Anterior horn disorder

This study tests a single injection of TRCN-1023 into the spinal fluid of adults with ALS. The goal is to see if it is safe and shows early signs of slowing the disease over 24 weeks. Participants are randomly assigned to receive the drug or a placebo, and neither they nor their …

This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who have already received gene therapy. The drug aims to help the body make more SMN protein, which is needed for muscle function. About 42 babies with two copies of the SMN2 gene will be rando…

This early-phase trial tests a gene therapy called SKG0201 in 11 infants with spinal muscular atrophy (SMA) type I, a severe muscle-weakening disease. The therapy uses a harmless virus to deliver a working copy of the missing SMN1 gene. The study aims to see if it is safe and can…

This study will follow 36 people with spinal muscular atrophy (SMA) who receive a gene therapy called onasemnogene abeparvovec (ITVISMA) injected into the spinal fluid. The goal is to see how well it works and how safe it is in everyday medical practice. Participants must be at l…

This study tests an oral traditional Chinese medicine made from the fungus C. cicadae in people with ALS who have high levels of certain fats (sphingolipids) in their blood. About 84 participants will receive either the medicine or a placebo for 6 months, with follow-up for 9 mon…

This early-stage trial tests whether a daily supplement called HMB (beta-hydroxy-beta-methylbutyrate) is safe and can lower certain blood markers linked to ALS. One hundred adults with ALS will take 3 grams of HMB daily. Researchers will track changes in blood markers and functio…

This early study tests whether repeatedly opening the blood-brain barrier with ultrasound is safe and might slow ALS. Twenty-three adults with ALS will receive 9 ultrasound sessions over 24 weeks. The first part checks safety; the second looks for signs of slowing the disease.

This Phase 2 trial tests whether umbilical cord stem cells can safely improve movement and coordination in people with spinocerebellar ataxia (types 1, 2, 3, and 6). Forty-five participants will receive the cells through an IV and a spinal injection. The main goal is to see if sy…

This study tests a new drug, PLL001, in 153 people with ALS. It is given as a daily injection under the skin. The trial is split into two parts: first to check safety, then to see if it slows the disease and helps people live longer. Participants are randomly assigned to get eith…

This pilot study tests whether terazosin, a drug used for high blood pressure, can increase energy levels in people with ALS. Twenty participants will take either terazosin or a placebo for 12 weeks. The goal is to see if the drug is safe and if it raises ATP, a key energy molecu…

This study tests whether the experimental drug tazbentetol can slow the progression of ALS, a disease that weakens muscles and affects movement. About 430 adults with ALS will receive either the drug or a placebo. The main goal is to see if tazbentetol helps maintain daily functi…

This study will test a mobile brain-computer interface (iBCI) that lets people with severe paralysis control computers and mobile devices using only their thoughts. Two veterans with conditions like spinal cord injury or ALS will use the device at home. The goal is to see if the …

This study tests a custom-made drug designed for one person with ALS caused by a specific gene mutation (CHCHD10). The drug aims to slow disease progression by targeting the genetic root cause. The participant will be closely monitored for changes in muscle strength, breathing, a…

This early-phase trial tests an oral drug, Kamlanoflast, in 40 people with ALS. Over 24 weeks, researchers will check safety, tolerability, and whether the drug reduces inflammation and slows decline in function. It is not yet recruiting.

This study tests whether combining two drugs (galunisertib and nerandomilast) can slow the progression of ALS in 60 people who have high levels of a protein called GREM2. The drugs aim to reduce brain inflammation and nerve cell damage. Participants will receive either the drug c…

This phase 3 trial tests whether adding masitinib to standard ALS treatment (riluzole) can slow the disease better than placebo plus riluzole. About 412 adults with ALS will take the drug or placebo for 48 weeks. The main goal is to measure changes in daily function and breathing…

This early-stage trial is testing a new gene therapy called SNUG01 for people with ALS, a disease that damages nerves controlling movement. The study will involve 21 adults and aims to check if the treatment is safe and tolerable. Researchers will also look for early signs that i…

This study aims to find specific markers in blood platelets that can help doctors diagnose ALS (Lou Gehrig's disease) more accurately and earlier. Researchers will compare 60 participants—30 with newly diagnosed ALS and 30 with other motor neuron diseases—to see if platelet tests…

This study tests a robotic device designed to help people with stroke, Parkinson's disease, ALS, or mild cognitive impairment improve their arm and hand function. Thirty adults will receive standard rehabilitation, and half will also use the robot for up to 30 minutes per session…

This study tests whether using a virtual reality (VR) system during cycling exercise makes rehabilitation more motivating for adults with conditions like stroke, Parkinson's, multiple sclerosis, or frailty. About 70 participants will try cycling with and without VR over 4 weeks. …

This pilot study tests a non-invasive device that stimulates a nerve in the neck to see if it can increase how often people with ALS swallow and their urge to swallow. Up to 5 participants will receive supervised treatment sessions and be monitored with swallowing tests. The goal…

This study will test a special moving platform combined with virtual reality to help people with neurological disorders like Parkinson's disease, stroke, ALS, or mild cognitive impairment improve their balance and movement. The platform moves in three directions and adapts exerci…

