Proteostasis deficiencies

This study tests an experimental drug called ALN-APP in 200 adults with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. The drug is given via spinal injection and aims to slow disease progression and reduce new brain bleeds. Participants will be …

This trial tests a device called Lixelle, which attaches to a standard dialysis machine to filter out beta-2 microglobulin, a protein that builds up in the blood of people on long-term dialysis and can cause painful joint and bone deposits. The study includes 40 adults on thrice-…

This phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…

This trial tests whether delivering interdisciplinary care via video telehealth can improve access and satisfaction for people with Lewy body dementia (LBD) and their caregivers. Participants receive remote consultations with a team of specialists, including a neurologist, therap…

This Phase 2 trial tests the safety and effectiveness of YA-101, an oral drug, in 75 people with multiple system atrophy (MSA), a rare and progressive brain disorder. Participants will receive either YA-101 or a placebo twice daily for several weeks. The study will monitor side e…

This Phase 3 trial tests an experimental drug called ALXN2220 in about 1,180 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart, causing heart failure. The drug is designed to remove those protein deposits. Partic…

This phase 2 trial tests whether daratumumab, an antibody that targets cancer cells, can improve outcomes for people with multiple myeloma after they receive a stem cell transplant. The study involves 31 participants and is currently active but not recruiting. The main goal is to…

This early-stage trial tests a combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…

This phase 2 trial is testing whether combining daratumumab, venetoclax, and dexamethasone can produce a deep and rapid response in people newly diagnosed with light-chain (AL) amyloidosis who carry a specific genetic marker called t(11;14). The study enrolls 36 participants and …

This study tests a drug called ACP-204 for treating psychosis (hallucinations or delusions) in people with Lewy body dementia. It is a 52-week open-label extension, meaning all 126 participants will receive the drug. The main goal is to check for side effects over the long term. …

This study tests an experimental drug, NPI-001 (AT-001), in people with hereditary cystatin C amyloid angiopathy (HCCAA), a rare genetic condition that causes repeated brain bleeding and stroke. The trial includes about 15 participants aged 12 and older who carry the specific L68…

This study tests a drug called teclistamab in 30 people with AL amyloidosis, a rare disease where abnormal proteins damage organs. Participants have already had other treatments. The goal is to see if teclistamab can reduce or eliminate the harmful proteins in the blood. The stud…

This early-phase trial tests whether adding the drug isatuximab to standard chemotherapy can safely treat people with high-risk AL amyloidosis, a rare disease where abnormal proteins damage organs. Eleven participants will receive the combination to see if it reduces toxicity and…

This study tests a new treatment called NXC-201 CAR-T for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy the cells producing these proteins. The trial is open to …

This Phase 3 trial tests an experimental drug called amlenetug in 401 people with multiple system atrophy (MSA), a rare and serious brain disorder. The goal is to see if the drug can slow the worsening of symptoms and improve survival. Participants receive either the drug or a pl…

This phase 2b trial tests whether adding ALXN2220 to eplontersen works better than eplontersen alone for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 326 participants will receive either the combinati…

This study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find th…

This study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …

This study tests an experimental drug called ONO-2808 in people with multiple system atrophy (MSA), a rare and serious brain disease. About 92 participants will receive either the drug or a placebo daily for up to 80 weeks. The goal is to see if the drug is safe and might help co…

This phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

This phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…

This Phase 2 trial tests whether a patient's own stem cells, injected into the spinal fluid, can slow the progression of multiple system atrophy (MSA), a rare and fatal brain disease. The study involves 71 adults aged 30-70 with early-stage MSA. Researchers will measure changes i…

This study tests a drug called eplontersen in Chinese adults with a heart condition caused by abnormal protein buildup. The goal is to see if the drug lowers that protein and is safe over time. Participants will be randomly assigned to receive the drug or a placebo for 24 weeks, …

This study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…

This expanded access program offers the experimental drug zervimesine (CT1812) to people aged 50 to 86 with mild-to-moderate dementia with Lewy bodies. Participants take one 100 mg pill daily for up to a year. The goal is to provide access to the drug and collect long-term safety…

