Neurodegenerative disease

This early-stage trial tests whether transplanting lab-grown dopamine-producing cells into the brains of people with Parkinson's disease is safe and might help replace the cells lost to the disease. About 7 adults with Parkinson's who have not responded well to standard medicatio…

This early-stage study tests an experimental drug called ALN-HTT02 in 66 adults with Huntington's disease. The drug is given as a single injection into the spine to see if it is safe and how it affects the body. The goal is to reduce levels of the harmful protein that causes the …

This phase 2 trial tests an experimental drug called VIM0423 in adults with Parkinson's disease whose tremor does not respond well to standard dopaminergic therapy. Researchers want to see if VIM0423 can reduce tremor severity, improve daily function and quality of life, and be s…

This phase 2 trial tests whether donanemab, an antibody given by IV, can slow worsening of thinking and daily function in people with early cognitive decline and Lewy body dementia. The study enrolls 350 participants who also have signs of two brain proteins (amyloid and alpha-sy…

This trial tests whether repetitive transcranial magnetic stimulation (rTMS) can reduce or delay the involuntary movements, called dyskinesia, that many Parkinson's patients develop after taking levodopa. Researchers will apply targeted magnetic pulses to a brain region involved …

This early-stage study tests a new drug called LY3954068 in 48 people with mild Alzheimer's symptoms. The drug is given directly into the spinal fluid, either once or twice, to see if it is safe and how it affects Alzheimer's markers. The study lasts up to 100 weeks and includes …

This study tests whether exercising more frequently (5 times a week for one month, then three weeks off) is better than exercising once a week with no breaks for people with moderate Parkinson's disease. The exercise is a partnered dance program based on Argentine tango, adapted …

This study tests an experimental drug, fosigotifator, in adults and children with Vanishing White Matter disease, a rare genetic brain disorder. The main goals are to check the drug's safety and how the body processes it. About 50 participants will take the drug for up to 201 wee…

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

This study follows about 215 adults with advanced Parkinson's disease who are switching from a pump medication to a new intestinal gel called LECIGON®. Researchers want to see if the gel reduces daily 'off' time—when symptoms return—over 12 months. Participants continue their usu…

This study is testing a new knee implant (Univation XF Pro) in 150 people with knee arthritis. The goal is to see how well the implant works and how safe it is over several years. Participants will have their knee function, pain, and quality of life tracked using questionnaires a…

This early-stage trial tests a gene therapy called scAAV9/JeT-GAN for giant axonal neuropathy (GAN), a rare, fatal nerve disease in children. The therapy is injected directly into the left vagus nerve to target autonomic nervous system symptoms. Only 4 people who have already rec…

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…

This study tests a gene therapy called GS010 for people with Leber hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. Researchers will give the treatment as an injection into the eye at two different doses to see if it improves vision and mitoc…

This study is testing an upgraded amino acid-based nutritional supplement for 30 children and adults who have severe gut problems like inflammatory bowel disease, short bowel syndrome, or severe allergies. Participants will use the supplement as part of their diet for 28 days. Re…

This study tests a brain implant that records brain signals to help people with locked-in syndrome control a computer. Two participants will have 128 electrodes placed on their brain surface. Researchers will train them to use the system at home for one year, aiming for accurate …

This early-stage trial is testing whether a special type of immune cell, called regulatory T cells (Tregs), is safe for people with ALS. The cells are taken from each patient's own blood, multiplied in a lab, and then injected into the spinal fluid. The study will enroll 12 adult…

This phase 2 study tests the drug cobimetinib in children and adults with Langerhans cell histiocytosis and related disorders that have come back or not responded to standard treatment. Cobimetinib blocks a growth signal that goes haywire in these diseases. The study will enroll …

Researchers are studying whether a combination of blood tests, retinal imaging, and brain scans can improve the detection and staging of Alzheimer's disease in Chinese adults. The study enrolls 300 people, including those with probable Alzheimer's dementia and those without. Part…

This study tests whether a radioactive tracer called 18F-S16/T807 can help diagnose tau-related brain diseases. Tau is a protein that builds up abnormally in conditions like Alzheimer's and other neurodegenerative disorders. Researchers will give 500 adults a single injection of …

Researchers are testing whether a radioactive tracer called 18F-DPA714 can help PET/CT scans spot inflammation in the brain. The study includes adults with conditions linked to brain inflammation, such as stroke and neurodegenerative diseases. Each participant receives one inject…

This study tests a new radioactive tracer called [18F]NIDF that binds to tau tangles in the brain, which are linked to Alzheimer's and other neurodegenerative diseases. Researchers will give the tracer to 20 healthy volunteers and patients, then use PET scans to see how well it h…

