View clinical trials related to Motor Neuron Disease.
Filter by:The purpose of this study is to investigate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of TCD601 (siplizumab) in newly diagnosed adult ALS patients.
Amyotrophic lateral sclerosis (ALS) is a rapidly progressing and disabling disease with the majority of patients dying 3-5 years after symptom onset. Given the high symptom burden, many patients and its caregivers are highly distressed. However, few programs to improve mental health for this patient group exist, and the sparse research implies that programs effective in other medical conditions may not be feasible in ALS patients. Therefore, it is highly needed to involve ALS patients, caregivers and medical staff as contributors into the development of such programs to meet the needs they really have. The envisaged project has two aims: First, the investigators want to examine whether and how it is possible to involve ALS patients in the whole research process despite rapid disease progress and severe functional impairments. Second, the investigators are interested in how contributors (i.e., patients, caregivers and medical staff) would compile a concrete psychotherapeutic program, i.e., how they set priorities in terms of format, content and treatment techniques of such a program. The investigators will closely collaborate with contributors across the whole project in designing the research process, planning assessment as well as interpreting and disseminating the findings. At the end of the study, the investigators will gather contributor feedback on their experience with the participatory approach. Results will provide important information on how ALS patients can be effectively involved in psychosocial intervention research. Identified priorites regarding psychotherapeutic programs will serve as concrete starting points to develop and test a disease-specific program within a subsequent study.
The PRIME Study is a first-in-human early feasibility study to evaluate the initial clinical safety and device functionality of the Neuralink N1 Implant and R1 Robot device designs in participants with tetraparesis or tetraplegia. The N1 Implant is a skull-mounted, wireless, rechargeable implant connected to electrode threads that are implanted in the brain by the R1 Robot, a robotic electrode thread inserter.
Questionnaires and scales used to assess the clinical status and quality of life of patients with amyotrophic lateral sclerosis (ALS) are an important tool to monitor the disease progression and current needs of patients. The use of these tools (and in particular their combination) allows to cover the whole spectrum of potential patient difficulties and thus significantly facilitates the process of individualisation and optimisation of care. The aim of the study was to create and validate the Czech language versions of the following questionnaires or scales: (1) ALSFRS-R (ALS Functional Rating Scale - Revised Version) and (2) ALSFRS-EX (EXtended, i.e. extended, version of the same scale), both in the self-assessment version (incl. (3) the ALSAQ-40 (ALS Assessment Questionnaire including 40 questions), (4) the DYALS (Dysphagia in ALS), and (5) the Borg Dyspnoea Rating Scale. All questionnaires were translated using the forward-backward translation method. The scales and questionnaires were administered to ALS patients repeatedly at one-week intervals, first in writing during routine patient follow-up at the Neuromuscular Centre of the University Hospital Brno, and during repeated administrations by telephone.
To determine whether Spatially Distributed Sequential Functional Electrical Stimulation is more effective than Standard Electrical Stimulation During Functional Electrical Stimulation in Upper Motor Neuron Patients
The progressive loss of physical functioning resulting from ALS leads also to high psychosocial burden for those affected, and organizational challenges related to medical care and aids. A multidimensional and -professional care is advised in order to meet the complex requirements of this disease. In Germany, medical care structures may not fulfil these high requirements, since non-medical services such as psychological support or social counselling are not regularly included in care procedures for ALS patients. Specialised palliative care is not a standard and still commonly restricted to the last weeks of life. Additionally, it is well known that caregivers of ALS patients are highly burdened, but there is a lack of support services for them. By means of a cross-sectional, multicentre survey, we aim to investigate patients' and caregivers' perception of medical care for ALS, provided in Germany - with particular regard to psychosocial and palliative aspects. The extent to which physical, psychological, social, spiritual, practical and informational needs are subjectively met will be assessed and correlations with mental wellbeing, subjective quality of life, attitudes towards life-sustaining measures and physician-assisted suicide, as well as caregiver burden will be examined. Currently, study planning (questionnaires and ethical approval) is already completed and recruitment was started. The study aims to recruit 500 participants from nationwide ALS-centres. Cooperating ALS-centres will be recruited via the German Network for Motoneuron Diseases (MND-Net), of which our centre is a member. It is intended to provide data-based starting points on how care of ALS patients and their caregivers can be improved in Germany, in line with their needs.
A Not for Profit Phase II Study to Evaluate Safety, Efficacy and Biomarkers secondary endpoints of Human Neural Stem cell intracerebroventricular transplantation in amyotrophic lateral sclerosis patients: a randomized, placebo controlled, triple blind study. This is an approximate 24-months study (PHASE B) consisting, per patient, of a 30-day screening period, 12-month enrollment and follow up period. A preliminary 3+3 dose-escalation open-label phase (PHASE A) will be performed in order to test the toxicity of the two proposed cell doses. The study will be stopped when all the subjects included in the treatment period complete the study visits. The study uses an ATMP, for that reason all the patients follow up will be prosecuted long life.