This study is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…

This trial aimed to test an experimental drug called CM-336 for people with AL amyloidosis that has returned, not responded to treatment, or only partially responded after initial therapy. The drug works by helping the immune system attack and destroy harmful cells. However, the …

This study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidne…

This study tests an experimental drug called Lu AF82422 in 64 people with multiple system atrophy (MSA), a rare and serious brain disease. The goal is to see if the drug can slow down the worsening of symptoms like movement problems and daily living difficulties. Participants rec…

This study tests whether a person's own stem cells can be safely injected into the spinal fluid to treat multiple system atrophy (MSA), a rare and serious brain disease. About 30 adults aged 30-80 with MSA will receive the treatment. The main goal is to check for side effects, an…

This study tests whether adding daratumumab back to pomalidomide and dexamethasone can improve complete remission rates in 15 adults with relapsed or refractory AL amyloidosis who have already received daratumumab. Participants receive the three-drug combination to control the di…

This Phase 2 trial tests whether a sleep-deepening drug (lower-sodium oxybate) or a nerve-stimulating device (non-invasive vagus nerve stimulation), or both, can help clear amyloid-beta protein from the brain in people with cerebral amyloid angiopathy (CAA). Sixty participants wi…

This study is for people with a rare heart condition called ATTR amyloidosis with cardiomyopathy, where abnormal protein builds up in the heart. It tests the long-term safety and effectiveness of a drug called vutrisiran, given as an injection every three months. About 700 adults…

This study aimed to test a monthly injection called ION-682884 in people with TTR amyloid cardiomyopathy, a condition where abnormal protein deposits damage the heart. Only patients who had already completed a 2-year study of a similar drug (inotersen) were eligible. The goal was…

This early-stage trial tests a gene therapy called AAV2-GDNF for multiple system atrophy (MSA), a rare and serious brain disorder. Nine adults with MSA will receive either the gene therapy or a sham surgery. The main goal is to check safety, but researchers will also look for any…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests whether using focused ultrasound to temporarily open the blood-brain barrier can safely help standard antibody therapy reach the brain in people with mild Alzheimer's or mild cognitive impairment. About 15 participants will receive the combined treatment. The goa…

This study tests a drug called vutrisiran in 655 adults with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The drug is given as a shot every 3 months and aims to reduce deaths and heart-related hospital stays. The goal is to see…

This study tests whether a second round of treatment with ALXN2220 is safe and can improve heart function in people with transthyretin amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 35 adults who previously received the drug will get it a…

This study looks at the long-term safety and effects of a drug called eplontersen in people with a rare inherited nerve disease called hATTR-PN. About 151 participants who completed earlier studies will receive regular injections of eplontersen. The goal is to see if the drug rem…

This phase 2 trial is testing whether the oral chemotherapy drug ixazomib can help control AL amyloidosis after initial treatment. Seventeen adults with the disease will take ixazomib along with low-dose dexamethasone to see if it delays organ progression or the need for more the…

This study tests whether the drug idebenone can slow or prevent the progression of a sleep disorder called iRBD into Parkinson's disease or similar brain conditions. About 142 adults aged 40-75 with iRBD will receive either idebenone or a placebo for 5 years. Researchers will tra…

This study tests the long-term safety of an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy, a rare heart disease that can lead to heart failure. All 80 participants have already completed an earlier study and will receive the drug every …

This study is checking the long-term safety of a drug called acoramidis in people with a rare heart condition called transthyretin amyloid cardiomyopathy. About 389 participants who finished a previous 30-month trial will take acoramidis twice daily for up to 5 years. Researchers…

This study tests a new, more stable version of the chemotherapy drug melphalan in people with multiple myeloma or AL amyloidosis who are getting a stem cell transplant. The goal is to find the best dose that reaches a target level in the body, while possibly causing fewer side ef…

Researchers at Beijing Anzhen Hospital are studying whether a computer model that combines two types of heart scans can predict 12-month survival in people with light-chain cardiac amyloidosis, a condition where abnormal proteins stiffen the heart. The study enrolls 49 patients w…

