Proteostasis deficiencies

This study tests a new treatment called AZD0120 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 harmful cells. The study aims to see if it is safe and effectiv…

This study tests whether switching from tafamidis to acoramidis can raise blood levels of transthyretin (TTR) in adults with transthyretin amyloid cardiomyopathy (ATTR-CM). The drug acoramidis stabilizes TTR to prevent harmful amyloid clumps that stiffen the heart. Researchers wi…

This study tests whether semaglutide, a diabetes drug, is safe and tolerable for people with type 2 diabetes who are on dialysis. About 100 participants will either receive weekly semaglutide injections or standard care for 26 weeks. The goal is to see if patients can stick with …

This phase 2 trial tests whether dapagliflozin, a diabetes drug, is safe and can lower high urine protein levels in people with renal AL amyloidosis—a rare condition where abnormal proteins damage the kidneys. About 20 participants will take the drug daily for 6 months and be mon…

This study is testing whether stem cells taken from a person's own bone marrow and then given back through the veins and nose can improve brain and nerve function. It includes 500 people with various conditions like stroke, Parkinson's, Alzheimer's, and nerve damage. The goal is …

This study tests an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 1,280 adults will receive either the drug or a placebo by IV, alongside their usual heart trea…

This study tests a traditional Chinese medicine called Jiedu Huayu oral prescription in 436 people who have had a brain bleed due to a condition called cerebral amyloid angiopathy. The goal is to see if the herbal treatment can lower the chance of having another stroke or blood v…

This study tests an experimental drug called linvoseltamab in 220 people with AL amyloidosis that has returned or not improved after prior therapy. The drug aims to reduce abnormal proteins that damage organs like the heart and kidneys. The trial first finds the safest dose, then…

This phase 2 trial tests whether donanemab, an antibody given by IV, can slow worsening of thinking and daily function in people with early cognitive decline and Lewy body dementia. The study enrolls 350 participants who also have signs of two brain proteins (amyloid and alpha-sy…

This study tests whether Enavogliflozin, a drug used for diabetes, can help people with amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 68 adults with heart failure symptoms will take the drug or a placebo for 12 weeks, then switch. The ma…

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 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 is testing the long-term safety of an oral drug called Emrusolmin (TEV-56286) in 200 adults with multiple system atrophy (MSA), a rare and serious brain disorder. Participants who completed a prior trial will take the drug for about 100 weeks. The main goal is to see h…

This study tests an experimental oral drug, TEV-56286, in 350 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. Participants will receive either the drug or a placebo for 48 weeks to see if it slows disease worsening and is safe. The goal is to fin…

This study tests whether daily weight monitoring at home can reduce hospitalizations for heart failure in people with cardiac amyloidosis, a condition where protein buildup stiffens the heart. 320 adults with ATTR cardiomyopathy will either use a connected scale that alerts docto…

This early study tests a nasal spray called foralumab in 5 adults with multiple system atrophy (MSA), a rare brain disease that gets worse over time. The goal is to see if the drug can reduce harmful brain inflammation and slow down symptoms like movement problems. Researchers wi…

This Phase 3 trial tests a new drug called nucresiran in 125 people with a rare inherited condition that damages nerves (hATTR-PN). The drug is given as a shot under the skin every 6 months and aims to slow nerve damage and improve quality of life. Researchers will compare it to …

This Phase 3 study tests a drug called nucresiran in 1250 people with ATTR amyloidosis, a rare disease where abnormal proteins damage the heart. Participants receive either nucresiran or a placebo every 6 months. The goal is to see if the drug reduces deaths and heart-related hos…

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 study tests whether combining two drugs, teclistamab and daratumumab, can help people newly diagnosed with both multiple myeloma and light chain amyloidosis. About 30 adults will receive the treatment for up to 24 cycles. The goal is to see if the combo improves survival wit…

