Amyotrophic lateral sclerosis

This early-phase study tested the drug baricitinib in 17 people with Alzheimer's disease, ALS, or mild cognitive problems. The goal was to see if the drug reaches the brain and lowers inflammation linked to these diseases. Participants took baricitinib pills for 24 weeks, and res…

This early study tested whether infusing a patient's own regulatory T cells (immune-calming cells) along with interleukin-2 injections is safe for people with ALS. Only 3 participants were enrolled, and the main goal was to check for side effects. The approach aims to slow diseas…

This study tested whether people with ALS or Parkinson's disease, along with healthy volunteers, could follow the MIND diet — a mix of the Mediterranean and DASH diets thought to protect the brain. Over several weeks, 43 participants received a cookbook, educational materials, an…

This study tested the long-term safety of an oral form of edaravone in 124 people with ALS over 96 weeks. Participants took the drug in cycles of 10 days on and 14 days off. The main goal was to see how safe and tolerable the treatment was over time.

This study looked at the safety of an oral form of edaravone for people with ALS, a nerve disease that weakens muscles. Fifteen patients who had already been in earlier edaravone studies took the drug for 10 days every two weeks. Researchers tracked side effects to see if the med…

This study looked at the long-term safety of an oral form of edaravone in 185 adults with ALS. Participants took the medication for 24 to 48 weeks, and researchers tracked any side effects. The goal was to see if taking the drug by mouth is safe over time for people with this pro…

This study tested a daily ketone supplement (beta hydroxybutyrate ester) in 81 people with ALS to see if it could slow the disease. The idea is that ketones provide extra energy to cells, which may help counter the energy shortage seen in ALS. Participants took the supplement alo…

This Phase 3 trial tested a stem cell treatment called Lenzumestrocel in 123 people with ALS. The treatment uses the patient's own bone marrow stem cells and is given alongside the standard drug Riluzole. The goal was to see if it could slow the disease and improve survival and f…

This early-phase trial tested whether combining two drugs—abatacept and interleukin-2—is safe for people with ALS. The goal was to boost the body's regulatory T cells, which may help slow disease progression. Only 5 participants were enrolled, and the study focused on safety and …

This trial tested a custom-made drug called nL-TARD-001 in one person with ALS caused by a specific genetic mutation in TARDBP. The drug is an antisense oligonucleotide designed to target the faulty gene. The study measured changes in muscle function, breathing, and thinking over…

This study tested a drug called TPN-101 in 42 people with ALS or frontotemporal dementia caused by a specific gene change (C9orf72). The main goal was to check if the drug is safe and tolerable over 48 weeks. Researchers also looked at how the drug moves in the body and whether i…

This study tested whether deferiprone, a drug that removes excess iron, could safely slow the progression of ALS. Researchers gave the drug to 23 adults with ALS for several months. The goal was to see if reducing iron buildup in the brain might protect nerve cells and delay wors…

This study tested a drug called DNL343 in 249 people with ALS, a serious nerve disease that weakens muscles over time. The goal was to see if DNL343 could slow the disease and help people live longer. The trial used a flexible platform design to test multiple treatments at once.

This Phase 3 trial tested the drug edaravone (MCI-186) in 206 people with ALS. Participants received daily intravenous infusions for 14 days, followed by a rest period, repeated over 24 weeks. The goal was to see if edaravone could slow the decline in physical function compared t…

This study tested the drug edaravone (MCI-186) in 181 people with ALS, a progressive nerve disease. Participants received either edaravone or a placebo through IV infusions over 24 weeks. The goal was to see if edaravone could slow the decline in physical function. Results sugges…

This study tested a drug called MCI-186 in 137 people with ALS, a nerve disease that weakens muscles over time. The goal was to see if the drug could slow down the disease compared to a placebo over 24 weeks. The main measure was a scale of daily function. The trial did not show …

This study tested a drug called MCI-186 in 25 people with advanced ALS (severity grade III) to see if it could slow the disease. Participants received either the drug or a placebo daily through an IV for 24 weeks. The main goal was to measure changes in daily function and safety,…

This completed Phase 2/3 trial tested whether deferiprone, a drug that removes excess iron from the body, can slow the progression of ALS (Lou Gehrig's disease). 372 people with early-stage ALS took either deferiprone or a placebo for 12 months. The study measured changes in phys…

