ALS (Lou Gehrig's disease) clinical trials — recruiting now

The purpose of this study is to collect CSF and blood samples that can be used in future research studies to identify potential biomarkers in blood and cerebrospinal fluid (CSF) collected in Amyotrophic Lateral Sclerosis (ALS) patients.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterised by progressive diffuse muscular paralysis due to the inexorable loss of motor neurons in the primary motor cortex, the corticospinal tract, the brain stem and the spinal cord. Over the course of the disease, when the phrenic motor neurons are involved, diaphragmatic weakness develops, leading to restrictive respiratory failure, which is the main cause of morbidity and mortality. Non-invasive ventilation (NIV) compensates for diaphragm failure and corrects the associated symptoms, and has been shown to prolong patient survival and improve quality of life. Undernutrition is another recognised prognostic factor. Several mechanisms have been described, foremost of which are a state of hypermetabolism and a reduction in food intake secondary to chewing difficulties, dysphagia, a loss of dexterity in the upper limbs, a disturbance in salivary secretion or psychological disorders. In addition, diaphragmatic dysfunction plays a direct role in the onset of undernutrition, as compensatory contraction of the accessory neck muscles increases resting energy expenditure. However, the hedonic sensations triggered by a meal play a role in controlling food intake beyond the simple energy balance between calorie intake and energy expenditure. Olfacto-gustatory sensoriality could therefore play a role in the nutritional status of patients suffering from ALS. Diaphragmatic dysfunction may also influence nutritional status by other mechanisms. For example, the reduction in inspiratory capacity associated with diaphragmatic insufficiency reduces olfaction in a group of tetraplegic patients. Central sensory impairment could exacerbate this phenomenon. Although it is conventionally considered that there are no sensory manifestations during the course of ALS, minor but diffuse abnormalities of the nerves and sensory action potentials have been observed. A central alteration in olfacto-gustatory sensoriality could be part of the neurological manifestations of ALS. In addition, olfactory deficits occur in other neuromuscular diseases with central involvement, such as myasthenia, Parkinson\'s or Alzheimer\'s disease, in the absence of concomitant cognitive or diaphragmatic impairment. Our hypothesis is that impaired olfacto-gustatory function favours the onset of undernutrition in ALS. Current nutritional management consists of ensuring adequate calorie intake by prescribing oral food supplements or inserting a gastrostomy. Taking personalised account of food preferences during dietary advice or of a potential olfacto-gustatory deficit, by reinforcing smells or tastes during food consumption, would be an interesting additional therapeutic avenue for improving patients\' nutritional status, quality of life and prognosis

The purpose of this study is to evaluate how well LY4256984 is tolerated and what side effects may occur in participants with sporadic amyotrophic lateral sclerosis (ALS). The study drug will be administered intrathecally (IT) into the spine. Blood tests will be performed to check how much LY4256984 gets into the bloodstream and how long it takes the body to eliminate it.

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.

The purpose of this study is to determine if treatment with a single dose of RE104 for Injection reduces depressive symptoms or depressive symptoms mixed with anxiety symptoms in participants with Adjustment Disorder due to cancer or other illnesses such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Parkinson's Disease (PD) or Idiopathic Pulmonary Fibrosis (IPF) as compared to active-placebo.

This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms

Neurodegenerative diseases are debilitating conditions characterized by chronic inflammation, leading to dysfunction of both the non-neuronal cellular components of the central nervous system and peripheral blood immune cells. Thus, it is crucial to develop an innovative therapeutic strategy that not only effectively contrast neurodegeneration but also aims to reduce inflammation. The overall aim of the study is to provide a preclinical in vitro demonstration of the immunomodulatory and pro-regenerative potential of the human amniotic mesenchymal stromal cell (hAMSC) secretome in counteracting neurodegeneration. This potential will be evaluated in three-dimensional in vitro models of neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) and chronic demyelinating disease (multiple sclerosis - MS). To this end, the study includes sample collection from patients without pharmacological treatment and without medical devices. Patients diagnosed with ALS, patients diagnosed with MS, and healthy volunteers will be recruited to collect blood samples and skin biopsies. Patient-specific and control organoid platforms, mimicking cellular heterogeneity and tridimensional interactions within the central nervous system including the inflammatory compartment, will be developed to be used as a valuable tool to investigate the in vitro efficacy of the hAMSC secretome.