This study will test if artificial intelligence (AI) can spot a rare heart condition called ATTR-CM in people with heart failure. Researchers will use AI to analyze electrocardiograms (ECGs) and echocardiograms (Echo) from 150 adults. The AI's findings will be checked against a s…

This study investigates whether a combination of skin biopsies, speech analysis, and ultra-high field 7T MRI can detect early signs of Parkinson's disease and dementia with Lewy bodies. It focuses on people with a specific type of REM sleep behavior disorder triggered by serotone…

This study enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This study tests an AI tool called AIDP that analyzes MRI scans to help diagnose Parkinson's disease and two similar conditions (MSA and PSP). Researchers at 21 sites will compare the AI's diagnosis to that of movement disorder specialists. The goal is to see if the tool can impr…

This study tests whether a special heart scan using a radioactive drug called 123I-MIBG can help identify Lewy body disease, a type of dementia that also affects movement and sleep. Researchers will compare scan results with clinical diagnoses in 50 people who have conditions lik…

This study is testing whether artificial intelligence can detect transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often underdiagnosed heart condition, earlier than usual. Researchers will use AI algorithms to analyze routine ECGs and heart ultrasounds from up to 1.5…

This trial tests whether an artificial intelligence tool called EchoNet-LVH can help screen for cardiac amyloidosis, a rare but serious heart condition that is often misdiagnosed. The AI analyzes routine echocardiograms to flag suspicious patterns that might indicate the disease.…

This study tests whether a radioactive tracer called 124I-evuzamitide can detect amyloid deposits in the heart and elsewhere in people aged 60 and older who have lumbar spinal stenosis or carpal tunnel syndrome. Participants will receive a single dose of the tracer and undergo a …

This study uses special brain scans (PET imaging) to measure amyloid buildup—a protein linked to Alzheimer's—in healthy older adults, people with mild memory problems, and those with dementia. Participants receive scans every 2-3 years for up to 20 years to see how amyloid change…

This study compares a new ultra-high resolution PET/CT scanner, the NeuroEXPLORER, with standard clinical PET/CT scanners for imaging the head and neck. Researchers aim to see if the new device provides more detailed images to improve diagnosis of conditions like dementia, Parkin…

This study is looking for better ways to diagnose Parkinson's disease using samples that are easy to collect, like blood, skin, and nasal swabs. Researchers will compare these samples from 340 people, including those with Parkinson's, Alzheimer's, and healthy volunteers. The goal…

This study uses a special brain scan called DaTscan to look at how certain brain diseases affect the brain. Researchers will scan 500 adults with conditions like Parkinson's disease, dementia, mild cognitive impairment, or REM sleep behavior disorder. The goal is to see if DaTsca…

This study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…

This study aimed to test a computer algorithm that uses insurance claims data to identify people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers planned to check how well the algorithm worked by reviewing medical records. However, the study wa…

This study tests a new drug called CST-3056 in 12 adults with neurogenic orthostatic hypotension (a sharp drop in blood pressure when standing) caused by Parkinson's disease or pure autonomic failure. The goal is to see if the drug can improve blood pressure and reduce symptoms l…

This study tests a new way to match dementia caregivers with each other for emotional support using a computer algorithm. Researchers want to see if matching based on personal preferences leads to better satisfaction and less stress than random matching. About 355 caregivers of p…

This study tests a biodegradable tube that is placed over a cut nerve during a leg biopsy. The goal is to see if it is safe and reduces common side effects like pain, redness, and swelling. Twenty adults with various nerve conditions are taking part, and half will get the tube wh…

This study tests whether a home-based telenursing program, led by a specialist nurse, can improve quality of life and reduce complications like falls for people with atypical parkinsonism (MSA or PSP). About 164 participants will receive proactive and reactive remote monitoring a…

This study tests a 12-week program called PERSEVERE for 502 family caregivers of people with Lewy body dementia (LBD). Caregivers are matched with a trained peer mentor who guides them through lessons on LBD and provides support. The goal is to see if this reduces caregiver strai…