This phase 2 trial tested whether low-dose interleukin-2 (IL-2) can help control nerve inflammation in people newly diagnosed with ALS. Over 18 months, 304 participants received either IL-2 or a placebo, alongside standard riluzole treatment. The goal was to see if boosting regul…

This phase II study tested whether low doses of interleukin-2 (IL-2) can safely boost regulatory T cells in people with amyotrophic lateral sclerosis (ALS). 36 participants received either IL-2 or a placebo for five days. The goal was to see if this immune-modulating approach cou…

This completed Phase 2/3 trial tested an oral drug called ABBV-CLS-7262 in 310 people with ALS. Participants took the drug or a placebo once daily for 24 weeks. The study measured how fast the disease progressed and survival rates. The goal was to see if the drug could slow ALS a…

This phase 2 trial tested whether adding IFB-088 to standard riluzole treatment is safe and effective for people with bulbar-onset ALS (a form that first affects speech and swallowing). 51 participants received either IFB-088 plus riluzole or a placebo plus riluzole for 6 months.…

This study looked at the long-term safety of an experimental drug called Engensis for people with ALS (Lou Gehrig's disease). Eight people who had already received Engensis in an earlier study were followed for 6 more months. Researchers tracked side effects to see if the drug re…

This completed Phase 2 trial tested a combination of liposomed curcumin (from turmeric) and resveratrol (from grapes) plus a drug called G04CB02 in 90 people with ALS. The goal was to see if this mix could improve muscle function, thinking, and inflammation over 2 months of treat…

This study tested a drug called troriluzole in 141 adults with spinocerebellar ataxia, a rare disease that affects balance and coordination. Participants took either the drug or a placebo daily for 8 weeks. The main goal was to see if troriluzole could improve symptoms like walki…

This completed Phase 2 pilot trial tested whether personalized supplements could slow the progression of ALS. Researchers used a genetic analysis platform to sort 50 participants into four disease categories—neuroinflammation, oxidative stress, impaired autophagy, or mitochondria…

This phase IIb trial tested PrimeC, a combination of two common drugs (ciprofloxacin and celecoxib), in 69 people with ALS. Participants took PrimeC or a placebo for 6 months, followed by a 12-month open-label extension where everyone received PrimeC. The study measured safety, s…

This phase 1 study tested a single dose of the experimental drug QRL-101 in 12 people with ALS. The main goals were to see how the drug moves through the body and whether it is safe. Participants received either QRL-101 or a placebo, and researchers monitored side effects and dru…

This study tested a new PET imaging tracer to see if it can safely show signs of ALS in the body. Eight people (some healthy, some with ALS) received the tracer and were scanned. The goal was to check safety and how the tracer spreads, which could lead to earlier diagnosis and be…

This study looked at whether chemicals in tears and eye surface cells can help diagnose amyotrophic lateral sclerosis (ALS) or predict how the disease will progress. Researchers collected tear samples and performed eye exams on 55 people with ALS and healthy controls. The goal wa…

This study aimed to develop a non-invasive prenatal test using fetal cells from a mother's blood to detect triplet repeat diseases like Huntington's disease, Fragile X syndrome, and certain types of muscular dystrophy and ataxia. Researchers enrolled 60 pregnant women at risk and…

Researchers tested an artificial intelligence program that analyzes retinal images to diagnose several nerve and brain conditions, such as optic neuropathy and brain tumors. The study used data from 693 patients with confirmed diagnoses. The goal is to create a fast triage tool f…

This study tests whether a wheelchair-mounted robotic arm called Jaco helps people with tetraplegia perform daily tasks like grasping and reaching at home. Participants use the device for two months after training. Researchers measure changes in performance and satisfaction with …

This study tested whether people with ALS can start using a breathing machine at home instead of in the hospital. 46 patients were randomly assigned to start either at home with remote monitoring or in the hospital. The goal was to see if home initiation works just as well, measu…

This pilot study tested whether adding a vibrating chest vest to a standard cough-assist machine could reduce lung infections and hospital stays in people with ALS who use breathing support. Fourteen patients were randomly assigned to use either the vest plus cough assist or the …

This study tested a 12-week online peer support programme for family caregivers of people with motor neurone disease who are becoming dependent on breathing or feeding technology. The programme included private messaging, weekly chats, a discussion forum, and resources. Researche…

This completed trial from Harvard University tested whether an online mindfulness program and social community could improve quality of life for 100 people with ALS and their primary caregivers. Participants used a three-week e-learning program with cognitive exercises and videos…