This early-phase trial is testing a new type of gene therapy called CAR 70-BCMA dual-target CAR-T for people with relapsed or refractory plasma cell neoplasms, including multiple myeloma. The therapy involves taking a patient's own immune cells, re-engineering them to recognize a…

This early-phase trial tests a personalized cell therapy called BCMA-targeted CAR-T cells in 30 adults with relapsed or refractory light chain amyloidosis, a rare disease where abnormal proteins build up in organs. The treatment uses the patient's own immune cells, modified to at…

This early-phase study tests an experimental drug called JNJ-79635322, a trispecific antibody designed to attack cancer cells in people with relapsed or refractory multiple myeloma or AL amyloidosis. About 180 participants will receive the drug to find a safe dose and check for s…

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 an experimental drug called etentamig (ABBV-383) in adults with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 76 participants will receive the drug by infusion to see if it is safe and can control the disease. The trial has two …

This study tests a drug called belantamab mafodotin in people with a rare blood disease called AL amyloidosis that has come back or not responded to other treatments. The goal is to find the safest and most effective dose. The study has two parts: first, different doses are teste…

This study tests a new drug called TQB2934 for people with systemic light chain amyloidosis, a rare disease where abnormal proteins damage organs. The drug is a bispecific antibody that helps the immune system target and destroy harmful cells. The trial will enroll 70 adults who …

This Phase 2 trial tests whether ambroxol, a common cough medicine ingredient, can slow cognitive decline and improve symptoms in people with early dementia with Lewy bodies. 180 participants will receive either ambroxol or a placebo for 18 months. The study measures changes in t…

This phase 3 trial tests whether adding a stem cell transplant to standard chemotherapy helps people with newly diagnosed AL amyloidosis. About 338 participants will receive either chemo alone or chemo followed by a stem cell transplant. The goal is to see which approach better d…

This early-phase trial is testing a new treatment called GC012F for people with AL amyloidosis that has come back or not responded to other therapies. GC012F is a type of immunotherapy that uses a patient's own immune cells, modified to attack two specific targets on the abnormal…

This early-stage trial tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. The drug is given via a spinal injection and aims to reduce levels of a protein linked to the disease. The main goal is to check…

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 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 study offers expanded access to an experimental stem cell treatment for up to 7 adults with Multiple System Atrophy (MSA), a rare and serious brain disorder. Participants will receive 12 intravenous infusions and 6 spinal injections of donor stem cells over 44 weeks. The goa…

This phase 3 trial tests a single dose of NTLA-2001, a gene-editing therapy, in 60 adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. Participants are randomly assigned to receive either the active treatmen…

This early-stage trial tests a single injection of dopamine-producing cells directly into the brain for people with multiple system atrophy (MSA), a rare and severe movement disorder. The main goal is to check safety and side effects in 9 participants aged 30 to 70. Researchers w…

This program offers ongoing access to the experimental drug ATH434 for people with multiple system atrophy (MSA) who completed a prior Phase 2 study. Participants take the drug twice daily by mouth. The goal is to allow continued treatment for those who may benefit, as judged by …

This early study tests a drug called coramitug in 14 people with ATTR-CM, a heart condition caused by abnormal protein buildup. The drug is designed to bind to and help remove these protein clumps from the heart. Researchers will use special scans to see where the drug goes and w…

This phase II trial at Mayo Clinic is testing whether longer maintenance therapy with daratumumab can improve survival for people with AL amyloidosis. About 96 adults who have already responded to initial daratumumab treatment will be randomly assigned to shorter or longer mainte…

This early-stage study tests if implanting a small piece of the participant's own leg nerve into a key brain area is safe and feasible for people with early Parkinson's disease or Multiple System Atrophy. Seven people will take part; four will get the nerve graft and three will h…

This observational study follows up to 2,000 adults with ATTR-CM, a serious heart condition, who are starting treatment with acoramidis as part of their normal care. Researchers will track patient characteristics, how the drug is used, and its effects on heart function and qualit…

