Hereditary dementia

This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare inherited disease that damages the nervous system. Researchers take a patient's own blood stem cells, add a corrected gene, and infuse them back. The goal is to see if this approach is safe and can slo…

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 trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 1…

This trial tests a non-invasive brain stimulation technique called transcranial temporal interference stimulation (tTIS) in people with spinocerebellar ataxia type 3, a genetic condition that affects coordination and balance. The stimulation targets a deep brain region involved i…

This trial investigates whether combining two behavioral therapies can help people with non-fluent aphasia—a condition where stroke damages the brain's language centers, making speech difficult. The approach uses Melodic Intonation Therapy (MIT), which uses singing-like intonatio…

This trial tests whether deep brain stimulation (DBS) targeting the cerebellum is safe and feasible for people with spinocerebellar ataxia types 1 and 3, a group of inherited disorders that cause progressive problems with movement and coordination. The study will enroll 12 adults…

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 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 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 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 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 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 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 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 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 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 early study tests whether a single session of focused ultrasound—using sound waves to gently stimulate a specific brain region—can safely reduce emotional symptoms like agitation, irritability, and anxiety in people with mild dementia. Fifteen participants aged 45 to 80 with…

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 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 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…