Early-onset progressive encephalopathy-spastic ataxia-distal spinal muscular atrophy syndrome

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This early-stage trial tests a brain-computer interface called BrainGate for people with paralysis from conditions like ALS or spinal cord injury. A small sensor is placed in the brain to interpret movement-related signals, allowing users to control a tablet computer just by thin…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests several already-approved drugs to see if they can slow down motor neuron disease (MND) and help people live longer. About 1150 adults with MND will be randomly assigned to receive one of the study drugs or a placebo. The trial uses a flexible design, allowing ine…

This study compares a new approach to managing infections in nursing home residents with dementia against usual care. The goal is to see if the new method reduces antibiotic use and avoids stressful procedures like hospital transfers. About 750 residents aged 60 and older with de…

This study tests whether a non-invasive brain stimulation technique called TMS can help people with mental illness quit smoking. Forty daily smokers with psychiatric conditions will receive either TMS or standard care. The goal is to see if TMS is practical and effective in real-…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study tests a new approach for people with motor neuron disease (ALS) that combines personalized brain stimulation with mixed reality exercise games. The goal is to slow disease progression and improve quality of life. One hundred adults with mild to moderate ALS will be ran…

This study tests a new PET imaging method that uses 10 to 100 times less radiation than current scans to detect Alzheimer's disease and other dementias. Researchers will scan 200 adults (including healthy volunteers) after injecting a radioactive tracer, aiming to see if shorter …

This study is testing a smartphone app that records and analyzes speech to help diagnose and monitor neurodegenerative conditions like dementia, Parkinson's disease, multiple sclerosis, and motor neuron disease. Researchers will compare speech patterns from 150 people with these …

This study tests whether a safe, non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) can improve working memory in people with dementia. The stimulation is timed to each person's brain waves, measured by EEG, to help brain regions communicate b…

This study tests a device called Walkasins, worn on the lower legs, that vibrates to give sensory feedback about foot pressure. The goal is to see if using it daily for six months improves balance and walking in 200 adults aged 55+ with peripheral neuropathy. Participants will do…

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 program called SKILLS that focuses on common factors across different mental health conditions, rather than treating each disorder separately. About 60 adults with stable psychiatric diagnoses will take part. The goal is to see if this approach reduces symptoms…

This study compares two support programs for family and friends caring for Veterans with dementia after a hospital stay. One program, Hospital GamePlan4Care, offers a handbook, online training, and phone coaching tailored to the caregiver. The other provides general education and…

This study tests whether combining mental exercises (cognitive stimulation therapy) with physical activities (motor stimulation) can improve thinking and mobility in people with mild-to-moderate dementia. About 421 participants aged 65 and older will attend 14 sessions over 7 wee…

This study tests whether listening to personalized music enhanced with binaural beats (sound patterns that may influence brain waves) or spatialized (3D-like) audio can reduce behavioral and psychological symptoms of dementia, such as agitation and anxiety. Researchers will enrol…

This study tests an online education program called iSupport-Korea, designed for family caregivers of people with dementia. About 160 caregivers will be randomly assigned to use the program or receive usual support, and researchers will track changes in their well-being and confi…

This study is testing a culturally adapted online education program called Tele-Savvy for Latino caregivers of people with Alzheimer's or related dementias. The program includes weekly video group sessions and short instructional videos over seven weeks. Researchers want to see i…

This study tests whether a virtual, home-based exercise program can reduce depression and anxiety in family caregivers of people with Alzheimer's disease. Twenty-five caregivers will be randomly assigned to either the exercise program or a wait-list group. The program includes we…

This study is testing a device called ViBandz, which uses small vibrating motors strapped to the body to provide vibration therapy. Researchers want to see if it is easy and comfortable for children with neurological conditions to use at home. The study will involve 30 children a…

This study tests whether cognitive stimulation therapy (CST) delivered by trained volunteers can improve thinking and quality of life for people with mild to moderate dementia. 520 participants in Hong Kong will either receive 7 weeks of volunteer-led CST sessions or continue wit…

