The trial is testing an oral capsule called NB-4746 and also includes the already approved medicine Riluzole. Some participants will receive a dummy pill, known as a placebo, so that researchers can compare how the real medicines work. The main goal of the study is to find out whether the new drug is safe for people with the disease, meaning it does not cause unacceptable side effects.

People who join the study will take the assigned capsule each day and will visit the clinic several times for simple check‑ups. During these visits doctors will look at basic health information such as blood pressure and heart rate, draw small amounts of blood for laboratory testing, and perform a quick heart rhythm test. The early part of the study lasts about four weeks, and later phases continue for a longer period to keep monitoring safety.

The treatment involves using inhalation powder contained in hard capsules that are breathed in through a special inhaler device. Some participants will receive PHENOGENE-1a, while others will receive placebo through the same type of inhaler. The maximum daily amount of medication is 64.8 mg, and treatment continues for 24 weeks.

During the study, participants will need to continue taking their regular ALS medication called riluzole. The study will monitor various aspects of the disease, including breathing function and overall physical abilities. All participants must be between 18 and 75 years old and have been diagnosed with ALS within the past 24 months.

Participants in the study will be given either pridopidine in a capsule form or a placebo. The study uses a double-blind method, which means that neither the participants nor the researchers know who is receiving the active medication or the placebo during the course of the trial. This is done to ensure the results are as accurate as possible.

During the trial, participants will be monitored over a period of time to observe changes in their physical abilities and overall health. This includes tracking how well they can speak and their ability to breathe effectively. The study also monitors any side effects or changes in health to ensure safety throughout the process.

Participants in the study will receive different doses of VTx-002 to see how the body reacts. During the course of the study, medical professionals will monitor various health indicators, including MRI scans to look at the structure of the brain and spine, and assessments of peripheral neuropathy, which refers to nerve damage that can cause numbness or weakness in the hands and feet. Other measurements may include tracking lung function through slow vital capacity, which is a way to measure how much air the lungs can hold during a normal breath.

During the course of the study, the way the body processes the medication, known as pharmacokinetics, and the drug’s effects on the body, known as pharmacodynamics, will be observed. Researchers will also look at NfL, which is a specific protein found in the blood and cerebrospinal fluid—the clear liquid that surrounds the brain and spinal cord—that can indicate nerve cell damage. The study will monitor how the disease progresses and look at how the treatment affects physical functions and survival over a period of time.

The purpose of this study is to evaluate the safety and tolerability of LTX-002 when given to adults with Amyotrophic Lateral Sclerosis. The study will look at different dose levels of the medication to understand how well it is tolerated by participants. During the study, participants will receive multiple doses of either LTX-002 or placebo through injections into the spinal canal. The study team will monitor participants closely through regular visits and various tests.

Throughout the study, several measurements will be taken to assess safety and how the medication moves through the body. These include checking for any side effects or adverse events, performing blood and urine tests, taking heart tracings using electrocardiograms, measuring vital signs like blood pressure and heart rate, and conducting physical and neurological examinations. The study will also measure the levels of LTX-002 in the cerebrospinal fluid, which is the liquid surrounding the brain and spinal cord, and in the blood to understand how the medication is processed by the body. Participants may need to take the medication over several doses and will be followed for a period of time after the last dose to monitor their health and any effects of the treatment.

The purpose of this study is to find out how the body takes up, breaks down, and removes SAR443820 after it is given by mouth. Researchers want to understand how much of the medicine gets into the blood, how the body changes the medicine into other substances, and how these substances leave the body through urine and stool. They will also identify the main ways the body processes the medicine and the routes it uses to remove it from the body.

The study will involve healthy male adults between 18 and 55 years of age. Participants will receive a single dose of the medicine as a liquid taken by mouth. After taking the medicine, samples of blood, urine, and stool will be collected over a period of time to track how the medicine moves through the body. During the study, participants will have regular check-ups that include physical examinations, blood tests, and heart monitoring to ensure safety. The study will be conducted at a single center and is expected to last for a limited treatment period.

Participants in the trial will receive either masitinib or a placebo, both in combination with their standard ALS treatment. Masitinib is a type of medication known as a tyrosine kinase inhibitor, which works by blocking certain proteins that can contribute to disease progression. The trial will last for a period of 48 weeks, during which participants will take the medication orally in the form of coated tablets. The study will monitor changes in the participants’ condition and any side effects they may experience.