This study tests a single dose of NTLA-2001, a gene-editing therapy, in 1200 adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart. The goal is to see if it reduces heart-related deaths and events compared t…

This phase 2 trial is testing a drug called CM336 in 21 people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. CM336 is a bispecific antibody that helps the immune system target and destroy the cells making these proteins. The study…

This study tests a personalized approach for people with AL amyloidosis who have a specific genetic change called t(11;14). All 41 participants start with a standard three-drug combination. If their blood responds quickly within a week, they continue that treatment; if not, they …

This study tests a new drug called MSA-01 to see if it can slow the worsening of multiple system atrophy (MSA), a rare and serious brain disease. About 140 people with MSA will get either MSA-01 or a placebo for 12 months. The main goal is to measure changes in movement and daily…

This study tests a drug called elranatamab in people with AL amyloidosis that has come back or not responded to prior treatment. The goal is to find a safe dose and see if the drug can reduce abnormal protein levels. About 49 adults will take part in this early-phase trial.

This phase 3 trial tests whether adding the experimental drug SCTC21C to standard chemotherapy (bortezomib, cyclophosphamide, dexamethasone) works better than chemo alone for people newly diagnosed with AL amyloidosis. The study will enroll 90 participants and measure how well th…

This program offers early access to venetoclax, a targeted cancer pill, for people with certain blood cancers like leukemia or lymphoma who have no other good treatment options. A doctor must decide if the potential benefit outweighs the risks. The drug is not yet approved in all…

This phase 3 trial tests whether AP301, a drug taken with meals, can safely lower high blood phosphate in people on kidney dialysis. About 264 participants will receive either AP301 or a low-dose version for 8 weeks, then all get AP301 for 24 weeks. The goal is to see if AP301 he…

This study tests a medication called acoramidis in 200 people with a rare heart condition called transthyretin cardiac amyloidosis. The goal is to see if the drug can slow down the disease and prevent worsening of heart function. Participants either are newly diagnosed or switch …

This phase 2 trial is testing whether a powerful four-drug combination can achieve such deep cancer remission that newly diagnosed multiple myeloma and amyloidosis patients can safely delay or avoid a stem cell transplant. Forty participants will receive six cycles of the drug co…

This study tests an experimental drug called CM336 in 90 adults whose primary light-chain amyloidosis has come back or not responded to prior treatments. The drug is designed to help the immune system attack the abnormal cells causing the disease. The main goal is to see how many…

This early study tests a spinal cord stimulation therapy called ARC-IM in 5 people with Parkinson's disease or related conditions who experience severe drops in blood pressure when standing (orthostatic hypotension). The main goal is to check safety, and researchers will also mea…

This study tests whether two drugs, teclistamab and daratumumab, can safely and effectively treat AL amyloidosis, a rare disease where abnormal proteins damage organs. The treatment lasts about 6 months. Researchers will check if the drugs can make the abnormal proteins disappear…

This study tests whether a supplement called tributyrin can improve memory, thinking, and walking in people with Parkinson's disease who also have cognitive problems. 45 participants will take either tributyrin or a placebo three times daily for about 90 days. Researchers will me…

This early-phase trial tests a drug called Aleeto, a nerve repair protein made from stem cells, in 20 people with Multiple System Atrophy (MSA), a rare brain disease. The study checks if Aleeto is safe and might help with symptoms. Participants receive the drug via injection into…

This study tests a new radioactive tracer called 18F-mFBG to see if it can detect nerve damage in the heart caused by Lewy body diseases like Parkinson's and Lewy body dementia. About 20 adults will get a PET scan after an injection of the tracer. The goal is to see if the scan c…

This early-stage study tests a new radioactive tracer called SST001 that lights up on PET scans. Researchers want to see if it is safe and can help tell the difference between multiple system atrophy (MSA) and Parkinson's disease. The study will include 30 healthy volunteers and …