This study tests whether PET/CT scans using two radioactive tracers, 18F-92/AV45 and 11C-PIB, can detect beta amyloid buildup in the brain. Researchers will enroll 500 adults with suspected or known neurodegenerative diseases linked to amyloid. Each participant receives one injec…

This study is testing a non-invasive eye camera that takes pictures of the retina using different colors of light. Researchers want to see if these images can help detect signs of Alzheimer's, Parkinson's, and other neurodegenerative diseases. About 930 adults over age 30 with or…

This study is testing a new serious game called REALSoCog to see if it can detect social behavior problems in people with Alzheimer's, Lewy body dementia, or frontotemporal degeneration. Researchers will ask 120 patients to play the game on a laptop, which presents 16 social situ…

This study aims to build an AI model that can help doctors diagnose brain diseases like tumors, strokes, and neurodegenerative disorders using MRI scans. Researchers will collect data from 1,000 patients to train and test the AI. The goal is to see if the AI can improve diagnosti…

This study tests whether a virtual reality (VR) platform called VIRADIA can accurately assess neurological and cognitive functions like memory, balance, and coordination. Researchers will compare VR test results with standard paper or in-person tests in 200 healthy volunteers and…

This study tests whether a prenatal education program can help pregnant women learn about and avoid toxic chemicals found in contaminated seafood. The program teaches about seafood benefits, risks, and safe handling. Researchers will measure changes in knowledge and whether women…

This trial tests whether a smartphone app can help people with REM sleep behavior disorder, a condition that often precedes Parkinson's, become more physically active over two years. Participants receive personalized activity goals through the app and track their steps and exerci…

This trial tests whether a smartphone app can help 600 people with Parkinson's-related gene mutations increase their daily physical activity over two years. Participants set their own exercise goals and get feedback through the app. Researchers will measure changes in step counts…

This study tests whether increasing physical activity through a motivational smartphone app can slow cognitive decline in people with isolated REM sleep behavior disorder (iRBD), an early sign of Parkinson's disease and related disorders. Researchers will enroll 130 participants …

Researchers are testing whether a home-based program combining adapted exercise and mindfulness training can improve walking ability and sleep quality in people with rare neurodegenerative diseases such as leukodystrophy, ataxia, and adrenomyeloneuropathy. The study enrolls about…

This Phase 2 study tests an experimental drug, ML-007C-MA, to see if it can safely reduce hallucinations and delusions in people with Alzheimer's disease. About 300 participants aged 55 to 90 will receive either the drug or a placebo. The main goal is to measure changes in psycho…

This study is developing and testing an online platform called LEND that shares personal stories from people with dementia and their carers. The goal is to see if these relatable narratives can boost wellbeing, confidence, and quality of life for both groups. The trial involves i…

This study tests whether custom 3D-printed cutlery handles can help people with neurodegenerative diseases eat more independently. 75 patients in rehabilitation care will try handles in different sizes, designed to fit their grip. Researchers will measure changes in eating abilit…

This study tests a speech therapy program for bilingual Spanish-English or Spanish-Catalan speakers with primary progressive aphasia, a condition that slowly damages language abilities. Sixty participants will receive personalized script training via video sessions, while 30 othe…

This study tests a new drug called EI-1071 in 15 people with mild to severe Alzheimer's disease. The main goal is to see if the drug is safe and tolerable over 4 weeks. Researchers will also check if it reduces brain inflammation using special brain scans. Participants must have …

This study tests whether 8 weeks of mindfulness classes, taken together by people with Parkinson's and their family caregivers, can reduce negative emotions like stress, anxiety, and depression. About 200 Chinese patient-caregiver pairs will either take the classes or continue us…

This study tests a personalized, virtual speech therapy program for people with primary progressive aphasia (a type of dementia that affects language) and their communication partners. The goal is to see if the program improves communication and quality of life. About 30 particip…

This study tests a device that combines magnetic brain stimulation with cognitive exercises to see if it can improve thinking and memory in people with mild Alzheimer's disease. About 180 participants aged 60-85 will receive either real or sham stimulation along with real cogniti…

This study tests personalized speech therapy delivered online for people with primary progressive aphasia (PPA), a condition that slowly destroys language skills. Researchers will enroll 30 adults with mild PPA and tailor exercises to their specific variant. The goal is to see if…

This study tests whether a non-invasive brain stimulation technique called patterned repetitive transcranial magnetic stimulation (prTMS) can reduce bradykinesia—slowness of movement—in people with Parkinson's disease. Researchers will apply the stimulation over a brain area invo…

This 8-week study tests whether a daily supplement called Sabroxy® (made from Oroxylum indicum bark) can improve insulin resistance and memory in adults aged 40-80 with mild cognitive impairment and insulin resistance. About 140 participants will receive either Sabroxy® or a plac…