In addition to masitinib, some participants will continue taking another medication called Riluzole, which is commonly used in ALS treatment. The trial will compare the outcomes of those taking masitinib with those taking a placebo to determine the added benefits of masitinib. The goal is to provide more information on how masitinib can be used to improve the quality of life for people living with ALS.

The purpose of the study is to use this new MRI technique to identify and describe metabolic changes in patients with ALS and TIA. Participants in the study will receive an injection of Hyperpolarized [1-13C]pyruvate, which will help highlight certain metabolic processes in the brain during the MRI scan. This approach aims to provide a clearer picture of how these diseases affect brain function.

Throughout the study, participants will undergo MRI scans after receiving the injection. The study will monitor how the injected substance interacts with the brain’s metabolism, focusing on how it converts to other substances like bicarbonate, lactate, and alanine. This information will be gathered alongside clinical examinations and patient interviews to better understand the impact of ALS and TIA on brain health.

Participants in the study will receive either the ION363 treatment or a placebo, which is a substance with no active medication. The treatment is given through a method called intrathecal use, meaning it is injected into the space around the spinal cord. The study will monitor participants over time to see how the treatment affects their ability to function and survive. This includes looking at changes in their physical abilities and overall quality of life.

The study will also measure various biological markers, such as levels of certain proteins in the blood and spinal fluid, to understand the treatment’s impact on the disease. The trial is expected to continue for several years, allowing researchers to gather comprehensive data on the long-term effects of ION363 in treating FUS-ALS. Participants will be closely monitored throughout the study to ensure their safety and to collect detailed information on how the treatment affects their condition.

Participants in the study will be randomly assigned to receive either ILB or Riluzole, and some may receive a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment. This helps ensure that the results are not biased. The study will last for a period of 48 weeks, during which participants will have regular check-ups to monitor their health and the effects of the treatment. These check-ups will include various tests and assessments to track changes in the participants’ condition.

The study aims to gather information on the safety and effectiveness of ILB, as well as its impact on certain biological markers related to ALS. By comparing the results from the ILB group with those from the Riluzole group, researchers hope to determine if ILB can offer a new and effective treatment option for people living with ALS. The study will also collect data on any side effects experienced by participants to ensure the safety of the treatments being tested.

Participants in the study will be randomly assigned to receive either the combination therapy or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This approach helps ensure that the results are unbiased. The treatment will be administered orally, and participants will be monitored over a period of time to observe any changes in their condition, particularly focusing on their ability to perform daily activities and their overall quality of life.

The study aims to gather valuable information on how the combination of NAC, EH-301, and Riluzole affects the progression of ALS. By using the ALS Functional Rating Scale-Revised (ALSFRS-R), researchers will evaluate changes in participants’ physical abilities. Additionally, the study will look at other factors such as muscle strength, respiratory function, and survival rates. The findings from this study could contribute to developing more effective treatments for individuals living with ALS.

Participants in the study will be randomly assigned to receive either masitinib with Riluzole or a placebo with Riluzole. A placebo is a substance with no active medication, used to compare the effects of the actual drug. The study is designed to be “double-blind,” meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo, ensuring unbiased results. The study will last for a period of 48 weeks, during which participants will take the medication orally in the form of coated tablets.

Throughout the study, participants will be monitored for changes in their condition, focusing on their ability to perform daily activities and any side effects they may experience. The main goal is to see if there is an improvement in the symptoms of ALS when masitinib is added to the standard treatment with Riluzole. This research aims to provide valuable insights into potential new treatment options for those living with ALS.

The purpose of this study is to evaluate the safety and tolerability of AMT-162 in adults with SOD1-ALS. Participants will receive a single dose of the treatment administered directly into the spinal fluid, a method known as intrathecal use. The study will monitor participants for any side effects and changes in their health over time. Researchers will also explore how the body responds to the treatment, including any immune reactions and changes in specific proteins related to ALS.

The study will take place over several years, allowing researchers to gather comprehensive data on the long-term effects of the treatment. Participants will undergo regular health assessments, including laboratory tests and heart monitoring, to ensure their safety throughout the trial. The ultimate goal is to determine if AMT-162 can be a safe and effective treatment option for individuals with SOD1-ALS.

The purpose of the study is to assess how effective acetyl-L-carnitine is in slowing down the loss of self-sufficiency in people with ALS. Participants will be randomly assigned to one of the treatment groups or the placebo group. The study will last for 48 weeks, during which participants will be monitored to see how well they maintain their ability to perform daily activities, such as swallowing, cutting food, handling utensils, and walking. The study will also look at changes in quality of life and other health markers over this period.