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 is testing a cloud-based AI platform called Willem that analyzes electrocardiogram (ECG) data to detect Transthyretin cardiac amyloidosis (ATTR-CA), a rare and serious heart condition. Researchers will compare ECGs from 2000 adults—some with confirmed ATTR-CA and some …

This early-phase study tests a new radioactive tracer called [¹⁸F]MODAG-009 to see if it can safely highlight abnormal protein clumps in the brains of people with Parkinson's disease or multiple system atrophy (MSA). About 13 participants, including healthy volunteers, will recei…

This study looks at whether taking a small piece of ligament during routine carpal tunnel surgery can help diagnose transthyretin amyloidosis, a serious condition that often goes unnoticed until it affects the heart. Researchers will compare this new biopsy method to the standard…

This study looks at tissue removed during back surgery to find early signs of a heart condition called ATTR cardiac amyloidosis. Researchers will test spine samples from 1,663 older adults for abnormal protein deposits. If found, participants will get further heart tests to confi…

This study tests whether a radioactive tracer called Florbetaben F18 can reliably detect amyloid deposits in the heart using PET scans. Researchers will scan 15 people with and without cardiac amyloidosis twice to see if the results are consistent. The goal is to improve diagnosi…

This study tests a radioactive tracer called 124I-AT-01 that binds to amyloid clumps in the body. Researchers want to see if PET/CT scans can detect amyloidosis in organs of people with CAPS who developed it from anakinra injections. Thirty adults will be scanned every 6 months f…

This study tests a new PET scan that uses a special tracer to find blood clots inside the heart. It involves 20 people with cardiac amyloidosis and atrial fibrillation. The goal is to see if this scan can spot clots better than current methods, which could help prevent strokes.

This early-phase study tests a radioactive tracer called 11C-M503 to see if it can detect abnormal alpha-synuclein protein clumps in the brain, which are linked to Parkinson's disease and multiple system atrophy. About 70 adults aged 40-85 with Parkinson's, multiple system atroph…

This study is testing a non-invasive eye camera that takes pictures of the retina using different colors of light. Researchers want to see if these images can help detect signs of Alzheimer's, Parkinson's, and other neurodegenerative diseases. About 930 adults over age 30 with or…

This study tests whether artificial intelligence can help doctors interpret echocardiograms (heart ultrasounds) more accurately and quickly. Researchers at Mayo Clinic will deploy AI algorithms to flag signs of genetic cardiomyopathy, ischemic heart disease, and cardiac amyloidos…

This study tests a radioactive imaging agent called Iodine-124 evuzumitide (AT-01) in 150 people with systemic amyloidosis. The goal is to see how well it detects amyloid deposits in the heart and other organs using PET/MRI or PET/CT scans. If accurate, it could become a new tool…

This study is testing whether new blood tests can accurately diagnose the cause of memory loss or dementia, potentially replacing the need for a spinal tap. Researchers will compare blood test results with standard spinal fluid tests in 1,200 people at a memory clinic. If success…

This study aims to make it easier for family doctors to detect early signs of Alzheimer's and other dementias. Researchers will test 1,200 adults with memory or thinking problems using blood tests and brain scans. The goal is to see if these tools can accurately identify who has …

This study is testing a new blood test to help doctors diagnose Parkinson's disease and similar brain disorders more accurately and earlier. Researchers will use a technique called RT-QuIC to detect misfolded proteins in blood samples from 458 participants, including healthy peop…

This study aims to find the best cutoff values for key Alzheimer's-related proteins in spinal fluid and blood to improve diagnostic accuracy. Researchers will compare results from Alzheimer's patients, those with mild memory problems, people with other dementias, and healthy olde…

This study is testing a new blood test called the AD-seeds-detector that looks for specific protein clumps linked to Alzheimer's disease. Researchers will compare the test results from 1,500 people aged 55-75, including those with Alzheimer's, other dementias, and healthy individ…

