Proteostasis deficiencies

This early-stage study tests whether injecting special stem cells into the brain is safe for people with a rare, severe movement disorder called MSA-P. Only 3 participants will receive the treatment, and the main goal is to check for serious side effects within the first month. T…

This early study tests whether the drug CAEL-101/anselamimab can bind to and shrink abnormal protein deposits (amyloid) in people with AL amyloidoma. Five adults with measurable amyloid deposits will receive the drug and be monitored for up to 72 weeks. The goal is to see if the …

This early-phase trial will test whether a special form of resveratrol (JOTROL) is safe and tolerable for people with Parkinson's disease. About 30 participants will receive either a low dose, high dose, or placebo daily for three months. The study will also measure how the drug …

This Phase 2 trial tests a new drug called QLS4131, given under the skin, in combination with other medications for people with malignant plasma cell neoplasms like multiple myeloma. The study will compare different drug combinations to see which works best at shrinking tumors an…

This study tests an experimental drug called elranatamab in people newly diagnosed with AL amyloidosis who have not yet had treatment. The drug helps the body's immune cells find and destroy the abnormal cells causing the disease. Researchers want to see if it can produce deep re…

This early-phase study tests a new treatment called CAR19-BCMA CAR-T for people with certain blood cancers (plasma cell neoplasms or lymphomas/leukemias) that have returned or not responded to at least three prior treatments. The therapy uses a patient's own immune cells, modifie…

This early-stage study tests a new drug, ALT001, in 20 people with a rare brain disease called multiple system atrophy-cerebellar type (MSA-C). The main goal is to check if the drug is safe and tolerable. Researchers will also look for early signs that it might help control the d…

This study tests a new treatment for people with a rare disease called light-chain cardiac amyloidosis that has come back or not responded to other treatments. The treatment uses specially engineered immune cells (CAR-NK cells) from donated umbilical cord blood to target and atta…

This early-phase trial tests a drug called ALT001, a nerve repair protein, in 60 people with multiple system atrophy (MSA), a rare and progressive brain disorder. The study is open-label, meaning everyone knows they are getting the drug, and it focuses on safety and whether the d…

This Phase 2 trial tests a new drug, belantamab mafodotin, combined with standard chemotherapy in 60 adults newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins damage organs. The goal is to see if the combination improves blood and organ responses. The stu…

This study tests whether a person's own stem cells, processed and given by IV, can safely help with multiple system atrophy (MSA) — a rare, worsening brain disease that affects movement and automatic body functions like blood pressure. Fifty adults aged 35 to 65 will receive eith…

This study tests a home-based dual-task training program for people with Parkinson's disease who have mild cognitive impairment and are at high risk for dementia. Participants will cycle on a stationary bike while playing brain-training games on a tablet, three times a week for 3…

This early-phase trial tests a new type of immune cell therapy called GPRC5D CAR-T in 18 people with relapsed or treatment-resistant plasma cell disorders, such as multiple myeloma. The therapy involves giving patients a single infusion of their own modified immune cells designed…

This phase 2 trial tests a drug called sonrotoclax, combined with dexamethasone and sometimes daratumumab, in 39 people with a specific genetic form of AL amyloidosis (t(11;14)). The goal is to see if the treatment can quickly reduce harmful protein levels and improve organ funct…

This pilot study tests a procedure called left atrial appendage occlusion (LAAO) using the Watchman Flx PRO device in 40 dialysis patients with atrial fibrillation. The goal is to see if it safely reduces stroke and major bleeding compared to standard care. Participants will be f…

This study looks at whether eating more protein can help people on dialysis avoid hospital stays. Dialysis removes nutrients like protein from the blood, and current guidelines are based on old research. The study will give some participants a high-protein diet or supplements and…

This study tests a special radioactive tracer called [68Ga]Ga-FAPI-46 for PET scans in 40 people with transthyretin cardiac amyloidosis (ATTR-CM), a condition where abnormal proteins build up in the heart. The goal is to see if this scan can measure scar-like activity in the hear…