This study tests whether a non-invasive brain stimulation technique called tDCS, combined with cognitive training, can improve thinking skills and mood in people with mild cognitive impairment or early dementia. Fifty participants will receive both real and placebo stimulation in…

This small pilot study tests whether repetitive transcranial magnetic stimulation (rTMS), a non-invasive brain stimulation technique, can reduce apathy in people with dementia or mild cognitive impairment. Ten participants will receive rTMS, some also taking the medication methyl…

This study tests a program called Connect-Home ADRD that helps nursing home staff and caregivers support dementia patients when they move to home, assisted living, or long-term care. About 360 patients and their caregivers will take part. The goal is to see if the program reduces…

This study tests a workshop series designed for people aged 60 and older with early-stage memory loss who live alone. The program provides education and skills training via Zoom to reduce stress, improve well-being, and help with future planning. Researchers will gather feedback …

This study is testing whether a virtual reality version of a therapy exercise is practical and helpful for adults (ages 18-65) with psychiatric disorders. Participants will try one VR session and answer questions about their experience. The goal is to see if this approach is acce…

This study is testing a type of talk therapy called Emotion Regulation Therapy (ERT) delivered over video for people in New York who struggle with worry, overthinking, or being hard on themselves. The goal is to see if the therapy helps people better manage their emotions and fee…

This study is testing a mobile app called CarePair that helps family caregivers of people with dementia find services and support. About 80 caregivers will use the app for six weeks to see if it is easy to use and helps reduce stress and depression. The goal is to see if the app …

This study looks at how well and how safely a new sleep medication, lemborexant, works for people who have both a mental health condition and trouble sleeping. About 121 adults will take the drug for 8 weeks, and researchers will track changes in their sleep quality and any side …

This study tests whether combining two types of non-invasive brain stimulation can improve balance and coordination in people with ataxia, a condition that affects movement. Researchers will enroll 30 adults with a confirmed ataxia diagnosis. Participants will receive a combinati…

This study tests whether sailing can help people with psychiatric disorders feel better. Forty adults in France will either take sailing lessons or continue their usual care. Researchers will measure changes in quality of life, mental well-being, and self-esteem to see if sailing…

This study tests a program called COTID, where an occupational therapist visits the homes of older adults with dementia who have recently been hospitalized for a fall. The therapist helps make the home safer and trains the caregiver to support the patient. The goal is to see if t…

This study tests if a liquid medicine called midazolam can help elderly patients with moderate to severe memory problems who refuse care. About 30 hospitalized patients will receive the medicine before care to see if it reduces their resistance. The goal is to make necessary care…

This study looks at whether adjusting deep brain stimulation (DBS) can improve movement, speech, and swallowing in people already implanted with DBS for conditions like tremor, stroke, or traumatic brain injury. Researchers will test hand dexterity, grip strength, facial movement…

This study tests whether a gentle electrical current applied to the scalp (called transcranial electrical stimulation) can improve memory and thinking in people with dementia. Two hundred participants will receive different types of stimulation (tACS or tRNS) while doing memory g…

This study tests a program that teaches family caregivers how to help their loved ones with mild to moderate dementia manage chronic pain. Researchers will work with 60 people (30 patients and their caregivers) to see if the training improves pain coping and quality of life. The …

This study tests whether a personal sound amplifier, along with hearing training, can reduce troubling symptoms like agitation and distress in older adults with Alzheimer's or related dementias who also have hearing loss. Researchers will enroll 300 people aged 60-100 and their c…

This study tests whether mild electrical stimulation on the scalp, combined with memory games on an iPad, can improve thinking and memory in people with dementia. About 100 older adults with mild to moderate dementia will receive either real or fake stimulation to see which works…

This study tests an 8-week online program called STELLA-R that teaches dementia caregivers a simple ABC method to understand and respond to upsetting behaviors. About 238 caregivers in Oregon, Washington, and Idaho will try the program and report how it affects their stress and r…

