Inherited neurodegenerative disorder

This Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes i…

This study tests a new nasal spray treatment for frontotemporal dementia (FTD), a brain disease that changes personality and language. The spray contains tiny particles from umbilical cord stem cells that may protect brain cells and reduce harmful protein buildup. About 33 adults…

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 tests whether adding hyaluronic acid gel to standard deep cleaning (scaling and root planing) improves gum healing in people with both diabetes and advanced gum disease. Twenty-three participants will receive the standard cleaning on all teeth, and then two gum pockets…

This study tests whether the drug pridopidine can slow the worsening of Huntington's disease (HD) in adults. About 400 participants will take either pridopidine or a placebo twice daily for one year, then everyone can receive pridopidine for two more years. The goal is to see if …

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 early-stage study tests whether a focused ultrasound can safely and temporarily open the brain's protective barrier in people with early Alzheimer's disease. The goal is to see if this helps clear harmful amyloid and tau proteins that build up in the brain. Six participants …

This early-phase trial tests a gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. Twelve children will receive a single injection of the therapy into the fluid around the spinal cord. Researchers will check if it is safe and if it can slow the di…

This phase 3 trial tests whether PLX-200 (gemfibrozil) can safely slow the progression of CLN3 disease, a rare genetic disorder that causes loss of motor skills, vision, and seizures in children. About 39 kids aged 6-18 will receive either the drug or a placebo for up to 96 weeks…

This study tests whether low-dose radiation to the whole brain can slow down Alzheimer's disease in people with early-onset symptoms. Fifty participants will be randomly assigned to receive either the radiation treatment or a sham procedure. Researchers will track changes in memo…

This study tests several experimental treatments for progressive supranuclear palsy (PSP), a rare brain disease that affects movement and thinking. About 440 people with early-stage PSP will receive one of the treatments or a placebo. The goal is to see if any of the treatments c…

This study tests a vaccine called AADvac1 in 146 people with progressive supranuclear palsy (PSP), a rare brain disorder that affects movement and thinking. The vaccine aims to target a protein linked to the disease and slow its progression. Participants will receive injections o…

This Phase 2 trial tests whether the drug LM11A-31 can slow the progression of Progressive Supranuclear Palsy (PSP), a rare brain disorder that affects movement and balance. About 147 people with early-stage PSP will receive either the drug or a placebo twice daily for several mo…

This phase 3 trial tests whether short-course radiation followed by chemotherapy and an immunotherapy drug (serplulimab) works better than standard long-course chemoradiation for high-risk locally advanced rectal cancer. 612 patients will be randomly assigned to one of the two tr…

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 vaccine called AADvac1, which targets tau tangles in the brain, a hallmark of Alzheimer's. It will be given alone or with another drug (donanemab) to 450 adults aged 50-80 with very early Alzheimer's. The goal is to see if this approach can reduce tau buildup a…

This study tests whether efavirenz, a drug used for HIV, can help people with Creutzfeldt-Jakob disease (CJD) live longer. CJD is a rare, rapidly fatal brain disorder. The trial will enroll 246 adults and randomly assign them to receive either efavirenz or a placebo. The main goa…

This early-stage trial tests whether stem cells and their exosomes (tiny particles that help cells communicate) can safely improve movement in people with spinocerebellar ataxia, a condition that affects coordination and balance. Ninety participants will receive the treatment thr…

This study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, fix the genetic defect in the lab, and then see if these cells can help brain cells work better. The goal is to show the …

This study tests whether a special low-calorie, plant-based diet taken for 5 days each month can safely improve blood flow to the brain in middle-aged adults who carry the APOE4 gene, which raises Alzheimer's risk. Forty participants aged 45-65 will be randomly assigned to either…

This early study tests whether high-dose vitamin B3 (nicotinamide) can help preserve or improve vision in people with Leber's hereditary optic neuropathy (LHON), a rare genetic disease that causes sudden vision loss. Researchers will give 13 participants 2 grams of vitamin B3 dai…

This study tests a new drug called NS101 in 15 people with semantic variant primary progressive aphasia (svPPA), a rare type of frontotemporal dementia that affects language and memory. The drug is an antibody designed to help repair connections between brain cells. The main goal…

This study is testing a new cognitive assessment called the ExéSem battery, designed to help doctors tell the difference between language problems caused by memory loss versus those caused by thinking difficulties. Researchers will enroll 140 people, including those with Alzheime…

This observational study will enroll 440 Danish-speaking adults over 50 to see if an artificial intelligence model can detect dementia and mild cognitive impairment from speech recordings. Participants will complete standard cognitive tests and describe a picture while being reco…