Throughout the study, researchers will collect information on any side effects experienced by participants and measure various health indicators to understand the treatment’s impact. The goal is to determine whether acetyl-L-carnitine can help people with ALS maintain their independence and improve their quality of life. This research could provide valuable insights into new ways to manage ALS and support those living with the condition.

Participants in the study will receive either VHB937 or a placebo through an intravenous infusion, which means the treatment is given directly into a vein. The study will last for 40 weeks, during which participants will be monitored for changes in their condition. After this period, there will be an open-label extension, where all participants may receive VHB937, allowing researchers to gather more information about the treatment’s long-term effects.

The main goal of the study is to compare the effectiveness of VHB937 against a placebo in terms of survival without permanent assisted ventilation and overall function. Researchers will also look at other factors, such as changes in muscle strength, breathing capacity, and quality of life. Safety and any side effects will be closely monitored throughout the study to ensure the well-being of all participants.

The purpose of this study is to see how well Triumeq works in improving the overall survival of people with ALS over a period of 24 months. Participants will be randomly assigned to receive either Triumeq or the placebo. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are not influenced by expectations or bias.

Throughout the study, participants will have regular check-ups to monitor their health and assess the effects of the treatment. These check-ups will include evaluations of daily functioning, respiratory function, and overall quality of life. The study will also track any side effects or adverse events that may occur. The goal is to gather comprehensive information on how Triumeq affects people with ALS and to determine its safety and effectiveness in treating this condition.

Participants in the study will receive either the cannabis extract or a placebo. The study will be conducted over a period of time, during which participants will take the treatment as an oral solution. The study aims to assess the safety and tolerability of the cannabis extract, as well as its impact on the quality of life, which will be measured using a health survey. Additionally, the study will look at how the treatment affects caregiver distress and patient-reported outcomes.

The study will also explore specific improvements in each disease group. For Alzheimer’s Disease, it will assess cognitive functions and agitation. For Amyotrophic Lateral Sclerosis, it will evaluate functional status. For Parkinson’s Disease, it will monitor the progression of symptoms. The results will help determine if the cannabis extract is a beneficial treatment option for these neurodegenerative diseases.

The study will begin with a period where participants’ natural health progression is observed. After this, participants will be randomly assigned to receive either Tofersen or a placebo, which is a solution that looks like the treatment but does not contain the active substance. This part of the study will help researchers understand the effects of Tofersen compared to no active treatment. Participants will be monitored over time to see if they develop symptoms of ALS and to assess any changes in their health status.

Following the initial phase, there will be an open-label extension, where all participants will have the opportunity to receive Tofersen. Throughout the study, researchers will collect information on various health measures, including changes in physical abilities and breathing function. The study aims to provide valuable insights into the potential benefits of Tofersen for individuals at risk of developing ALS due to the SOD1 mutation.

The purpose of the study is to assess the safety and tolerability of ARGX-119 in people with ALS. Participants will be randomly assigned to receive either the treatment or a placebo, which is a substance with no active medication. The study will be conducted in a double-blinded manner, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased. The study will also include an active-treatment extension phase, allowing researchers to gather more information about the treatment’s effects over time.

Throughout the study, participants will undergo various assessments to monitor their health and the effects of the treatment. These assessments will include checking for any adverse events, conducting clinical laboratory tests, and measuring vital signs. The study will also look at how the body processes ARGX-119 and whether the immune system produces any antibodies against it. The trial is expected to continue until 2027, providing valuable insights into the potential benefits and safety of ARGX-119 for individuals with ALS.

The main purpose of this research is to determine if lithium can improve survival rates in ALS patients. The study will specifically look at how long patients survive without developing respiratory problems severe enough to require breathing support. The treatment involves taking lithium capsules by mouth, with doses up to 1200 milligrams per day over a period of 24 months.

During the study, participants will be monitored for changes in their daily functioning, breathing ability, quality of life, and thinking skills. The research team will also track any changes in laboratory measurements and watch for possible side effects. Patients who are already taking riluzole, a common ALS medication, may continue using it if they have been on a stable dose.

The main goal of the study is to assess the safety of the treatment by monitoring any side effects that may occur during the study period. Participants will receive the treatment and be observed for any changes in their condition. The study will also look at how the treatment affects the progression of ALS and its impact on the quality of life of the participants. This will be done by measuring various health indicators and collecting data over a period of time.

