View clinical trials related to Neuromuscular Diseases.
Filter by:The study will be a non-randomized open label pilot study using an observational design comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.
Non-Invasive Ventilation (NIV) is an established treatment to manage respiratory muscles dysfunction in neuromuscular disease, preventing the progression of respiratory failure to intubation and/or a tracheotomy. NIV is commonly needed at first during the night, but when the disease worsens, it is required during the day. It is provided via nasal or oronasal masks, causing discomfort and/or aesthetic issues that result in poor compliance. Intermittent Abdominal Pressure Ventilation (IAPV) is a valid, though unconventional, alternative to daytime NIV: it consists of a portable ventilator with an internal battery and a corset as interface. The IAPV corset is lightweight, comfortable and, thanks to velcro fasteners, easier and better fitting than a face mask. Cyclical inflation of a rubber bladder inside the corset moves the diaphragm upwards like a pneumobelt causing air to enter in the lungs via the upper airways as gravity draws the diaphragm back to its resting position. IAPV is indicated in neuromuscular disease and has already been tested in few preliminary studies and case reports. This study wants to verify the hypothesis of its application in population of neuromuscular patients.
The purpose of this study is to assess the efficacy of using intelligent volume assured pressure support (iVAPS-AE) versus spontaneous timed (ST) modes of non-invasive ventilation (NIV) in patients diagnosed with amyotrophic lateral sclerosis (ALS). The investigators believe that the use of iVAPS-AE mode NIV over a 90 day period will produce NIV compliance data and health-related quality of life (HRQOL) scores that are equivalent or no worse compared to ST mode NIV.
The hand is important to perform activities of daily living (ADL). However, many people experience a loss of hand function as result of a traumatic brain injury, spinal cord injury, stroke or orthopedic problems, or due to ageing. To improve hand function, or reduce its decline, one can benefit from exercise therapy or use of assistive aids to improve ADL independence. A promising innovative approach combining both is a wearable soft-robotic glove that supports hand grip. With this glove, performance of functional activities can be supported directly, while also facilitating repeated use of the affected arm and hand during functional daily activities. One of our previous studies showed that besides a direct support effect, a therapeutic effect on performance was found after several weeks of using the soft-robotic glove as support during ADL. However, several participants reported complaints of increased pain and/or overload, mainly at the beginning of the trial. Clinicians suspect that a (too) high intensity of hand use compared to normal is contributing to this observation. This might be related to more fatigue experienced when using the glove in high-demand tasks, due to a larger movement capacity (faster, further, more repetitions) and can be associated with decreased blood perfusion/lower saturation levels at muscular level and altered muscle activation and movement coordination. Therefore, the primary objective is to examine the effect of use of the assistive soft-robotic glove during strenuous ADL tasks on the kinematic movement profile, compared to not using the soft-robotic glove. Secondary objectives are to examine whether pain or discomfort is experienced in strenuous activities with the soft-robotic glove as well as the characteristics and locations of such pain/discomfort, and to examine whether use of the glove is associated with increased handgrip strength, larger number of ADL task repetitions, diminished blood perfusion / reduced tissue saturation at the muscle and/or changes in muscle activity.
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.
The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a programmable ambulation exoskeleton (KeeogoTM Dermoskeleton System, B-Temia Inc., Quebec, Canada) 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.
Objectives: - To establish if physiotherapists can use the waveform traces from the cough assist machine to work out when patients are having an abnormal airway response to cough assist - To establish how cough assist device settings, particularly in breath and cough pressures affect a patient's response to using the cough assist device - To provide some clinical guidance to physiotherapists on methods for assessing and treating abnormal airway responses to cough assist devices Methodology: Subjects will complete breathing tests; spirometry, peak cough flow (PCF) and sniff nasal inspiratory pressure (SNIP) to establish baseline breathing function and rule out anyone with breathing conditions. A nasal camera will be used to look at the voice box at rest. Cough assist will be delivered via a face mask which will allow for simultaneous use of the nasal camera and cough assist carried out in the same way as another research team have done previously. The nasal camera will be attached to a video camera to allow recording, analysis and documentation of the observations. The cough assist protocol will be delivered by a physiotherapist experienced in delivering cough assist. Cough assist waveforms will be downloaded into Care Orchestrator software (Philips Respironics, Murraysville, USA) and reviewed at the same time as the nose camera recordings to establish if voice box responses can be identified from the waveform patterns. For confirmation of Care Orchestrator software waveforms, a device that records airflow during breathing (spirometer) will be connected (Alpha touch, Vitalograph, Ennis, Ireland) into the cough assist circuit in the same way another research team has before.
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. 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 the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.
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. Since 2008, children, adolescents and adults with DMD, BMD and SMA are registered with the help of all major muscle centers in Switzerland. After nearly ten years of activity, the Swiss Patient Registry for DMD/BMD and SMA implemented several adaptations in 2018 to meet current and future expectations of patient's organizations, health authorities and research organizations.
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology of respiratory impairment and guide therapeutic management. The use of an invasive technique (esogastric probe) makes it possible to specifically explore the diaphragm, the accessory inspiratory muscles and the expiratory muscles in order to detect dysfunction or paralysis of these muscles, and to estimate the work of breathing in order to better guide the respiratory management. The primary objective of the study is to evaluate the respiratory effort in children with primary or secondary impairment of the respiratory muscles during spontaneous breathing or during mechanical ventilation.