This Phase 3 study tested whether injections of MYOBLOC (a botulinum toxin type B) into the salivary glands can safely reduce troublesome drooling in adults. 187 participants received either MYOBLOC or a placebo. The main goals were to measure changes in saliva flow and overall i…

This study looked at whether a moderate-intensity breathing exercise program is safe and doable for people with ALS. Nine adults with ALS did four 3-week cycles of respiratory muscle training at home, with weekly check-ins via video call. The main goal was to see if participants …

This study tested the safety of an experimental drug called engensis in 18 people with amyotrophic lateral sclerosis (ALS). Participants received either engensis or a placebo injection into their muscles. The main goal was to check for side effects, while also exploring effects o…

This early-phase trial tested whether a gene therapy called VM202 is safe when injected into muscles of 18 people with ALS. The study focused on side effects over 9 months, not on whether the treatment improves symptoms. All participants had ALS for less than 2 years and had at l…

This study follows about 74 adults with ALS who have already decided to take the drug Edaravone (Radicava). Researchers collect blood and urine samples over 24 weeks to measure various biomarkers—molecules that might show how the drug affects oxidative stress, inflammation, and n…

This study is testing whether a smartphone app can reliably track speech and swallowing problems in people with ALS. Participants will record their voice and swallowing sounds at home each week using the app. The goal is to see if this remote monitoring is feasible and accurate c…

This study tests whether wearable sensors and digital home tasks can track changes in movement, speech, and falls in people with ALS. About 20 participants will wear neck and wrist sensors and complete speech and handwriting tests over 48 weeks. The goal is to see if these digita…

This study explores whether digital tools on a tablet can measure changes in speech and handwriting in people with motor neuron diseases like ALS. Participants complete a single session of speaking and tracing tasks. Researchers compare results between patients and healthy contro…

This study followed 50 adults with a C9ORF72 gene mutation that can cause ALS or frontotemporal dementia. Over three years, researchers measured changes in strength, thinking, memory, and behavior using brain scans, spinal fluid tests, and other assessments. The goal was to under…

This study tested whether a brain-computer interface (BCI) device is ready for commercial use to help people with minimal movement communicate. Researchers worked with 8 device users, plus speech therapists and support staff, to evaluate how well the device worked at home. The go…

This study looks at how ALS (Lou Gehrig's disease) affects social thinking, like recognizing emotions in faces and voices. Researchers will compare 74 ALS patients to healthy volunteers using video-based tests. The goal is to better understand these challenges, which can impact d…

This study looked at whether vitamin D levels in the blood are linked to how fast ALS (Lou Gehrig's disease) gets worse. Researchers measured vitamin D in 127 people with ALS and tracked their motor decline, breathing, and weight loss. No treatment was given—the goal was simply t…

This early-stage study looked at how a single dose of oral edaravone moves through the bodies of 9 Japanese adults with ALS. The main goal was to measure drug levels in the blood and urine, not to test if it helps symptoms. Researchers also tracked any side effects. The study is …

This early-stage study looked at how the body handles a single dose of oral edaravone in 6 Japanese ALS patients who have a feeding tube (gastrostomy). The goal was to measure drug levels in the blood and urine, not to test if it works. No treatment benefit was evaluated.

This study tested whether a telehealth system could help people with motor neurone disease (MND) receive specialist care from home, avoiding long trips to the clinic. It involved 40 patients and their caregivers, with half using the system for up to 18 months. The goal was to see…

This completed study looked at how the drug deferiprone affects blood cells from 90 people with Parkinson's disease, ALS, or healthy volunteers. Researchers measured markers of oxidative stress and iron levels in the lab. The goal was to better understand these diseases, not to t…

This completed study of 500 people with ALS (Lou Gehrig's disease) aimed to find biological signs, or biomarkers, that can predict how the disease will progress. Researchers collected clinical, imaging, and lab data to better understand different forms of ALS. The goal is to help…

This completed study looked at how muscle oxygen levels change during exercise in people with various neuromuscular diseases compared to healthy volunteers. Seventeen participants did a knee-extension exercise while a device measured muscle oxygen. The goal was to understand diff…

This study tested whether home automation and remote monitoring can help elderly people (65+) with multiple chronic conditions live safely at home. Over 500 participants had their homes equipped with sensors that tracked health signs and sent alerts to doctors. The main goal was …