This study tests a walking meditation program called iWalk for adults with neurodegenerative diseases like multiple sclerosis. The goal is to see if it's feasible and helps improve resilience, stress regulation, and thinking skills. Researchers will enroll 90 participants and mea…

Researchers are building a large database of cognitive, behavioral, lifestyle, and genetic information from people with neurodegenerative diseases such as Parkinson's disease, multiple system atrophy, and progressive supranuclear palsy. The goal is to find biomarkers that help do…

This study gathers medical information from 200 adults with possible nerve, muscle, or autonomic nervous system disorders who are not already in other NIH studies. Participants undergo standard tests like nerve conduction studies, EMG, and physical exams. The data is stored anony…

OPERA is a large observational database that collects health records from people treated for chronic conditions such as HIV, liver disease, inflammatory bowel disease, heart disease, rheumatic disease, and neurodegenerative disease. Researchers use the records to study how these …

This observational study will enroll up to one million adults to understand how health, environment, and lifestyle affect aging and disease. Researchers will link participants' clinical data with information from public records, such as pollution and social factors, to create a d…

This study will follow 1,000,000 adults aged 18 and older for the rest of their lives. Researchers will collect health measurements and track who develops diseases, becomes frail, or dies. The goal is to find which tests best predict how well people age, so doctors can one day of…

This observational study collects health information from up to 500,000 adults with and without Parkinson's disease. Researchers aim to identify risk factors and early signs that predict who may develop the disease and how it progresses. Participants report their symptoms and med…

This early-stage study is testing an experimental drug called LY3439539 in 30 people with mild-to-moderate Alzheimer's disease. The main goal is to see if the drug changes certain proteins in the spinal fluid that are linked to Alzheimer's. Participants will receive the drug thro…

This study follows 600 people with neurodegenerative diseases such as Parkinson's, Alzheimer's, and related conditions over 1 to 10 years. Researchers collect data from routine clinical exams, brain imaging, movement analysis, and blood tests. They use statistical tools and artif…

This study follows 3,000 people with Friedreich ataxia, a rare inherited disease that affects movement and coordination, to map how the condition changes over time. Researchers will measure symptoms yearly using standard rating scales for up to 25 years. The goal is to build a de…

Researchers at the University of Pennsylvania are building a large repository of blood, DNA, and family history data from people with age-related brain diseases like Alzheimer's, Parkinson's, and ALS, as well as their family members. The goal is to support studies into the geneti…

This study screens up to 5,000 healthy volunteers and people at risk for psychiatric or neurodevelopmental disorders like autism. Participants undergo interviews, cognitive tests, and other evaluations to see if they qualify for future research studies at NIH. The goal is to buil…

This study aims to understand brain changes in people with mild cognitive impairment (MCI) and Alzheimer's disease. Researchers will use MRI, EEG, cognitive tests, and blood and stool samples to find differences between healthy individuals and those with cognitive impairment. The…

This study uses advanced MRI (3T fMRI) to look at brain activity in people who have or will have deep brain stimulation (DBS) implants. DBS is a treatment for conditions like Parkinson's disease, tremor, and obsessive-compulsive disorder. The researchers will scan participants un…

This study aims to understand why people with Alzheimer's disease differ from each other and from healthy older adults. Researchers will use advanced brain imaging (7T MRI), brain wave recordings (EEG), and blood and stool samples to look for patterns that might reflect different…

This study uses a special PET scan tracer, [18F]MH.MZ, to measure serotonin 2A receptors in the brains of people with neurodegenerative diseases like Parkinson's. Researchers want to see if these receptors differ between patients with psychosis and healthy people of the same age.…

This observational study, led by the Michael J. Fox Foundation, aims to understand how Parkinson's disease progresses over time. It will follow 4,500 people, including those with Parkinson's, those at risk, and healthy volunteers, collecting clinical, imaging, and genetic data. T…

This study uses advanced PET scans and blood tests to examine brain inflammation and white matter damage in people with corticobasal syndrome, a rare movement and thinking disorder. Researchers will compare these brain changes across different subtypes of the condition and agains…

This study gives a single dose of kisspeptin, a natural hormone, to 40 people with and without Long COVID. Researchers will take frequent blood samples to measure how the brain's reproductive hormone system responds. The goal is to understand if Long COVID disrupts this system, w…

This study collects tissues and MRI scans from 40 adults with ALS to find markers of disease progression. Researchers will compare imaging and genetic data from spinal cord and brain areas. The goal is to better understand ALS and guide future research.