This study tests an online course for people who have recently become caregivers for a family member or friend with dementia. The program includes weekly video sessions and self-paced materials to help caregivers feel more confident and less stressed. Researchers will measure cha…

This study is adapting a program called SHARE to better support African-American families dealing with early to moderate dementia. Researchers will work with 120 pairs of people with dementia and their caregivers to test the adapted program. The goal is to see if the program is h…

This study explores whether personalised music playlists can help improve behavioural and psychological symptoms in people with dementia. Researchers will work with care home residents and families to create custom playlists and monitor emotional responses. The goal is to develop…

This study aims to better understand and treat people with certain inherited metabolic or genetic disorders. Researchers will use standard medical tests like blood work and imaging to diagnose and care for participants, who may also join other related studies. The goal is to expa…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …

This study at Necker Hospital in Paris is checking how well patients with inherited metabolic diseases follow their daily oral medication routines. About 200 patients (children aged 7 and up, teens, and adults) will fill out a questionnaire during a regular visit. The goal is to …

This study screens 360 adults with neurodegenerative disorders like ALS, frontotemporal dementia, and progressive supranuclear palsy. Researchers will use medical history, physical exams, memory tests, movement analysis, MRI scans, and other tests to find common features and diff…

This study aims to understand the link between genes and symptoms in people with inherited movement disorders. Researchers will study up to 2,500 participants, including patients and their family members, to identify new disease-causing genes and improve diagnosis. The goal is to…

This study enrolls up to 15,000 children with known or suspected endocrine or metabolic disorders, along with their family members. Researchers will collect medical records, blood, saliva, and other samples to identify genetic changes linked to these conditions. The goal is to be…

This study is creating a large collection of blood, urine, stool, and heart tissue samples from 10,000 people with and without heart or metabolic conditions. The goal is to store these samples along with medical information to speed up future research into what causes these disea…

This natural history study is collecting information from up to 4,000 people with movement disorders (like Parkinson's disease or tremor) and their family members. Participants undergo exams, blood tests, and imaging, but receive no new treatments. The goal is to gather data that…

This study aims to find the genetic causes of movement disorders and dementias by analyzing DNA from up to 12,000 participants, including patients and their family members. Researchers will collect blood or saliva samples and look for gene mutations linked to these conditions. Th…

This study looks at early changes in mitochondria (the energy centers of cells) and metabolic markers in blood cells of people with Alzheimer's or related dementias. Researchers will compare these measures between those with and without Alzheimer's to find clues about how the dis…

This study is gathering medical information and samples from up to 5,000 people with neurosurgical conditions like brain tumors, epilepsy, and Parkinson's disease. Participants receive standard care while their data is collected for future research. No new treatments are being te…

This study compares two ways to help adults with mental health issues get follow-up care. One group gets usual care after an emergency room visit, while the other uses a new system that books a psychiatrist appointment within 48 hours through their general doctor. Researchers wil…

This study looks at the mental health of 200 teenagers aged 12 to 16 who attend special educational institutes (ITEPs) in France. Researchers will use psychiatric interviews to find out how many have conditions like depression or anxiety. The goal is to better understand their ne…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study aims to improve suicide prevention for people who move between healthcare and the criminal-legal system. Researchers will link health records, Medicaid data, and jail booking information to identify individuals at risk and coordinate care. Over 1,000 adults with past p…

This study is testing a new, non-invasive ultrasound technique called Tissue Pulsatility Imaging (TPI) to measure tiny brain movements. Researchers will compare these measurements in 300 people with mild versus major cognitive disorders. The goal is to see if brain pulse patterns…

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study aims to see if a quick bedside sensory test can predict which painkiller—pregabalin, duloxetine, or a placebo—works best for people with nerve pain in their feet. About 190 adults with diabetic, chemotherapy-related, or idiopathic nerve pain will try each treatment for…

This study tests a new AI-powered conversation tool called LUMEN. It helps caregivers describe changes in a loved one's memory and thinking. The goal is to see if the tool is easy to use and can improve the quality of information doctors receive. Sixty caregivers and doctors will…