This study tests whether a home-based virtual reality guided breathing program can reduce anxiety and improve quality of life in people with ALS. Sixty participants will use a VR headset to follow breathing exercises. The goal is to see if this simple, immersive tool can help man…

This study looks at whether giving people with ALS and their caregivers video recordings of their clinic visits helps them feel better and manage care more easily. About 800 participants (400 patient-caregiver pairs) from eight U.S. sites will be split into two groups: one gets s…

This study tests whether a multicomponent support group can improve the confidence (self-efficacy) of primary caregivers of children with Duchenne muscular dystrophy or spinal muscular atrophy in Pakistan. Thirty caregivers will join group sessions with doctors, therapists, and o…

This study tests whether a special type of horseback physiotherapy can improve movement, posture, breathing, and quality of life in children with spinal muscular atrophy (SMA). Twenty children aged 2 to 9 years will receive both the horse therapy and standard physiotherapy in ran…

This study tests a mind-body program designed to help people recently diagnosed with ALS and their caregivers manage emotional distress. About 20 patient-caregiver pairs will try a 6-session coping intervention. The main goal is to see if the program is practical and well-liked, …

This study will test a new device that combines a brain-sensing headband with Apple's Vision Pro goggles to help people with ALS, brain injury, or spinal cord injury communicate. Up to 10 participants will use the system for 3-4 months to see how well they can type and control a …

This study tests a robotic exoskeleton called CLINICAL EXPLORER in 60 children aged 2 to 17 with neurodevelopmental disorders like cerebral palsy. The device supports walking during 8 therapy sessions. Researchers will check if it is safe, easy to use, and helps improve movement …

This study will observe 30 children with type I or type II spinal muscle atrophy who are taking risdiplam. Researchers will track motor function improvements over 24 months using standard tests. The goal is to see how well the drug works in everyday medical practice.

This study will interview 15 parents or guardians of children with spinal muscular atrophy type 1 to learn about their daily challenges with feeding and communication. Even though newer treatments have improved survival, their impact on swallowing and speech is not well understoo…

This study is testing a robot named Alter-Ego to see if it can safely help doctors, nurses, and therapists care for people with ALS. The robot can be controlled remotely or work on its own to do tasks like guiding remote medical visits, delivering supplies, and reminding patients…

This study looks at bone health in children with spinal muscular atrophy (SMA), a condition that causes muscle weakness. Researchers want to find out how common fractures are and whether newer SMA treatments affect bone strength. They will review medical records from about 550 ch…

This study tests a driving simulator designed to help people with neurological conditions like stroke, Parkinson's, ALS, or mild cognitive impairment improve their motor and thinking skills. About 60 participants (including healthy volunteers) will use the simulator to see if it …

This early-phase study tests a special dye called [11C]CPPC used in PET scans to see if it can safely detect brain inflammation in people with ALS. Researchers will compare scans from 20 ALS patients and healthy volunteers to check if the dye highlights areas of inflammation and …

This study will enroll 35 Spanish-speaking adults with ALS and a similar group without ALS to see if a smartphone app can reliably measure changes in speech and breathing over time. Participants will use the app to record speaking and breathing tasks, and researchers will compare…

This observational study will follow 100 children in Denmark who receive a stem cell transplant for blood cancer or other serious blood disorders. Researchers will test their thinking skills, physical abilities, and daily activity before the transplant and for one year after. The…

This observational study screens 500 people with early-stage ALS to see if they qualify for specific early-phase clinical trials. Participants undergo tests, genetic analysis, and biomarker checks to match them with the right trial. The study also builds a research database for f…

This study aims to understand how care for people with Motor Neurone Disease (MND) is coordinated between specialist clinics and local community services. Researchers will talk to patients, carers, and healthcare professionals, and observe how care is transferred. The goal is to …

This study follows 600 Chinese children who received standard vaccines against polio, measles, mumps, rubella, and hepatitis A. Researchers will test their blood to see if protective antibodies remain 5 years after vaccination. The goal is to understand how long vaccine protectio…

This study will compare how well an AI program (Blings) measures speech clarity in 40 adults with speech disorders from stroke or other neurological conditions. Two speech therapists will also rate the same speech samples. The goal is to see if the AI can reliably replace or assi…

This study looks at how parents feel after their newborn is screened for spinal muscular atrophy (SMA). Researchers will interview 36 parents in two French regions to understand their anxiety, stress, and support needs. The goal is to improve how screening results are shared and …

This study tests a Polish version of a questionnaire called DALS-15, which measures how severe breathing difficulty (dyspnea) is in people with ALS. About 120 Polish-speaking ALS patients who have trouble breathing will fill out the questionnaire along with other breathing tests.…

This pilot study will use ultrasound to measure diaphragm movement in 20 ALS patients, both while they breathe on their own and while using non-invasive ventilation (NIV). The goal is to see if ultrasound can detect changes in how the diaphragm works with and without the breathin…

This study will follow 175 children and young adults who had a bone marrow transplant before age 18. Researchers will test their thinking, memory, and attention skills, and check for links to physical health issues like metabolism and fitness. The goal is to find early warning si…