1. Background Cognitive screening procedures via performance-based tests represent an essential, albeit preliminary, element within the diagnostic and interventional process as addressed to patients with chronic neurological disorders. Furthermore, in these populations, cognitive screening measures are often employed as outcomes in epidemiological settings, as well as endpoints in clinical trials. Therefore, cognitive screeners need to possess robust clinimetric and clinical usability properties - the investigation of which must be country-specific (i.e., specific to each language and culture). The need for such clinimetric and feasibility studies is even more true if referred to telephone-based cognitive screening (TBCS) procedures - which, until recently, have been mostly neglected in Italy, despite having the potential to bring clear benefits to clinical practice and research. In fact, TBCS techniques allow, through the use of a very widespread, accessible and easy-to-use telecommunication medium, to break down the geographical, logistical, socio-demographic and organizational barriers that make it difficult and/or prevent 1) access to these clinical services and 2) the continuity of their provision, as well as the creation and completion of 3) large-scale epidemiological studies and 4) decentralized clinical trials. However, although some TBCS tests have recently been developed and standardized in Italy, their clinimetric properties and clinical usability in populations with chronic neurological disorders have not yet been investigated. Furthermore, currently, the "paper-and-pencil" version of the international gold-standard for TBCS procedures . i.e. the Telephone Interview For Cognitive Status (TICS), which has been recently standardized in this country - is not available within the Italian scenario. In fact, although the feasibility of a de visu version of the TICS (i.e., In-Person TICS; IP-TICS) has been demonstrated in this country, an actual standardization of this test has not yet been implemented to date. Such a tool would, however, allow flexible use of screening assessments, regardless of the delivery method, both in clinical and experimental contexts. 2. Aims The present study primarily aims to provide exhaustive evidence regarding the psychometric, diagnostic and both cross-sectional and longitudinal clinical usability of TBCS that are currently available within the Italian scenario in populations with chronic neurological disorders. Secondly, this study aims to derive, in normotypical Italian subjects, 1) normative data for the IP-TICS and 2) the conversion algorithms between the latter (and other widely used "paper-and-pencil" screeners ) and the TICS. 3. Methods The study is monocentric, observational, prospective. Over a period of 3 years, patients who have already undergone an in-person cognitive screening session within 6 months prior to recruitment and falling under the following diagnostic categories will be recruited: 1) amyotrophic lateral sclerosis (N≥88); 2) Alzheimer's disease (N≥66); 3) Lewy body dementia (N≥30); 4) frontotemporal dementia (N≥30); 5) chronic cerebrovascular disorders (N≥66). Furthermore, N≥287 normotypical subjects representative of the Italian population will be recruited. The following TBCS tests will be administered to patients: 1) TICS; 2) Telephone-based Frontal Assessment Battery; 3) Telephone Language Screener; 4) Telephone-based Verbal Fluency Battery; 5) ALS Cognitive Behavioral Screen-Phone Version. Additionally, patients will undergo a functional evaluation using caregiver-report questionnaires evaluating instrumental and non-instrumental skills of daily living and behavioral changes. Normal subjects will instead be administered: 1) TICS; 2) IP-TICS; 3) Mini-Mental State Examination (MMSE); 4) Montreal Cognitive Assessment (MoCA). In patients, telephone follow-ups are expected after 6, 12 and 18 months. Statistical analyses will be carried out aimed at 1) the detailed study, in patients, of the psychometrics, diagnostics and cross-sectional/longitudinal clinical usability of the aforementioned TBCS test, as well as at 2) the derivation, in normotypical subjects, of the normative data of the IP-TICS and MoCA Memory Index Score (MIS), as well as the conversion algorithms between TICS and IP-TICS/MMSE/MoCA.
Diagnosis of Amyotrophic Lateral Sclerosis (ALS) is considered a traumatic life event for both the patient and their next-of-kin/carers, due to the lack of treatment. Clinical Trials can offer pioneering treatment to reduce the impact of the disease and improve future treatments worldwide. Research protocols may involve routine diagnostic and/or therapeutic procedures which the patients may be already aware of and, therefore, expecting specific sensations. These could compromise participation or drop-out rate. Despite everything, participation in a clinical trial can guarantee continuity of care also thanks to the execution of these same procedures, through preferential access compared to other patients. Aim of this study is to investigate the unpleasant sensations perceived by ALS patients during procedures in clinical trials. Analysing what type of pain/discomfort frightens patients during diagnostic and/or therapeutic procedures, including the different methods of administration of the study drug. Provide data to implement effective therapy and offer constant patients support throughout ALS specific and needed procedures. Evaluate if this support could influence adherence rate of ALS patients to conduct clinical trials as required. Provide information for future studies to create an ALS Clinical Trials multiple-retention-factors adherence scale. Create and implement an ALS-specific pain scale accounting for its impact on daily activities, aiding an interdisciplinary approach of pain management. Identify the best pain management strategies and compliance techniques to address ALS, not merely in clinical trials. Provide the best individualized care for ALS patients improving their quality of life and mental state. This is a descriptive phenomenological study and data will be analyzed according to Sundler's method. Based on the experience of the researchers and the recommendations proposed by Sandelowski, a total of 20 interviews are estimated in order to reach the theoretical saturation per category of reference. Data collection will be carried out through in-depth semi-structured interviews recorded (13 open-ended questions after the execution of the procedures).
Substantial variability exists in the onset, and rate of degeneration across individuals with Motor Neurone Disease (MND) or Amyotrophic Lateral Sclerosis (ALS). This variability requires biomarkers that accurately classify and reliably track clinical subtypes as the disease progresses. Degeneration occurs in the brain and spinal cord, however, non-invasive diagnosis of spinal cord function remains highly challenging due to its unique alignment in spine. Disruption of complex spinal and cortical circuits that transmit and process neural signals for position sense and movement has not been adequately captured in the neurophysiological profiling of ALS patients. The overarching aim of this study is to reveal and quantify the extent of change in the sensorimotor integration and its potential contribution to network disruption in ALS.