This study tests whether a special training toolkit helps doctors and nurses have better conversations about future medical care with people living with Alzheimer's or related dementias. The researchers will compare clinics that use the toolkit to those that continue usual care, …

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 is testing a web-based platform called LEAD that helps people in the early stages of Alzheimer's or dementia talk about and document their end-of-life care preferences. The goal is to make it easier for them and their care partners to feel confident in making future me…

This study aims to find early markers of Parkinson's disease, multiple system atrophy, and Lewy body dementia by analyzing samples from the nose, blood, and urine. Researchers will compare results from 180 people with these conditions and healthy volunteers. The goal is to improv…

This study is creating a shared database of children who have received deep brain stimulation (DBS) for movement disorders like dystonia, epilepsy, or Tourette syndrome. By pooling data from multiple hospitals, researchers hope to answer key questions about who benefits most and …

This study looks at whether a program with a nurse case manager can help people leaving prison visit a doctor or clinic instead of the emergency room. About 488 adults with a history of substance use and a chronic health condition will take part. Some will get extra support from …

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…

This study is collecting information from 4,000 adults with peripheral neuropathy (nerve damage) to better understand the condition. Researchers will combine standard tests with nerve ultrasound to see if it helps diagnose and track the disease. No new treatments are being tested…

This study is following 1,200 people from two memory clinics in Paris to see how sleep patterns, daily routines, and other personal factors relate to different types and stages of dementia. Participants will wear an activity tracker, answer questionnaires, and have cognitive test…

This study uses MRI scans to look for brain differences in people with metabolic diseases compared to healthy volunteers. Researchers will track changes over time and link them to body fat and other health measures. About 126 adults will take part at Ulm University Hospital. No d…

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 how dementia and mild cognitive impairment (MCI) affect thinking, daily activities, and mood. Researchers will assess 151 adults aged 50-86 with these conditions to better understand their challenges. The goal is to improve care by tailoring treatments to each…

This study is collecting nerve samples and biofluids from 400 people with ALS or peripheral neuropathies to find biological markers for early diagnosis and disease tracking. By analyzing individual cells, researchers hope to identify molecular changes that happen before symptoms …

This study aims to translate the Hypertonia Assessment Tool (HAT) into Turkish and check if it works correctly. The HAT helps doctors identify different types of increased muscle tone in children with motor disorders. Researchers will test the tool on 70 children under 18 who hav…

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 looks at how often young children (ages 1 to 6) with inherited metabolic diseases develop eating disorders when they are on special diets. Researchers will use a feeding scale to measure eating problems in 200 children. The goal is to understand the link between these …

The SMILE study is tracking 10,000 adults aged 18 and older to see how sarcopenia (age-related muscle loss) and metabolic diseases like diabetes influence overall health and lifespan. Researchers will collect data from medical records, tests, and surveys over time. This is an obs…

This study collects blood or brain tissue from families with two or more members diagnosed with Alzheimer's or dementia. The goal is to build a large DNA bank that researchers can use to find genes linked to the disease. No individual results are returned, but the data may lead t…

This study is creating a program called START-VET to help veterans with mental illness or substance use disorders who have recently left prison. Researchers will work with 20 veterans to develop transition materials and tools. The goal is to improve quality of life and reduce cri…

This pilot study is testing whether a simple saliva test can help doctors prescribe mental health medications more safely and effectively. 200 adults in Manitoba who are starting or changing psychiatric drugs will provide a DNA sample. The results will be shared with their doctor…

This study looks at whether training psychiatric care teams to talk about sexual health with young patients (ages 15 and up) increases those conversations. Researchers will survey 770 teens hospitalized for psychiatric disorders about their sexual health and any side effects from…

This study follows 100 children under 18 who are already on a ketogenic diet for conditions like drug-resistant epilepsy, autism, chronic migraine, or brain tumors. Researchers want to see if the diet supports healthy growth, improves symptoms, and boosts quality of life. The die…