upper extremities a textile orthosis was developed that stabilizes the scapula against the thorax. The support level of the orthosis is continuously manually adjustable. To test the feasibility of our design and to improve the functionality of the textile orthosis, it needs to be investigated how the orthosis acts on people affected by shoulder instability. The investigators seek to explore how people with shoulder instability respond to the orthosis, and how they may benefit from the orthosis function. Therefore, the range of motion of arm elevation will be compared in different conditions: (i) without any support, (ii) with the support of a trained therapist, and (iii) when the device is engaged at the individual's optimal support level. Additionally, pilot tests will be performed to fix different parameters in our study protocol, such as the the optimal orthosis stiffness level and the ideal number of movement repetitions.