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.

The goal of this clinical trial is to learn about the effect of communicative interaction on verbal communication in people with amyotrophic lateral sclerosis (ALS) and their caregivers. The question is, What are the effects of communicative interaction on verbal communication in people with ALS when they interact with their caregivers and does this change over time? Participants will read words and sentences while they are interacting with their caregivers.

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.

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

The aim of this study is to assess the impact of a probiotic formulation on participants with ALS-FTDSD. It is hypothesized that participants given the probiotics will have different lipid profiles compared to participants receiving the placebo at different time points.

The goal of this study is to learn from discordant twins affected by Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia the contribution of genetic background versus environmental exposure. The main questions it aims to answer is: * How far does the genetic background explain the onset of ALS/FTD in discordant twins? * Which environmental factors and events occurring in post-fetal life influence the onset or progression of this neurodegenerative condition? Participants with ALS and/or FTD with a monozygotic or dizygotic twin willing to contribute to this research will be followed up for two years by specialized Motor Neuron Disorders centers in Italy, donating biological specimen for -omic sciences analysis, and checking out if prodromal signs/symptoms of these two conditions will develop during time.

This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients.

This is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of nasal drop exosomes derived from human umbilical cord blood mesenchymal stem cells (hUC-MSC-sEV-001) in amyotrophic lateral sclerosis.

1. Describe the distribution of ALS in mainland China, to explore the differences in the number of ALS in different times, regions and populations in order to further explore the causes affecting the distribution of ALS; 2. To investigate the cause of ALS in mainland China in the crowd disease development process and the corresponding characteristics change ; 3. To explore the effect of prognosis of ALS;

Neurodegenerative disorders (NDDs), such as Parkinson¿s disease (PD), Alzheimer¿s disease (AD), Frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS) are characterized by aggregation and intracellular accumulation of misfolded proteins, which are believed to play a key role in synaptic dysfunction and neuronal death. Protein structural complexes in biofluids have been proposed to mirror pathological conditions suggesting their use as biomarkers for NDDs characterized by protein aggregation. In this framework, we plan to: i) collect a large cohort of NDD and prodromal patients and healthy subjects using standardized clinical and genetics procedures; ii) apply a novel method based on genomics, proteomics and bioinformatic analysis to map protein complexes in biofluids; iii) identify novel circulating biomarkers and correlate them to genetic profiling and disease endophenotypes, and; iv) validate the biological properties in human brain tissue and dopaminergic cultures.

This study investigates whether an individualized physiotherapy program, tailored to each patient's specific motor deficits, can better support physical function compared with usual care physiotherapy in people with ALS. The individualized program is guided by diagnostic assessments using a robotic leg press system, which helps identify strengths and weaknesses in muscle function and movement control. Participants will receive either individualized physiotherapy or standard physiotherapy and will be followed for 12 months. The aim of the study is to improve physiotherapy strategies for people with ALS in a safe and patient-centered manner.

The purpose of this 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is two to three years. The patient physical and psychological sufferings in ALS are immense, and apart from Riluzole, there is no effective treatment. Care of advanced ALS have an estimated cost of 4-8 million NOK per year. Perhaps the most challenging topic of ALS care is the decision to extend ventilation support into the stages of disease that require treatment both during day and night. In these cases, treatment is clearly life-sustaining and although quality of life may be maintained, the burden of caregiving imposed upon family or health care workers is huge, regardless of tracheostomy (TIV) or non-invasive (NIV) modality. The present study is a longitudinal questionnaire study in Norway measuring overall quality of life, health-related quality of life, and disease-specific quality of life in ALS patients, partners and children before and after the introduction of life sustaining ventilation support. The investigators aim to increase the knowledge on how life-sustaining ventilation support with NIV or TIV affects the quality of life in ALS patients, life partners and children. The results from the study may provide crucial information for clinicians and patients on one of the most difficult ethical issues of ALS treatment. The investigators anticipate that this information will facilitate a shared decision making processes, weighing benefits and disadvantages in a wider perspective.