AMYOTROPHIC LATERAL SCLEROSIS

This early study tests a single injection of a gene therapy called NTF001 in 12 people with ALS. The therapy aims to deliver a protein that supports nerve cell health. The main goal is to check safety, but researchers will also look for signs of benefit over 52 weeks.

This early-stage study tests the safety of acamprosate, a drug used for alcohol dependence, in 30 adults with ALS who have a specific mutation in the C9orf72 gene. Participants take the pill three times daily for 24 weeks and undergo regular checkups, breathing tests, and spinal …

This early-stage study tests whether a new drug called QRL-201 is safe and tolerable for people with ALS (Lou Gehrig's disease). About 69 adults aged 18-80 with recent ALS symptoms will receive either the drug or a placebo. The main goal is to check for side effects, not to cure …

This was a planned phase 2 trial to test IC14, a monoclonal antibody, in 50 people with rapidly progressing ALS. Participants would have received intravenous IC14 or placebo twice weekly for 12 weeks. The study was withdrawn before any patients were enrolled, so no data on safety…

This study planned to test an experimental drug called IC14 in people with rapidly progressing ALS. The drug was given by IV over two weeks, and brain scans measured inflammation. However, the trial was withdrawn before enrolling any participants, so no data on safety or effectiv…

This early-stage study tests whether a new drug, prosetin, is safe for people with ALS (Lou Gehrig's disease) and healthy volunteers. About 72 participants will receive either the drug or a placebo to check for side effects and how the drug moves through the body. The goal is to …

This study aimed to see if a mist-inhaled drug called RNS60 could help people with ALS, a nerve disease that weakens muscles. It planned to measure changes in daily function, breathing, and survival. However, the trial was withdrawn before enrolling any participants, so no result…

This study tested an experimental drug called ARGX-119 in 60 adults with ALS. The goal was to see if it was safe and could help with muscle function. The trial was stopped early because early results showed it was unlikely to make a meaningful difference. No safety concerns trigg…

This program provides early access to an experimental drug called CNM-Au8 for people with amyotrophic lateral sclerosis (ALS), a progressive nerve disease. Participants must be 18 or older and have a confirmed ALS diagnosis. The goal is to offer treatment to those who cannot join…

This program provides CNM-Au8, a suspension of gold nanocrystals, to up to 300 people with ALS. The main goal is to give access to the drug and monitor safety, not to test if it works. Participants must have a confirmed ALS diagnosis and be at least 18 years old.

This study tests a new treatment for ALS (Lou Gehrig's disease) using stem cells taken from the patient's own belly fat. The cells are injected into the fluid around the spine every 3 months for a year. The goal is to see if this treatment is safe and can slow down the disease. A…

This clinical trial tests whether ibudilast (MN-166), a drug previously used for asthma, can slow the progression of amyotrophic lateral sclerosis (ALS). The study involves 234 adults with ALS, who will receive either ibudilast or a placebo for 12 months, followed by a 6-month op…

This study tests an experimental drug called ION363 in people with a rare, inherited form of ALS caused by FUS gene mutations. The goal is to see if the drug can slow the disease and help people live longer. About 89 participants will receive the drug via spinal injection. This i…

This trial provides expanded access to RAPA-501, a therapy made from a patient's own immune cells, for people with ALS. The goal is to control the disease and slow its progression. Participants must be 18 or older and have a confirmed ALS diagnosis.

This study tests whether a combination of nicotinamide (a form of vitamin B3) and pterostilbene (a plant compound) can slow the progression of ALS, a fast-moving nerve disease. About 380 adults with ALS will take either the supplement or a placebo for a period of time. The goal i…

This study tests a new drug called NX210c in 80 adults with ALS, a nerve disease that weakens muscles over time. Participants receive multiple IV infusions of the drug or a placebo to see if it slows the disease and is safe. The main focus is on changes in blood markers that refl…

This study is an open-label extension for people with early amyotrophic lateral sclerosis (ALS) who completed a previous placebo-controlled trial. Participants will take a daily oral dose of CNM-Au8, a suspension of gold nanocrystals, to see if it safely slows disease progression…

This early-stage trial tests a gene therapy called AMT-162 in 20 adults with a specific genetic form of ALS (SOD1-ALS). The treatment is given as a single injection into the spinal fluid. The main goal is to check safety and tolerability, while also looking for early signs that i…

This study tested a stem cell treatment for amyotrophic lateral sclerosis (ALS), a progressive nerve disease. It involved one patient who received their own fat-derived stem cells to try to control the disease. The treatment is no longer available.

This early-phase trial tests a substance made from stem cells from baby teeth (SHED-CM) in 10 people with ALS. The goal is to see if it is safe and if it might slow the disease. Participants receive the treatment intravenously over 12 weeks, with follow-up for 4 more weeks.

This program gives ALS patients who cannot join other clinical trials access to an experimental drug called trehalose (SLS-005). Trehalose is a sugar solution given once a week through a vein. The goal is to see if it can help control the disease. The program is no longer availab…

This study is testing whether a simple saliva sample can help diagnose amyotrophic lateral sclerosis (ALS). Researchers will analyze RNA in the saliva of 653 people, including ALS patients and healthy volunteers. If the test works, it could offer a faster, less invasive way to di…

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

This study tests whether using a mechanical insufflation device (BiWaze Cough) twice daily for 6 months can slow the decline in cough strength in 20 people with early ALS. Participants will use a facemask or mouthpiece to help inflate their lungs fully before coughing. Researcher…

This study is testing a device called MemorEM, which is a cap that sends low-level electromagnetic waves to the head. The goal is to get feedback from 1,000 patients with neurological diseases like Alzheimer's or Parkinson's, and their caregivers, on how easy the device is to use…

This study looks at two new ways to stage amyotrophic lateral sclerosis (ALS) based on key disease milestones. Researchers will check how well these systems work in 50 patients and see if adding a blood marker called neurofilament can improve tracking of the disease. The goal is …

This study is observing 217 people with ALS and related conditions like frontotemporal dementia and hereditary spastic paraplegia. Researchers aim to connect each person's genetic makeup with their symptoms and find biological markers in blood and spinal fluid. No new treatments …

This study looks at people with ALS and similar diseases like frontotemporal dementia and hereditary spastic paraplegia. Researchers want to understand how genes affect the disease and find biological markers (biomarkers) to help develop future treatments. About 708 participants,…

This study enrolls people with ALS and related disorders, as well as healthy volunteers, to track biological markers (biomarkers) in urine, blood, and spinal fluid. The goal is to better understand how these markers change over time, which could help design more effective future …

This study planned to see if mindfulness meditation using virtual reality could help people with ALS feel better emotionally and improve their quality of life. It was designed for adults with ALS who speak French. However, the study was withdrawn before enrolling any participants…

This study tests whether advanced MRI techniques can measure brain changes in people with ALS (Lou Gehrig's disease). Researchers will scan 119 participants three times over 8 months to see if these scans can track the disease and help diagnose it earlier. The goal is to create b…

This study looks at people with early ALS who still have some unaffected body areas. Researchers want to find the very first signs of the disease spreading. By observing 100 participants over time, they hope to learn how ALS starts and progresses, which could help with earlier di…

This study is testing a new, comfortable headset that reads brain signals to help people with ALS type on a virtual keyboard. The goal is to improve the device so it can be used in daily life. Five people with ALS will try the headset, and researchers will measure how easy it is …

This study looks at 80 people with ALS to see if electrical tests (EMG) and ultrasound scans of breathing muscles can predict when they will need breathing support. The goal is to start noninvasive ventilation at the right time. Researchers will also check for links between muscl…