Adrenoleukodystrophy

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 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 tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

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 is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

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 Phase 3 study tests whether the drug leriglitazone can help adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The main goal is to see if the drug delays death or the need for permanent breathing support compared to a placebo. About 40 men…

This trial tests a gene therapy for X-linked adrenoleukodystrophy (X-ALD), a rare genetic disease that damages the brain. The therapy uses a modified virus to deliver a working copy of the faulty gene directly into the spinal fluid and bloodstream. Up to 30 patients aged 1 year a…

This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…

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 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 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 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 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 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 at the National Institutes of Health is looking at 1,000 adults with known or suspected hormone gland (endocrine) disorders. The goal is to better understand these conditions and help train doctors. Participants may receive tests, treatments, or surgery as part of thei…

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 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 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 enrolls adults with known or suspected endocrine (hormone gland) disorders, such as diabetes or thyroid disease, to help researchers learn more about these conditions. Participants will undergo medical exams, blood and imaging tests, and possibly genetic testing. The m…

This study is a national registry for people with adrenoleukodystrophy (ALD), a rare genetic disorder. Researchers will collect medical records and survey data from up to 1,000 patients and their families over time to understand how the disease progresses. The goal is to find bet…

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 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 is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

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 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 follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…

This study follows up to 600 people with leukodystrophy, a rare genetic disorder affecting the brain's white matter. Researchers aim to track how the disease progresses, improve diagnosis through genetic testing and brain scans, and evaluate outcomes like hospitalizations and res…

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 tests a special radioactive tracer called [18F]FNP-59 to see if it can safely and clearly show cholesterol activity in the adrenal gland using PET scans. About 24 people will take part, including healthy volunteers and those with known adrenal problems. The goal is to …

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 …

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

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 study collects blood samples from 100 people with rare brain diseases like adrenoleukodystrophy and metachromatic leukodystrophy. Researchers will study immune cells called macrophages to see if they affect how the disease progresses. No treatment is given; the goal is to le…

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…