This study tests whether an artificial intelligence (AI) program can help doctors identify a rare heart condition called cardiac amyloidosis on routine echocardiograms. The condition causes protein buildup in the heart muscle, leading to heart failure, and is often missed early o…

This early-phase trial tests a new radioactive tracer called [11C]MODAG-005 that binds to abnormal protein clumps in the brain linked to Parkinson's disease and multiple system atrophy. Nine participants (patients with Parkinson's, MSA, and healthy volunteers) will receive the tr…

This study explores whether a PET/MR scan using a radioactive tracer called 18F-Florbetaben can detect amyloid deposits—abnormal protein clumps that can damage organs—in people with certain blood disorders. Participants receive a single injection of the tracer followed by a whole…

This study will test whether a faster type of bone scan (CZT-SPECT) can accurately diagnose transthyretin cardiac amyloidosis, a serious heart condition. Researchers will compare the new scan with the standard method in 200 patients. If it works, it could make diagnosis quicker a…

This study aims to see if a new radioactive dye can detect early signs of a heart condition called amyloidosis in people who carry a TTR gene mutation but have no heart failure symptoms. About 80 adults aged 30-80 will receive an injection of the dye and then have a PET/CT scan t…

This study tests whether a computer model can find signs of a heart condition called cardiac amyloidosis in bone scans that were done for other reasons. Researchers will look back at 57 patients' scans to see if the model can correctly identify those with the disease. If it works…

This study looks for early signs of a heart condition called cardiac amyloidosis in people who have had carpal tunnel surgery on both hands. Researchers will use a special heart scan to check for abnormal protein buildup. The goal is to find the disease earlier so treatment can s…

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 tests a new way to find a hidden heart condition called cardiac amyloidosis in people with atrial fibrillation (AF). Researchers will use AI to analyze EKGs and CT scans to spot early signs of the disease. About 500 adults with AF and certain warning signs will be spli…

This study tests whether a special PET-CT scan using a drug called evuzamitide can detect signs of cardiac amyloidosis in people whose standard heart scans were unclear but who have high levels of a blood marker called TAD1. About 25 adults with heart failure or a genetic risk fo…

This early-phase trial will test a new radioactive tracer called 18F-FCOB04 in 40 people with neurodegenerative diseases like Alzheimer's or Parkinson's. The tracer targets a brain protein called MAO-B and is used with PET scans to see if it can help diagnose these conditions. Th…

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 will test a new intelligent diagnostic system for orthostatic hypotension (a sudden drop in blood pressure when standing up). The system continuously monitors blood pressure, brain blood flow, and heart signals to classify the condition more precisely. Researchers plan…

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 study tests a special helmet that shines near-infrared light on the brain (called transcranial photobiomodulation) in 40 people with Lewy body dementia. Participants use the helmet twice daily for 6 months. Researchers will check thinking skills, symptoms, and quality of lif…

This study tests whether a tailored online support group (VOCALE LBD) can reduce depression in family caregivers of people with Lewy body dementia (LBD). LBD caregivers often face high stress and depression rates. The 8-week program brings small groups together online to discuss …

This study tests whether laser acupuncture can reduce fatigue and improve quality of life in adults with end-stage kidney disease who are on dialysis. Fifty participants will receive either real laser acupuncture or a sham treatment during their regular dialysis sessions three ti…

This study tests whether butylphthalide, a drug used for stroke, can improve symptoms of Multiple System Atrophy (MSA), a rare and serious brain disorder. About 150 adults with MSA will take either the drug or a placebo three times a day for six months. Researchers will track sym…

This study tests whether a structured 5-week cardiac rehabilitation program can improve exercise ability and quality of life in 40 adults with cardiac amyloidosis. Participants will undergo supervised aerobic and strength training five days a week, with tests before and after the…

This early-stage trial will test whether terazosin, a drug currently used for high blood pressure and enlarged prostate, is safe and tolerable for people with dementia with Lewy bodies. Forty participants will receive either terazosin or a placebo for 15 weeks. The study focuses …

This pilot study tests whether listening to music in the evening can help people with Alzheimer's disease or related disorders fall asleep faster and behave more calmly. Researchers will compare music therapy to audiobooks in 48 adults living in secure care units. The goal is to …