Participants in the study will be monitored for several months to see how their condition changes with the treatment. The study will collect information on how the treatment affects the symptoms of ALS and whether it can slow down the progression of the disease. The results will help researchers understand the potential benefits and risks of using human neural stem cells as a treatment for ALS.

Participants in the study will receive multiple doses of NX210c over a period of time, and some may receive a placebo instead. The study will monitor the effects of the treatment on certain markers in the blood and spinal fluid, which can provide information about the disease’s progression and the treatment’s impact. The study will also assess how well participants tolerate the treatment and any side effects they may experience.

Throughout the study, participants will have regular check-ups and tests to track their health and the treatment’s effects. The study aims to gather important information that could help in understanding and potentially improving the treatment of Amyotrophic Lateral Sclerosis in the future.

The study compares PTC857 with placebo in people who have ALS. Participants will receive either PTC857 or placebo for 24 weeks. During this time, patients can continue taking their standard ALS medications, including riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, as long as they were already using these medications before joining the study.

The research will measure how the disease progresses by looking at changes in physical function and survival. The study will also check how safe PTC857 is and how well patients tolerate it. Additionally, researchers will examine the medication’s effects by measuring certain substances in the blood and spinal fluid that may indicate how the disease is developing.

The purpose of this study is to evaluate the safety and tolerability of AP-101 in patients with ALS. The study is divided into two phases: a double-blind phase, where neither the participants nor the researchers know who is receiving the actual medication or a placebo, and an open-label extension, where all participants receive the medication. During the study, participants will receive multiple doses of AP-101 through an intravenous (IV) injection over a period of six months. The study will monitor the participants’ health and any side effects that may occur.

Participants in the study will undergo regular health check-ups, including physical and neurological examinations, to ensure their well-being. The study will also measure certain markers in the blood and spinal fluid to understand how the body processes the medication. This research aims to gather important information that could lead to better treatments for ALS in the future.

The purpose of the study is to assess how AMX0035 impacts the progression of ALS in adult patients. Participants will be randomly assigned to receive either AMX0035 or a placebo, and neither the participants nor the researchers will know who is receiving which treatment. This is known as a double-blind study. Throughout the 48 weeks, participants will have regular visits to the clinic to monitor their health and the progression of the disease.

During the trial, researchers will measure changes in the participants’ abilities to perform daily activities and their overall survival. The study aims to provide valuable information on whether AMX0035 can slow down the progression of ALS and improve the quality of life for those affected by this challenging condition.

Participants in the study will receive either salbutamol or a placebo, which is a substance with no active medication. Salbutamol is administered as an oral solution, meaning it is taken by mouth in liquid form. The study will last for a period of six months, during which the effects of salbutamol on walking ability will be closely monitored. The main goal is to see if there is an improvement in the distance patients can walk in six minutes, a common test used to measure walking capacity.

The study is designed to provide valuable information on the potential benefits of salbutamol for people with ALS, specifically in terms of enhancing their ability to walk. This research could lead to new treatment options that help improve the quality of life for those living with this challenging condition.

Participants in the study will receive either Rituximab or a placebo, in addition to their regular treatment. The study will last for a period of 79 weeks, during which participants will undergo regular assessments to monitor their health and the progression of their symptoms. These assessments will include tests to measure lung function, changes in body weight, and overall survival without the need for a tracheostomy, which is a procedure to help with breathing. The study will also look at the safety of Rituximab by checking laboratory parameters and conducting neuropsychological tests to assess cognitive function.

The goal of this study is to determine if Rituximab can provide additional benefits to patients with ALS by slowing down the progression of the disease and improving their quality of life. The results will help to understand the potential of Rituximab as a treatment option for ALS and its impact on patients’ health and well-being over time.

The purpose of the study is to assess how well dazucorilant works and how safe it is for people with ALS. Participants in the study will be randomly assigned to receive either dazucorilant or a placebo. The study is designed to be “double-blind,” meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are unbiased. The study will take place over several months, with regular check-ups to monitor the participants’ health and any changes in their condition.

Throughout the study, participants will be asked to take capsules of the study medication by mouth. The researchers will track changes in the participants’ ability to perform daily activities and any side effects they may experience. The study aims to provide valuable information about the potential benefits and risks of using dazucorilant for treating ALS, which could lead to new treatment options for this challenging condition.