This study looks at whether online community support and telemedicine can improve quality of life for people with Parkinson's disease, Lewy body dementia, and related conditions, as well as their caregivers. About 632 participants will receive palliative care training for their d…

This Mayo Clinic study tests a new device designed to clear fluid and debris from surgical drain tubes more effectively than the current method of using fingers. The trial will enroll 500 adults who have abdominal surgery with Jackson-Pratt drains. Nurses will rate how easy the d…

This study tests a new device called the Catheter Caps Case (C3) for dialysis patients. The goal is to see if it is more comfortable and easier to use than the usual gauze and tape. Ten adults with a dialysis catheter will try the device for 15 minutes, and researchers will measu…

This study is testing whether a daily supplement made from baker's yeast can improve digestion and reduce gut symptoms in healthy adults aged 50 to 99. Participants take 15 grams of the supplement each day and report any changes in how their stomach feels. The goal is to see if t…

This study tests whether a drug called siltuximab can reduce symptoms like weakness, fatigue, nausea, and pain after a stem cell transplant in patients aged 60-75 with multiple myeloma or AL amyloidosis. The drug blocks a protein linked to inflammation, which may help patients re…

This phase 3 study tests a drug called ampreloxetine for people with multiple system atrophy (MSA) who experience dizziness and fainting due to low blood pressure when standing. About 102 adults with MSA will take the drug for 20 weeks, then some will switch to placebo to see if …

Researchers in Argentina are developing and testing a self-administered questionnaire to measure how hereditary transthyretin amyloidosis affects patients' physical, emotional, and social quality of life. The study includes input from neurologists, a cardiologist, a psychologist,…

Researchers are checking whether tests of muscle strength, walking, balance, and fine motor skills give consistent results in people with polyneuropathy. About 520 adults with various forms of nerve damage, plus healthy volunteers, take part. Participants complete physical tests,…

This pilot study tests a program that screens older dialysis patients for common age-related problems like falls, memory trouble, and medication issues. A geriatrics team then gives personalized recommendations to the dialysis clinic. The goal is to see if this approach is practi…

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 a disease registry that collects information from routine medical care of about 350 adults diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Taiwan, Hong Kong, and Malaysia. Researchers will look at patient characteristics, treatments received, and ho…

This study follows 20,000 healthy Chinese adults aged 40-75 living at various altitudes to see if chronic low oxygen levels increase the risk of multiple system atrophy (MSA), a rare and fatal brain disease. Participants undergo checkups and overnight oxygen monitoring, then are …

This study uses anonymous health insurance records from Germany to learn more about a rare heart condition called ATTR-CM, where abnormal protein builds up in the heart. Researchers will look at survival rates, hospital visits, and how common the disease is. No new treatments are…

This observational study is looking at a protein called neurofilament light chain (NfL) in the blood of people who carry a gene variant for hereditary ATTR amyloidosis, both those without symptoms and those with nerve damage. Researchers want to see if NfL levels can help detect …

This study investigates whether people with neurodegenerative diseases—like Alzheimer's, Parkinson's, or ALS—show changes in behavior, social circumstances, or health before and after their diagnosis. Researchers will compare the records of over 61,000 patients with those of matc…

This study follows people who previously received NTLA-2001, an experimental gene-editing therapy, for a type of amyloidosis that can affect nerves or the heart. The goal is to monitor long-term safety and see if the therapy's effects on disease markers persist over time. Partici…

This study follows 240 patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) to monitor the long-term safety of the drug TEGSEDI. Researchers will compare patients who have taken TEGSEDI with those who have not, focusing on side effects like low platel…

This study examines walking patterns in people with Parkinson's disease and similar neurological conditions, as well as healthy volunteers. Researchers aim to identify unique 'gait signatures'—speed-dependent measures of walking—that could help distinguish between different disor…

This study tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …

This study tests a short guide called the 'Jumpstart Guide' to help doctors have better conversations with dementia patients and their families about healthcare goals. Over 1,800 patients and their caregivers are taking part. Researchers will compare whether those whose doctors u…