This study tests whether increasing the pacemaker's lower rate from 60 to 80 beats per minute improves quality of life and daily function in people with ATTR cardiac amyloidosis. The heart in this condition is stiff and depends on a faster heart rate to pump blood. Thirty-four pa…

This study tests whether a home-based exercise program combined with nutritional supplements can improve walking ability and muscle strength in people aged 70 and older with transthyretin cardiac amyloidosis, a heart condition that causes weakness and frailty. About 102 participa…

This study tests whether adding platelet-rich plasma (PRP) to microneedling improves itching and dark patches in macular amyloidosis better than microneedling alone. Thirty adults with the condition will have each side of their face treated differently, and results will be checke…

This study will test whether wearable sensors can accurately monitor movement problems in people with progressive supranuclear palsy (PSP) and dementia with Lewy bodies (DLB). About 60 participants will wear small sensors on their chest, ankles, and wrists while doing simple task…

This observational study monitors the long-term safety of eplontersen, a treatment for transthyretin amyloidosis, in patients who have had a liver transplant or have severe liver impairment. Researchers will track side effects, lab changes, and serious health events in about 320 …

This study from Stanford University will look at how people react when they learn their Lewy Body Disease biomarker test results. Researchers will interview and survey 40 participants, both with and without symptoms, to measure distress and other responses over time. The goal is …

This study uses advanced CT scans to find out how common blood clots are inside the heart chambers of people with cardiac amyloidosis, a condition where abnormal proteins build up in the heart. Researchers will scan 200 adults diagnosed with this disease to detect clots that may …

This study looks at whether a digital tool called Luscii is practical and acceptable for people with a rare heart condition called ATTR-CM. About 60 adults in Austria, Germany, and Italy will use a smartphone app and home devices to share health information with their care team. …

APT-KIDNEY is a large, flexible clinical trial platform designed to test multiple treatments for advanced kidney disease more efficiently. It will enroll up to 5,000 adults with severe kidney problems or those on dialysis. By using electronic health records and adaptive methods, …

This study is a registry that will collect health information from 1,000 people who carry the gene for hereditary amyloidosis, including those with and without symptoms. Researchers will track who develops the disease and how it progresses, including the need for heart transplant…

This observational study will enroll 200 people—100 with Parkinson's disease, 50 with related conditions, and 50 healthy controls—to find molecular markers in blood, urine, and stool that can distinguish Parkinson's from atypical parkinsonism. Participants provide samples and und…

This study will follow 200 people with suspected or confirmed cardiac amyloidosis, a condition where abnormal proteins build up in the heart and cause heart failure. Over two years, participants will undergo heart MRI scans, exercise tests, and blood work at regular visits. The g…

This study tests how the diabetes drug canagliflozin (Invokana) spreads through the body in 10 dialysis patients with severe kidney disease. Using PET scans, researchers want to see if the drug reaches tissues like the heart and blood vessels, not just the kidneys. The goal is to…

This study aims to develop and test advanced MRI methods to study kidney disease, including in patients with chronic kidney disease, those on dialysis, and kidney transplant recipients. Researchers will also use brain MRI to explore links between kidney function and cognitive pro…

This study looks at whether a painless skin scan can measure long-term inflammation in people with Familial Mediterranean Fever (FMF). Researchers will compare these skin measurements with a blood-based inflammation score called the CALLY index. The goal is to better understand o…

This study will follow 2,800 adults aged 55 and older in China for many years to understand how aging affects the brain and thinking skills. Researchers will collect information on diet, lifestyle, medical history, brain scans, and gut bacteria. The goal is to learn what helps pe…

This study will observe 143 older adults with severe aortic stenosis who are scheduled for a minimally invasive valve replacement procedure called TAVR. Researchers want to find out how many also have cardiac amyloidosis, a condition where abnormal proteins build up in the heart.…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…

This 10-year observational study will follow 10,000 adults with diabetes, metabolic disorders, and related conditions like high blood pressure and fatty liver disease. Researchers will collect routine medical data to identify markers of disease severity and activity. The goal is …