Active clinical trials for "Motor Neuron Disease"

Individuals suffering from tetraplegia as a result of cervical spinal cord injury, brainstem stroke, or amyotrophic lateral sclerosis (ALS) cannot independently perform tasks of daily living. In many cases, these conditions do not have effective therapies and the only intervention is the provision of assistive devices to increase independence and quality of life. However, currently available devices suffer from usability issues and are limiting for both the patient and caregiver. One of the most progressive alternative strategies for assistive devices is the use of brain-computer interface (BCI) technology to translate intention signals directly from sensors in the brain into computer or device action. Preclinical primate research and recent human clinical pilot studies have demonstrated success in restoring function to disabled individuals using sensors implanted directly in motor regions of the brain. Other preclinical primate research has demonstrated effective intention translation from sensors implemented in cognitive regions of the brain and that this information complements information from the motor regions. The current proposal seeks to build on these studies and to test the safety aspects related to implanting two sensors, each a microelectrode array, into both the motor and cognitive regions of the brain in motor impaired humans. Secondary objectives include feasibility evaluation of the complementary sensors in their ability to support effective assistive communication.

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.

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.

Erythropoietin is neuroprotective in animal models of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The aim of this study was to determine the safety and feasibility of repetitive high-dose recombinant human erythropoietin (rhEPO) therapy in ALS patients.

Single-center, prospective pilot study on patients with amyotrophic lateral sclerosis fitted with noninvasive ventilation. The objective is to assess the satisfaction of remote monitoring of patients on non-invasive ventilation after 12 months.

This project adds to non-invasive BCIs for communication for adults with severe speech and physical impairments due to neurodegenerative diseases. Researchers will optimize & adapt BCI signal acquisition, signal processing, natural language processing, & clinical implementation. BCI-FIT relies on active inference and transfer learning to customize a completely adaptive intent estimation classifier to each user's multi-modality signals simultaneously. 3 specific aims are: 1. develop & evaluate methods for on-line & robust adaptation of multi-modal signal models to infer user intent; 2. develop & evaluate methods for efficient user intent inference through active querying, and 3. integrate partner & environment-supported language interaction & letter/word supplementation as input modality. The same 4 dependent variables are measured in each SA: typing speed, typing accuracy, information transfer rate (ITR), & user experience (UX) feedback. Four alternating-treatments single case experimental research designs will test hypotheses about optimizing user performance and technology performance for each aim.Tasks include copy-spelling with BCI-FIT to explore the effects of multi-modal access method configurations (SA1.3a), adaptive signal modeling (SA1.3b), & active querying (SA2.2), and story retell to examine the effects of language model enhancements. Five people with SSPI will be recruited for each study. Control participants will be recruited for experiments in SA2.2 and SA3.4. Study hypotheses are: (SA1.3a) A customized BCI-FIT configuration based on multi-modal input will improve typing accuracy on a copy-spelling task compared to the standard P300 matrix speller. (SA1.3b) Adaptive signal modeling will allow people with SSPI to typing accurately during a copy-spelling task with BCI-FIT without training a new model before each use. (SA2.2) Either of two methods of adaptive querying will improve BCI-FIT typing accuracy for users with mediocre AUC scores. (SA3.4) Language model enhancements, including a combination of partner and environmental input and word completion during typing, will improve typing performance with BCI-FIT, as measured by ITR during a story-retell task. Optimized recommendations for a multi-modal BCI for each end user will be established, based on an innovative combination of clinical expertise, user feedback, customized multi-modal sensor fusion, and reinforcement learning.

The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.