This study uses a special PET scan to look at tau protein buildup in the brain and see if it relates to memory and thinking problems. Researchers will study 900 adults, some with normal memory and some with mild dementia. The goal is to better understand Alzheimer's disease, not …

This study looks at how stroke changes the way the brain handles walking while doing another task, like listening or navigating. Researchers will monitor brain activity in 50 stroke survivors as they walk on different paths and respond to sounds. The goal is to better understand …

This study follows 150 people with early-stage Parkinson's disease to see if the condition starts in the brain or the body first. Researchers will track symptoms like walking problems and thinking changes over 5 years using tests, scans, and questionnaires. The goal is to better …

This study looks at people with brain damage from strokes, tumors, or other conditions to understand why they have specific thinking problems. Researchers will use brain scans and cognitive tests to link brain areas to deficits. The goal is to learn more about how the brain works…

This study aims to uncover the genetic causes of hereditary spastic paraplegia (HSP), a group of rare neurological diseases that cause progressive muscle stiffness and weakness. Researchers at Boston Children's Hospital will analyze DNA from 200 people with HSP to find genetic va…

This study tests whether a non-invasive brain stimulation technique called temporal interference stimulation (TIS) can temporarily improve language processing in people with mild cognitive impairment due to Parkinson's disease, Lewy body disease, or amnestic MCI. Seventy particip…

This study looks at how cholesterol and its byproducts relate to different types of dementia, including Alzheimer's and frontotemporal dementia. Researchers will measure these substances in brain tissue, spinal fluid, and blood from 80 adults aged 40-85. The goal is to find patte…

This study uses non-invasive eye scans (OCT and OCTA) to look for changes in the retina that might be linked to brain diseases like Alzheimer's, Parkinson's, and multiple sclerosis. Researchers aim to find early markers that could help diagnose or track these conditions. The stud…

This study looks at how moderate wine consumption changes tiny particles called extracellular vesicles, which help cells communicate. Eight healthy adults aged 35-65 will drink red wine, white wine, or water, and researchers will analyze their blood for changes in these particles…

This study looks at how flexible brain activity is in people with Lewy body disease, a condition that causes thinking and behavior problems and is often hard to diagnose. Researchers will analyze past EEG recordings from 40 adults to see if certain brain patterns are linked to th…

This study uses a non-invasive brain stimulation technique called TMS to measure brain activity in 52 people with Parkinson's disease. The goal is to see if these measurements can tell the difference between normal thinking, mild cognitive impairment, and dementia, and predict fu…

This study is looking at how people with conditions like schizophrenia, bipolar disorder, depression, and neurological diseases (such as Alzheimer's or stroke) perform on computer-based thinking tests. The goal is to create a detailed map of cognitive strengths and weaknesses acr…

This study looks at whether using a swallow gel or a semi-solid vehicle can help people with neurological conditions swallow pills more easily and safely. Researchers will observe 500 participants, including patients with swallowing disorders and healthy volunteers, as they swall…

This study is creating a large collection of blood, spinal fluid, and urine samples from 800 ALS patients and 200 healthy volunteers. Researchers will also track clinical changes and at-home measures over time. The goal is to provide a valuable resource for scientists worldwide t…

This study tests whether a radio-wave sensor placed in the home can measure sleep, movement, and walking speed in 40 people with mild cognitive impairment or mild Alzheimer's who are taking nicotinamide riboside (a form of vitamin B3). The goal is to find digital biomarkers that …

This study is building a database of information from 300 adults who have had spine surgery using a special device called the Implanet Jazz System. The goal is to collect measurements like pain levels and quality of life to help doctors learn more and improve future treatments. P…

This study uses a special brain scan called PET to measure a protein called tau, which builds up in the brains of people with diseases like Alzheimer's and frontotemporal degeneration. Researchers will look at how tau buildup relates to memory and thinking problems over time. The…

This study uses advanced brain scans (PET, SPECT, and MRI) to find markers of Parkinson's disease in people with certain genetic mutations. Researchers will look at changes in brain chemicals and structure. The goal is to better understand the disease and help develop new treatme…

This study aims to understand how inflammation in the brain works in Parkinson's disease. Researchers will give a mild inflammatory compound (LPS) to 30 volunteers—some with Parkinson's, some with a sleep disorder linked to Parkinson's, and some healthy—and use special PET-MR bra…

This study uses special brain scans (PET, SPECT, and MRI) to look for a marker that shows how Parkinson's disease changes over time. Researchers will scan 44 people with Parkinson's to see if a compound called BU99008 can highlight brain cell activity linked to the disease. The g…

This study uses advanced brain scans (PET and MRI) to examine serotonin activity in people with Parkinson's disease. Researchers will compare brain images from 42 participants at different stages of the disease. The goal is to find biological markers that could lead to new treatm…