Motor neuron disorder

This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assig…

This phase 2 trial tests whether the experimental drug VHB937 can slow the decline in function and delay the need for breathing support in people with early-stage ALS. About 251 participants will receive either VHB937 or a placebo for 40 weeks, followed by an open-label extension…

This study planned to test whether injecting botulinum toxin (Botox) into the salivary glands could reduce drooling in people with true bulbar palsy, a condition that causes swallowing problems after a brain stem injury. The trial was designed to compare the injections against st…

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…

This study tests a gene therapy called vesemnogene lantuparvovec in about 20 people with spinal muscular atrophy (SMA), a genetic condition that causes muscle weakness. The therapy delivers a working copy of the SMN1 gene to help muscles work better. The main goal is to check saf…

This study tests whether adding an experimental drug (RO7204239) to an existing SMA medicine (risdiplam) can help people with spinal muscular atrophy build stronger muscles and move better. The trial includes about 259 children and young adults, ages 2 to 25, who can walk. Resear…

This study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …

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 study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy (SMA). About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to…

This early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …

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 study was designed to test whether the drug govorestat could improve symptoms of Charcot-Marie-Tooth disease caused by sorbitol dehydrogenase deficiency (CMT-SORD), a rare nerve condition. It planned to enroll people aged 16 to 65 and compare govorestat to a placebo over 36 …

This study tests a higher dose of nusinersen (Spinraza) in 45 teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken risdiplam. Participants receive two 50 mg loading doses two weeks apart, then 28 mg every four months for about two years, …

This early-stage trial tests a gene therapy called PBFT02 for frontotemporal dementia (FTD) in people with specific genetic mutations. The therapy delivers a working copy of the GRN gene directly into the brain. The study involves 30 adults and primarily checks for safety and sid…

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 expanded access program provides apitegromab, an experimental drug that blocks myostatin to help maintain muscle strength, to eligible patients with spinal muscular atrophy (SMA) before it is officially approved. Participants must be at least 2 years old and have a confirmed…

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 early trial tests whether a transplant of umbilical cord blood can help people with ALS, a fast-progressing nerve disease. Eight adults will receive the transplant after a mild chemotherapy-like prep. Researchers will check safety and whether the treatment slows decline in m…

This study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and…

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 study tests the drug risdiplam (Evrysdi) in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment can help them reach motor milestones like sitting without support. The trial involve…

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 phase 3 trial tests whether the drug tofersen can delay or prevent ALS in adults who carry a SOD1 gene mutation but have no symptoms yet. About 158 participants will receive either tofersen or a placebo, and researchers will track how many develop ALS within two years. The g…

This pilot study tests whether combining intensive hand-arm and leg therapy with strength training can improve motor function in children with spinal muscular atrophy (SMA). Five children will attend one 6-hour session each weekend for 15 weeks. Researchers will check if the ther…

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 long-term study is testing higher doses of nusinersen (Spinraza) in people with spinal muscular atrophy who already completed a previous nusinersen study. The main goal is to check safety over several years, including side effects, heart health, and growth. Participants rece…

This clinical trial is testing an oral drug called NUZ-001 in 160 people with ALS. Participants take the drug or a placebo daily for 36 weeks. The study measures how the disease progresses using a scale of physical function and survival. The goal is to see if NUZ-001 can slow ALS…

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 the drug usnoflast can slow the progression of ALS, a severe nerve disease that causes muscle weakness and breathing problems. About 240 adults with ALS will receive either usnoflast or a placebo for 36 weeks. Researchers will measure changes in daily fun…

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 study tests whether metformin, a widely used diabetes drug, is safe for people with a specific genetic form of ALS (C9orf72). Over 24 weeks, 41 participants will take metformin to see if it reduces toxic proteins linked to the disease. The goal is to determine if metformin c…

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINR…

This early-stage trial tests a single injection of RJK002, a gene therapy, into the spinal fluid of 9 people with ALS. The main goal is to see if it is safe and to find the best dose for future studies. Researchers will also track changes in daily function, but the trial is too s…

This study tests a new gene therapy called SKG0201 in 12 infants with spinal muscular atrophy type 1, a severe muscle-weakening disease. The treatment aims to improve survival and motor skills by delivering a working gene. Researchers are checking safety and how well the therapy …

This study tests whether digoxin, a heart medication, is safe and tolerable for people with ALS. It also looks at whether digoxin can slow nerve damage. About 40 adults with early-stage ALS will take digoxin for 24 weeks. The goal is to find a new way to manage this disease.