Background/scope There is growing recognition that family caregiving is a serious public health issue requiring supportive interventions. Family caregivers play an essential role in sustaining a stable environment enabling individuals with motor neurone disease (MND) that are technology dependent to live at home. The family caregivers can experi¬ence exceptional burden and significant decline in psychological wellbeing due to MND's rapid and pro¬gressive nature with profoundly debilitating effects and intensive support needs. Dependence on assistive technology adds an additional level of complexity to family caregiving due to the need to learn how to operate and troubleshoot medical devices, train other caregivers, and negotiate appointments with new specialties within the healthcare system. Despite the recognized impact of caregiving for individuals with MND, data are scarce as to effective interventions that provide direct practical and psychosocial supports. Difficulty accessing support may increase psychological distress. As the burden of caring increases due to disease progression and increasing technology dependence, access to existing informal support networks may diminish. Online peer support using virtual modalities is a flexible and low cost form of support. Peers, people who have experienced the same health problem and have similar characteristics as support recipients, can be a key source of emotional, informational, and affirmational support. Peer support improves psychological well-being of caregivers of people with conditions such as dementia, cancer, and brain injury. Although peer support programmes for family caregivers of people with MND exist, data as to their efficacy are limited. Therefore, we have developed an online peer support programme, completed beta and usability testing and now propose to test the effect on caregiver psychological wellbeing and caregiver burden. Aim/research question(s) Overall aim: to determine the efficacy of a 12-week online peer support programme on family caregiver psychological health and caregiver burden. Primary research question: What is the effect of the online peer support programme on psychological distress measured using the Hospital Anxiety and Depression Scale (HADS)? Secondary research questions: What is the effect on positive affect, caregiver burden, caregiving mastery, caregiving personal gain, and coping? How do participants use the programme (fidelity and reach)? What is the perceived usability and acceptability? Methods The investigators will conduct a parallel group randomised controlled trial with participants allocated to 12-week access to the online peer support programme or a usual care control group. The investigators will enrol family caregivers of an individual with MND who is referred for consideration or receiving any of the following assisted ventilation cough assist gastroscopy and enteral feeding i.e., entering King's clinical staging Stage 4A: nutritional support; or Stage 4B: respiratory support [51]: The 12-week peer-to-peer support programme entails: audio, video, or text private messaging; synchronous weekly chat; asynchronous discussion forum; and informational resources. The investigators will collect demographic and caregiving data including the Caregiver Assistance Scale and Caregiving Impact Scale, and caregiver measures (HADS, Positive and Negative Affect Schedule, Zarit Burden Interview, Pearlin Mastery Scale, Personal Gain Scale, Brief COPE) at baseline and programme completion. The investigators will download use of online peer support programme features, assess usability, and conduct semi-structured interviews to explore acceptability using the Theoretical Framework of Acceptability. To test for a medium size effect (d=0.5), at 5% level of significance (2-sided) with power 80%, 64 participants are required in each arm (128 total). Adjusting for 20% attrition requires 154 participants. Proposed findings The proposed study will demonstrate the effect of a online peer support programme on psychological distress, positive affect, caregiving burden, mastery, personal gain and coping. Data on programme fidelity will enable the investigators to objectively assess acceptability and interpret study results. Data on usability and acceptability will inform future scalability of the online peer support programme outside of the trial both nationally and internationally, and to other family caregiver populations.

The goal of this interventional non-pharmacological study is to evaluate, using a multimodal approach, the progression of autonomic and sensory involvement in in amyotrophic lateral sclerosis (ALS) patients enrolled within 18 months from motor onset and its relationship with the progression of overall clinical disability. The main questions it aims to answer are: Is autonomic dysfunction at diagnosis associated with disease progression and survival in patients with Amyotrophic Lateral Sclerosis ? Can we identify in the skin biomarkers to be used as reliable measures of disease progression and to apply in future clinical trials for patient stratification and to assess response to drug treatment ? Participants at time 0 will receive a full clinical and instrumental examination and a blood sample testing to check inclusion and exclusion criteria, genetic screening for the most common genes associated with ALS (SOD1, FUS, TARDBP and c9orf72), questionnaires about clinical characteristics, quality of life, pain and a multidomain battery of neuropsychological tests, multimodal assessment of the autonomic nervous system including skin biopsy for morphological study. At follow-up we'll perform clinical scales and skin biopsy. Researchers will compare results from ALS patients with data obtained from a population of age and sex matched healthy subjects.

Amyotrophic lateral sclerosis (ALS) is a degenerative neurological disease that causes progressive motor disability and is life threatening within a few years. The severity of the disease, the progressive loss of autonomy that leads to dependence on family and caregivers, and the lack of effective treatment sometimes leads patients to a loss of hope and to dark thoughts. The prevalence of suicidal ideation is high, with more than one third of people with ALS experiencing it. The psychological suffering of patients is often associated with that of their caregivers. The evaluation of the patients' feeling of being a burden has rarely been addressed in previous studies in ALS on the notion of burden. In this work, the investigators wish to evaluate the patient's ideas of death by also taking into account the caregiver's burden and the patient's feeling of being a burden. They wish to better understand this difficult experience by refocusing the study on the patient himself, which has rarely been addressed in studies on ALS and the notion of burden. By working on the caregiver's burden, both from the caregiver's point of view and as perceived by the patient, the investigators hope to find avenues of intervention and define actions that could help patients and their families and improve the quality of life of the patient-caregiver couple.