View clinical trials related to Amyotrophic Lateral Sclerosis.
Filter by:This is a prospective study in a cohort of about 45 patients with ALS participating in the Neurosense PrimeC drug study (NCT05357950). This study aims to evaluate the correlation between oculometric measures and clinical endpoints. Subjects will be evaluated every 2 months during a time period of 18 months. The evaluations will include ALSFRS-R examination, as well as an oculometric evaluation for eye movements.
The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).
This study will try to understand the difference in brain structure between ALS patients and healthy people of similar age. ALS is a condition affecting the nervous system with disruption of the brain networks. This study aims to understand these disruptions and determine their significance in ALS.
This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.
Autoimmune encephalitis (AE) and paraneoplastic neurological syndromes (PNS) are rare disease that could be difficult to diagnose. So it necessary to obtain numerous sample from different disease to develop more specific diagnosis kit It could be possible through the characterisation of new genetic biomarkers.
Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disorder with no effective disease-modifying therapies at present. The disease is sporadic in 90 % of the ALS patients. Up to 40 % familial ALS cases and up to 25% of familial frontotemporal dementia (FTD) are caused by autosomal dominant GGGGCC hexanucleotide repeat expansions in the C9orf72 gene. The presymptomatic phase of the disease represents a unique opportunity to evaluate mechanisms of disease propagation, characterise patterns of anatomical spread, validate staging systems and appraise the comparative sensitivity profile of emerging imaging modalities. Very few spinal cord imaging studies currently exist in ALS despite their potential to characterise both the lower and upper motor neuron components of the disease. This prospective longitudinal study of asymptomatic and symptomatic c9orf72 hexanucleotide carriers will use a purpose-designed spinal and brain imaging protocol and comprehensive clinical, genetic, electrophysiological and neuropsychological profiling. Newly developed imaging techniques such as spinal cord NODDI, spinal fMRI, quantitative thoracic cord imaging will be implemented in addition to established spinal cord and brain imaging techniques.
69 subjects with ALS will be enrolled in the study and randomized at a 2:1 ratio to receive the study drug or placebo tablets. Randomization sequences will be in random block sizes and stratified for ENCALS risk category [high risk ≥ -4.5 vs. low risk < -4.5], and for background ALS treatment (riluzole and/or edaravone and/or sodium phenylbutyrate and/or taurursodiol) vs. no background ALS treatment. All subjects will be administered the drug/placebo twice daily (BID), two tablets each time, for 6 months. Subjects will be allowed to receive standard of care (SOC) treatment of approved products (i.e., riluzole and edaravone). Additionally, subjects will be allowed to receive treatment with off-label sodium phenylbutyrate and taurursodiol, which are accepted for ALS treatment. Subjects will be evaluated every 2 months for safety, tolerability (adverse events, safety laboratory, vital signs, ECG, withdrawal rates and reasons) and efficacy (e.g. biomarkers, clinical outcomes (ALSFRS-R and SVC, quality of life and survival). All subjects who complete the 6 months dosing will be switched to the active arm for a 12-month open label extension (OLE).
The purpose of the HSP Sequencing Initiative is to better understand the role of genetics in hereditary spastic paraplegia (HSP) and related disorders. The HSPs are a group of more than 80 inherited neurological diseases that share the common feature of progressive spasticity. Collectively, the HSPs present the most common cause of inherited spasticity and associated disability, with a combined prevalence of 2-5 cases per 100,000 individuals worldwide. In childhood-onset forms, initial symptoms are often non-specific and many children may not receive a diagnosis until progressive features are recognized, often leading to a significant diagnostic delay. Genetic testing in children with spastic paraplegia is not yet standard practice. In this study, the investigators hope to identify genetic factors related to HSP. By identifying different genetic factors, the investigators hope that over time we can develop better treatments for sub-categories of HSP based on cause.
Amyotrophic lateral sclerosis (ALS) is a rare neuromuscular disease that occurs in adults. It is characterized by a progressive degeneration of the first and second motor neurons leading to muscle failure. In its spinal form, ALS manifests by a progressive worsening of limb involvement, whereas the bulbar form presents with swallowing disorders, dysarthria and feeding difficulties. Respiratory impairment is the most serious feature of ALS. Phrenic nerve damage causes diaphragmatic weakness, which inevitably leads to chronic restrictive respiratory failure. At the stage of symptomatic nocturnal or diurnal alveolar hypoventilation, non-invasive ventilation (NIV) prolongs survival while improving quality of life by relieving respiratory symptoms. The indication for the initiation of NIV is based on the appearance of respiratory symptoms but also on the demonstration of diaphragmatic insufficiency. A quarterly follow-up of diaphragmatic function has been recommended by the French Health Authority since 2006. It is based on functional respiratory explorations (VC in sitting and lying position, measurement of maximal inspiratory pressure) and screening for diurnal or nocturnal hypoventilation with the measurement of PaCO2 or the nocturnal recording of SpO2. Access to these examinations remains limited and they are sometimes complex to perform (in particular issues with mouth occlusion during respiratory manoeuvres in case of bulbar damage). Thus, only 60% of patients undergo a complete evaluation. Moreover, these explorations are only late markers of diaphragmatic dysfunction, and it has recently been shown that they do not correlate with histological diaphragmatic amyotrophy. The development of new, reliable, and easily available tools for the evaluation of diaphragmatic function, and that are capable of detecting diaphragmatic insufficiency early in the course of the disease, are therefore necessary. Such tools would make it easier to implement NIV at the optimal time, preventing episodes of acute respiratory distress. Recently, diaphragmatic ultrasound has appeared in the ICU as a new tool for assessing diaphragmatic function. It has the advantage of being highly available, inexpensive, non-irradiating, quick to perform, reproducible and very sensitive and specific for the diagnosis of diaphragmatic dysfunction. In ALS, few studies have investigated the contribution of ultrasound for the diagnosis or follow-up of diaphragmatic dysfunction. In addition, no study so far has compared diaphragmatic ultrasound to complete pulmonary function test (PFT) data or to direct measurement of diaphragmatic pressure (Pdi). Very few publications report the how the diaphragm changes on ultrasound imaging during the disease. Moreover, these studies do not analyse the interest of diaphragmatic ultrasound in the prediction of progression towards respiratory failure with respiratory support, or death. Finally, these studies use different ultrasound measurements of the diaphragm (stroke, thickness, thickening fraction, and thickening fraction ratio, among others) rather than a simple, consensual parameter. The aim of this study is to describe the evolution of diaphragmatic ultrasound parameters, to identify the parameter that best correlates with other respiratory measures (PFT, PaCO2, nocturnal oximetry) and to determine the prognostic value of diaphragmatic ultrasound in predicting the initiation of NIV or death at 6 and 12 months.
This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.