This study aims to identify biomarkers—biological clues in the body—that can help distinguish between Parkinson's disease and similar conditions like progressive supranuclear palsy and multiple system atrophy. Researchers will follow people with early-stage parkinsonism and healt…

This study uses special PET/CT scans to measure inflammation in the brains of people with Alzheimer's disease or Lewy body dementia. Researchers want to see if brain inflammation is linked to memory decline and other dementia symptoms. The study also tests a new radioactive trace…

This study enrolls 500 adults with a sleep condition called REM sleep behavior disorder (RBD), which often leads to Parkinson's disease or related brain disorders. Researchers will track symptoms like thinking, movement, and smell to develop a rating scale. The goal is to prepare…

This study follows 96 people with end-stage kidney disease who need a brachiocephalic fistula for dialysis. Researchers collect data from exams, blood tests, and imaging to track changes in the vein over time. The goal is to learn what causes cephalic arch stenosis, a common narr…

This study looks at whether advanced monitoring devices can accurately measure blood thickness (hematocrit) and oxygen levels during continuous dialysis in critically ill patients with acute kidney injury or end-stage kidney failure. About 30 adults in the intensive care unit wil…

This study looks at whether high-intensity cycling during hemodialysis can protect the heart from damage that dialysis sometimes causes. Fifteen adults on dialysis will exercise at different intensities during their sessions. Researchers will use echocardiograms to measure heart …

This study looks at how things like social support, income, and health habits are linked to mental health and brain diseases such as depression, Alzheimer's, and Parkinson's. Researchers will collect surveys, blood samples, and optional brain scans from over 1,300 adults in Europ…

This study follows 135 people with Lewy body dementia over up to 5 years, using special PET scans to measure brain changes linked to memory and thinking decline. Researchers hope to find imaging markers that show how the disease progresses. Participants will have regular tests an…

This study looks at how the body's automatic nervous system controls blood flow in abdominal veins and how that might lead to high blood pressure in people with Multiple System Atrophy (MSA). Researchers will measure vein responses in about 16 participants aged 40-80 who have MSA…

This long-term study uses yearly brain scans, genetic tests, and thinking/memory assessments to track changes in nearly 1,800 people with various dementias (like Alzheimer's and vascular dementia) and over 140 healthy older adults. The goal is to better diagnose, monitor, and eve…

This study is analyzing blood samples from 1,799 people with Alzheimer's, dementia, depression, bipolar disorder, schizophrenia, or Parkinson's disease. Researchers want to see if certain substances in the blood can help doctors diagnose and manage these conditions more accuratel…

This study creates a database of 476 patients in the Bronx who were tested for transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often missed heart condition. Researchers collected health records, imaging results, and outcomes like hospitalizations and deaths. The goa…

This study follows 20 adults with hereditary transthyretin amyloidosis (ATTRv) who have both nerve and heart involvement. They are treated with patisiran or vutrisiran for two years. Researchers use MRI scans and questionnaires to see how the drugs affect heart structure and func…

This study looks at 50 people with TTR amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. Researchers want to see if a special SPECT scan can measure blood flow in the heart's tiny vessels, which may be impaired even without blocked arteries. Parti…

This study follows 90 people with Multiple System Atrophy (MSA), a rare brain disease, for 12 months. Researchers measure changes in movement, thinking, brain scans, and blood markers to better understand how the disease progresses. The goal is to gather information, not to test …

This study aims to learn more about diseases that affect the brain, such as Parkinson's disease and different types of dementia. Researchers will follow 500 adults with symptoms of these conditions over time, using brain scans and genetic tests to see how the diseases progress. T…

This study follows 132 people with Parkinson's disease or multiple system atrophy who previously received the gene therapy AB-1005. Researchers will track safety and how well the treatment works over the long term. No new treatments are given; it's purely observational.

This study uses monthly MRI scans to closely watch brain changes in 75 people with cerebral amyloid angiopathy (CAA), a condition common in older adults that can cause bleeding and strokes. Researchers aim to understand how small bleeds and silent strokes develop over time. Parti…