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 early-stage trial tests a single injection of gene therapy for people with SMARD1 or CMT2S, rare diseases caused by mutations in the IGHMBP2 gene. The therapy delivers a working copy of the gene directly into the spinal fluid. Ten participants, ranging from infants to childr…

This study provides an experimental stem cell treatment (HB-adMSCs) to an 83-year-old man with Primary Lateral Sclerosis, a rare nerve disease that causes progressive muscle weakness. The patient's own banked stem cells are used, aiming to slow the disease. This is a single-patie…

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 offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

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 tests whether an AI tool can help radiologists read brain CT and MRI scans more accurately and quickly. Researchers will compare how well doctors, AI alone, and doctors using AI together can spot abnormalities, urgent findings, and classify diseases. The goal is to red…

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 a 12-week online program for mothers of infants (12-36 months old) with SMA Type 1. The program aims to teach mothers how to support their baby's development and improve their own knowledge. Thirteen mothers will take part, with 10 receiving the program and 3 rec…

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 tests whether a drug called istradefylline, combined with short periods of breathing low-oxygen air (acute intermittent hypoxia), can improve breathing in people with ALS. About 40 adults with ALS and healthy volunteers will take part. The goal is to see if this combin…

This pilot study tests whether using a robotic glove for 20 minutes a day, 5 days a week, can improve quality of life, grip strength, and fine motor control in people with ALS. Five participants will use the glove at home over 8 weeks, with some in-person and telehealth check-ins…

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 tests whether a home-based brain stimulation device (tDCS) combined with activity therapy can help improve cognitive and language problems in people with stroke, brain tumors, or neurodegenerative conditions like Parkinson's or Alzheimer's. Fifty-five participants will…

This study is testing whether a robotic exoskeleton called Atalante can safely help people with ALS maintain their ability to walk. The idea is that the suit provides natural leg movements and feedback to the brain, which may slow down walking decline. About 20 adults with slow-p…

This study tests a new, team-based rehabilitation program for 125 working-age adults with persistent physical symptoms and chronic fatigue. The program includes doctors, therapists, and counselors who work together to help patients manage their symptoms and improve daily function…

This study tests a wearable robotic exoskeleton called EXPLORER in 15 children with movement problems from conditions like cerebral palsy. The goal is to see if it helps them walk better in their everyday environments, such as home and the community. The study focuses on safety, …

This early-stage trial tests the drug dalfampridine (Ampyra), already used for multiple sclerosis, in 35 adults with primary lateral sclerosis or upper motor neuron ALS. The main goal is to see if it safely improves walking speed, measured by a timed 25-foot walk. Researchers wil…

This study tests a robotic feeding device called Obi for people who cannot use their arms to eat on their own. About 50 participants, including patients, caregivers, and healthcare providers, will use the device at home or in care settings for one week. Afterward, they will fill …

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 will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

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 uses advanced MRI scans and genetic tests to track how neurodegenerative diseases like frontotemporal dementia and ALS spread across the brain's network. Researchers will follow 645 participants, including patients, family members, and healthy controls, for up to 2 yea…

This study checks whether a French version of a scale called PLSFRS works well for people with primary lateral sclerosis (PLS), a rare nerve disease. Researchers will ask 80 patients to fill out the questionnaire during routine check-ups every 3 to 6 months. The goal is to see if…

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 follows 1000 people with upper motor neuron disease who are about to receive the Cionic Neural Sleeve. The sleeve uses electrical stimulation to help muscles contract at the right time during movement. Researchers will track changes in health-related quality of life us…

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 whether a power wheelchair that can lift a child from sitting to standing is practical for kids with conditions like cerebral palsy, spinal cord injuries, or genetic diseases. Only 4 children aged 5-17 are taking part. The goal is to see if the chair helps the…

This study follows 35 infants with spinal muscular atrophy (SMA) who are receiving gene therapy or other advanced treatments. Researchers use small wearable motion sensors to measure how well the babies move their arms and legs over two years. The goal is to create a more precise…

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…

This study is a registry that collects information on up to 600 children in China with spinal muscular atrophy (SMA). It aims to describe how the disease progresses and how treatments are used in everyday medical practice. The study does not test a new drug but gathers data to be…