This early study tested whether a special ultrasound technique could safely open the blood-brain barrier in the motor area of the brain in 4 people with ALS. The goal was to see if the procedure was safe and possible, not to treat the disease. Researchers measured side effects an…

This study aimed to translate and validate an Italian version of a tool that helps speech therapists assess swallowing difficulties (dysphagia) in people with neurodegenerative diseases like Parkinson's or ALS. Researchers tested the tool on 101 adults with such conditions to ens…

This study tested a new way to control a robotic arm mounted on a power wheelchair. The arm uses a camera to help guide movements, making tasks like grabbing a drink easier. 21 adults who use power wheelchairs and have trouble with everyday tasks took part. They compared the new …

This completed study looked at how different types of brain damage—from strokes, tumors, infections, or degenerative diseases—affect thinking and memory. Researchers used brain scans (MRI) and cognitive tests in 346 patients and healthy volunteers to map which brain areas are res…

This study tested whether advanced MRI scans can find new biomarkers for amyotrophic lateral sclerosis (ALS), a fast-progressing nerve disease. Researchers used high-field and very-high-field MRI to look at the brain, spinal cord, and muscles of 22 ALS patients over six months. T…

This study looked at how nerves in the spine work in people recently diagnosed with ALS compared to healthy volunteers. The goal was to find specific electrical signals that could help doctors understand the disease better and track its progress. 101 participants took part in tes…

This completed study looked at how metabolism differs in people with ALS. Researchers took blood and skin samples from 9 patients to analyze molecules and genes. The goal was to better understand the disease, not to test a treatment.

This study aimed to find biological markers (biomarkers) in muscle, blood, and cells to help diagnose amyotrophic lateral sclerosis (ALS) earlier and understand the disease better. Researchers analyzed samples from 37 adults with ALS using advanced techniques to look at molecules…

This completed Phase 1 trial tested the safety and how the body absorbs VRG50635 in 22 healthy adults aged 19 to 55. Participants received either the drug or a placebo. The study focused on side effects and drug levels in the blood, not on treating any disease.

This completed study looked at 60 people — 30 with ALS and 30 healthy volunteers — to measure immune system and nerve damage markers in their blood and spinal fluid. The goal was to better understand the role of a specific immune pathway (IFN-alpha) in ALS. By linking these marke…

This completed study looked for genetic and environmental factors that increase the risk of developing Parkinson's disease. Researchers compared DNA and lifestyle data from over 2,200 people, including Parkinson's patients, people with related brain diseases, and healthy voluntee…

This completed study looked at genetic differences in vitamin D processing and action in 400 people with ALS and 400 healthy controls. Researchers analyzed DNA samples to find variations in eight specific genes. The goal was to better understand the biology of ALS, not to test a …

This study looked at how amyotrophic lateral sclerosis (ALS) affects the brain's ability to process touch and other sensations, even when patients don't notice problems. Researchers used brain imaging and electrical recordings to compare 52 people with ALS and healthy volunteers.…

This study interviewed 15 people with ALS to learn about unpleasant sensations or pain they experienced during clinical trial procedures. The goal was to identify which procedures cause discomfort and how long it lasts, so future trials can be more patient-friendly. Researchers a…

This completed study looked at 40 people with spinocerebellar ataxia types 2 and 7, a rare brain disease that affects movement. Researchers used MRI scans and lumbar punctures over one year to track changes in the brain and body. The goal was to find reliable markers that could b…

This study looks at how ALS affects the way people start walking and keep their balance. Researchers will compare ALS patients with and without balance issues, plus healthy volunteers, using walking tests and brain scans. The goal is to find out if certain brain changes cause wal…

This small study tested whether briefly restricting blood flow to one arm (called remote ischemic conditioning, or RIC) could improve hand function in people with spinal cord injury or ALS. Participants did hand exercises while receiving either active RIC or a sham version. The g…

This study first surveyed 888 nurses across Spain and found that over 70% lacked adequate knowledge of advanced life support (ALS). Researchers then tested a training program with 38 nurses in one region, combining online materials with a four-hour hands-on workshop. After the tr…

This completed study looked at 44 people with ALS and healthy controls to see how gut bacteria and short-chain fatty acids change over time. Researchers also tested whether a 6-month Mediterranean diet could shift these levels. The goal was to better understand the gut-brain conn…

This study looked at 70 elderly women with long-term non-specific low back pain to see how their balance relates to disability, fear of falling, and social participation. Researchers measured balance, disability, and community involvement. The goal is to better understand these c…