View clinical trials related to Neuromuscular Diseases.
Filter by:ALCOTRA (Alpes Latines COoperation TRAnsfrontalière) is one of the European cross-border cooperation programmes covering France and Italy and financed by the ERDF (European Regional Development Fund). It includes the thematic plan (PITEM), called "PROSOL" (PROximity and SOLidarity), set up in the PIEDMONT region (Italy). The PITEM PROSOL strategy aims to develop new social and health services for vulnerable populations in the rural areas and cross-border Franco-Italian mountains of the South regions (Provence Alpes Cote d'Azur, Liguria, Piedmont and Aosta valley). As part of the PITEM PROSOL project, a PROSOL telemedicine platform has been developped for the management of isolated patients from the territory of the Latin Alpes and suffering from neurological diseases (neurodevelopmental disorders, neuromuscular diseases and neurodegenerative diseases). These patients are divided into 3 experimental groups: WOMEN (project 5106), SENIORS (project 4128) and YOUTH (project 5162). A PROSOL e-learning platform (https://www.prosol-elearning.com/) has also been developped for these patients, their caregivers and community physicians to improve knowledge and management of these diseases. Experimentation of these platforms by several participants (and their caregivers) has highlighted the need, often discontent, of a personalized management of physiotherapy for patients with neuromuscular diseases (MNM) and neurodegenerative diseases (Alzheimer's disease). As physical activity has a beneficial and protective effect of these diseases, and inactivity is one of the important risk factors in worsening symptoms contributing to the loss of patients' motor and cognitive functional abilities, a program of self-physical rehabilitation has been designed by neurologists and physiotherapists of expert centers for a personalized and adapted treatment for each patient. The PROSOL TELEKINECT project offers a physical rehabilitation program to be carried out autonomously at home, with coaching by physiotherapists via the telemedicine platform, as well as close monitoring of exercise response regarding the level of pain and fatigue of patients, thus ensuring their maximum safety. The objective of the PROSOL TELEKINECT project is to evaluate the value of an appropriate physical rehabilitation programme for each type of patient. The feasibility and beneficial effects of this program will be assessed using conventional assessments of motor function and patient quality of life, but also using a connected watch coupled with artificial intelligence algorithms to collect and analyze physiological data remotely in real time in the patients' natural environment. The results of this pilot study will be used to lay the foundation for a larger clinical study to test a new digital strategy for self-treatment rehabilitation, aimed at reducing unequal access to care for patients with neuromuscular and neurodegenerative diseases, and residents of transboundary territories, thus offering the establishment of a preventive and supportive approach to these diseases.
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 patients. The investigators will study TSG via concomitant PDT and PUG procedures, while TDG will occur per usual care. This study is a prospective randomized open-label blinded endpoint study to assess the effect of tracheostomy with bedside simultaneous gastrostomy (TSG) versus the usual care of tracheostomy with delayed gastrostomy (TDG) placement on outcomes of neurocritically-ill patients.
This present study will be devoted to the first clinical study on the efficacy of the BELK system in enhancing mobility and improve the knee rehabilitation process in people with NeuroMuscular Diseases (NMD) and Central Nervous System (CNS) disorders with knee instability that implies deficit in gait and in locomotion during ADL. Participants will receive a 6-week training program (3 weeks with the BELK orthosis knee module) in a clinical setting in add-on to the standard neuro-rehabilitation treatment. Gait analysis and Instrumental Test will be performed every week till the end of the six-week training period while the Clinical Scales and Questionnaires will be performed after three weeks and at the end of the training period. Finally, additional outcome measures comprise the scores on the System Usability Scale (SUS) to evaluate the hardware and the Software Usability Measurement Inventory (SUMI) to evaluate the software of the BELK orthosis. These two measures will be administrated at the end of training with Belk orthosis, to rate patients' and operators' (e.g., physical therapist, medical doctors) satisfaction. During 2018, the company GOGOA (www.gogoa.eu), specialized in design, manufacturing and commercialization of Robotic Assisted Rehabilitation (RAR) systems, has developed a first prototype of the BELK system, a powered wearable robotic device that can be used for knee rehabilitation, in the sub-acute phase of knee injuries. As BELK is a wearable device, it can be used both, by the physiotherapist in the rehabilitation centers, improving the knee rehabilitation process and increasing patients' comfort, and accelerating their rehabilitation process. Casa di Cura del Policlinico (CCP https://www.ccppdezza.it/en/) is a fully integrated multi-specialty clinical center aiming at providing both inpatient and outpatient services mainly directed to neurological patients. The Center is constituted as a Department of Neuro-rehabilitation Sciences, accredited by the Italian National Health System, and economically supported by Regione Lombardia. CCP offers to chronic neurological patients the highest comprehensive standards of care in a comfortable environment, through a multidisciplinary patient management approach organized around a core rehabilitation program.
Up to 20% of the adolescents worldwide are diagnosed with a chronic illness. Although it's known that these young people suffer from a wide range of physical discomfort, in real life they have to cope with much more challenges whether or not caused by their physical situation. Psychiatric disorders (e.g. ASS, ADHD or depression) are more often diagnosed in this population compared to their healthy peers. The causal mechanisms for this higher risk, however, are yet unknown. This project examines three underlying intrapersonal processes possibly having an impact on the evolution of these secondary symptoms. Previous research confirmed the significant impact of having a chronic illness on the development of identity and the association with a higher prevalence of depressive symptoms. Identity formation is a crucial developmental task to transfer from a child to an independent, responsible adult. We believe this developmental task is more at risk for youngsters and young adults suffering from a physical disability, caused by a neuromuscular disease. In this longitudinal research project, we investigate the development of identity in youth with a neuromuscular disease, compared to healthy peers and compared to a group of peers with a non-visible chronical illness. We also evaluate the impact of the degree of physical functioning on those mechanisms. Second, we examine if parental behavior and parental functioning is linked with the formation of identity in a neuromuscular populations. Finally, the impact of identity formation on psychosocial outcomes (e.g. feelings of depression and anxiety, quality of life, …) is measured. By addressing these research questions, this project will add substantially to our knowledge on identity in chronic illness and can inform future prevention and intervention efforts targeting illness adaptation and parental functioning in order to prevent negative psychosocial outcomes and to optimize quality of life.
Clinical trials organization in several neuromuscular disorders (NMD) has some specific issues. Nonambulant status and difficulties with transportation are among them. Moreover a lot of patients with NMD have so poor condition that even short transportation is able to worse it. Such situation forces researchers to limit a region of recruitment for clinical trials and to exclude from trials more severe subgroup of patients, which cause additional issues especially for rare diseases. The purpose of this study is to prove hypothesis about possibility to reliably monitor patient condition remotely, without trial site visiting. Visit-free study design is potentially able to widen eligible patient population and to decrease patient dropout rate as well as burden of numerous assessments. Meanwhile assessment frequency could be increased enabling monitoring of short fluctuations in patients' condition. Spinal muscular atrophy (SMA) is a rare neuromuscular condition to which all mentioned above issues are completely applicable. Direct current stimulation (DCS) of neural structures is well studied and safe intervention, however, its effects on SMA patients' strength and durability has not been reported for today. The investigators suppose that investigation of DCS action in SMA patient population is an adequate model for visit-free design feasibility, reliability and sensitivity evaluation.
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.
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. The investigators hypothesise that autotitrating NIV will ventilate patients with acute exacerbations of chronic respiratory failure as effectively as standard NIV.