This early-phase trial will test a new radioactive tracer called 18F-FCOB04 in 40 people with neurodegenerative diseases like Alzheimer's or Parkinson's. The tracer targets a brain protein called MAO-B and is used with PET scans to see if it can help diagnose these conditions. Th…

This study will test whether measuring a specific abnormal protein (alpha-synuclein) in spinal fluid can reliably distinguish dementia with Lewy bodies (DLB) from Alzheimer's disease. Researchers will collect samples and perform cognitive tests on 286 older adults with suspected …

This study tests a drug called N-acetyl-L-leucine for people aged 4 and older with CACNA1A gene disorders, which can cause coordination problems, dizziness, and migraines. About 60 participants will receive either the drug or a placebo, then switch, to see if it improves movement…

This pilot study will test whether a non-invasive brain stimulation technique called transcranial alternating current stimulation (tACS) can improve working memory in 30 adults with mild cognitive impairment or traumatic brain injury. Participants will receive low-level electrica…

This pilot study tests a 4-week program based on acceptance and commitment therapy (ACT) for women who have experienced a miscarriage, stillbirth, or newborn death within the past year. The program includes six sessions plus a booster, combining in-person and video meetings. Rese…

This pilot study tests whether listening to music in the evening can help people with Alzheimer's disease or related disorders fall asleep faster and behave more calmly. Researchers will compare music therapy to audiobooks in 48 adults living in secure care units. The goal is to …

This study looks at whether doing aerobic exercise at home, with or without the drug omaveloxolone, can safely improve fatigue and heart-lung fitness in people with Friedreich's ataxia. About 30 adults with the condition will take part. The goal is to see if the combination helps…

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 speech training using five musical tones can help people with aphasia (trouble speaking) after a stroke. Researchers will enroll 120 adults who had a stroke and have non-fluent aphasia. Participants will receive either five-tone therapy …

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 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 investigates how pomegranate dietary supplements influence gut bacteria and inflammation in people with alcohol use disorder and alcohol-related liver disease. Researchers will measure levels of beneficial gut metabolites called urolithins and inflammatory markers in b…

This study will test whether wearable sensors can accurately monitor movement problems in people with progressive supranuclear palsy (PSP) and dementia with Lewy bodies (DLB). About 60 participants will wear small sensors on their chest, ankles, and wrists while doing simple task…

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 observational study will enroll 200 people—100 with Parkinson's disease, 50 with related conditions, and 50 healthy controls—to find molecular markers in blood, urine, and stool that can distinguish Parkinson's from atypical parkinsonism. Participants provide samples and und…

This study will test if wearable sensors can effectively monitor symptoms and daily function in people with frontotemporal dementia (FTD). Sixty adults with FTD will wear pendant and wrist sensors for two weeks at a time, every six months over two years. The goal is to develop di…

This study tests whether wearable sensors and computerized voice and thinking tests can reliably track symptoms in people with frontotemporal dementia (FTD). Twenty participants will wear sensors and complete assessments at home over 12 months. The goal is to develop digital biom…

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 will test a wearable device called Syde® in 30 people with progressive supranuclear palsy (PSP-R). The device tracks movement and activity during daily life. Researchers want to see if patients find it easy to use and if the data matches standard clinic tests. The goal…

This study will see if an AI chatbot app is practical and acceptable for family caregivers of people with traumatic brain injury, dementia, or Huntington disease. About 60 caregivers will use the app for 8 weeks, focusing on physical and mental health. They will complete surveys …

This early-stage study will give 8 healthy men a single radioactive dose of LPM3770164, a drug being developed for Huntington disease. Researchers will measure how much of the drug is recovered in urine and stool, and identify its breakdown products. The goal is to understand how…

This study looks at how Huntington's disease impacts autobiographical memory—the ability to recall personal experiences and imagine future events. Researchers will use eye-tracking and neuropsychological tests in 80 participants (patients and healthy controls) to find markers of …

This observational study will follow 50 children aged 5 to 8 with non-progressive congenital ataxia, a rare condition that affects movement and coordination. Researchers will use detailed exams, brain scans, and genetic testing to better understand the disorder and its impact on …

This study will look back at medical records of 45 people with either Wolfram syndrome or a related eye condition caused by changes in the WFS1 gene. Researchers want to see if these two conditions are truly different by comparing vision loss over time and other health problems. …

This study aims to see if wearable sensors and digital health tools can accurately track symptoms of Huntington's disease over 12 months. About 75 adults with and without Huntington's will wear sensors on their wrist, ankle, and pendant to measure daily activity, and complete spe…

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 …