This study is testing a new serious game called REALSoCog to see if it can detect social behavior problems in people with Alzheimer's, Lewy body dementia, or frontotemporal degeneration. Researchers will ask 120 patients to play the game on a laptop, which presents 16 social situ…

This study tests a dye called AMDX-2011P that is injected into the vein to see if it can light up amyloid deposits in the retina, which may indicate cerebral amyloid angiopathy (CAA), a condition that raises the risk of brain bleeding. About 25 adults with CAA will receive a sing…

This phase 3 study is testing an improved PET/CT imaging method using a radioactive tracer called 18F-DOPA. Researchers want to see if it can better detect small lesions and reduce image artifacts in patients with conditions like congenital hyperinsulinism, neuroblastoma, neuroen…

This study is testing whether artificial intelligence can help diagnose Parkinson's disease by analyzing facial expressions and speech patterns. Researchers will record videos of 720 participants, including people with Parkinson's and similar conditions, as well as healthy volunt…

This study is testing three new radioactive tracers that light up alpha-synuclein clumps in the brain during PET/CT scans. Researchers want to see if these scans can accurately diagnose Parkinson's disease and related disorders. 500 adults with suspected or confirmed Parkinson's …

This study at Mayo Clinic is testing whether a special MRI technique called magnetic resonance elastography (MRE) can measure stiffness in the hearts of people with cardiac amyloidosis, a condition where abnormal proteins build up and stiffen the heart. Researchers will enroll 20…

This study tests whether a generative AI tool can help doctors diagnose complications and co-existing diseases in people with rheumatoid arthritis. The AI reviews patient records and suggests possible issues like lung or heart problems. Doctors then decide if the AI's suggestions…

This study is testing a new, non-invasive eye scan to see if it can detect Parkinson's disease in its early stages. Researchers will compare the eye scan results with standard brain imaging in 200 participants, including people with early Parkinson's, related movement disorders, …

This early-phase study tests a new radioactive tracer called [18F]ACI-15916, designed to detect clumps of α-synuclein protein in the brain using PET scans. These protein deposits are linked to Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies. The trial …

This study is testing a new imaging agent called [18F]FT8 to see if it can help doctors better see and diagnose amyloidosis, a disease where abnormal proteins build up in organs like the heart. The study will involve 20 patients with amyloidosis and 5 healthy volunteers. Research…

This early-phase study tests a new radioactive tracer called 11C-HY-2-15, designed to detect abnormal alpha-synuclein protein in the brain. Researchers will scan 70 adults with multiple system atrophy, Parkinson's disease, progressive supranuclear palsy, or healthy volunteers to …

This phase 3 trial tests whether acoramidis can prevent or delay transthyretin amyloidosis (ATTR) in 587 adults who carry a faulty gene but have no symptoms yet. ATTR causes sticky plaques to build up in the heart and nerves, leading to heart failure and nerve damage. Acoramidis …

This study tests whether increasing physical activity through a motivational smartphone app can slow cognitive decline in people with isolated REM sleep behavior disorder (iRBD), an early sign of Parkinson's disease and related disorders. Researchers will enroll 130 participants …

This study tests whether the experimental drug ACP-204 can reduce psychosis symptoms like hallucinations and delusions in people with Lewy body dementia. About 180 adults aged 55 to 85 will take either ACP-204 or a placebo daily for 6 weeks. The goal is to see if the drug safely …

This study tests whether a personalized form of transcranial magnetic stimulation (TMS) can improve motor and non-motor symptoms in people with Parkinson's disease, multiple system atrophy, or progressive supranuclear palsy. Fifty participants will receive 10 days of targeted TMS…

This study tests if shining a special light on the head (transcranial photobiomodulation) can improve memory and thinking in people with mild cognitive impairment. Forty participants will receive either real or fake light sessions for 12 weeks. Researchers will measure brain acti…

This study compares giving cancer treatment at home versus in a clinic for 27 adults with various cancers in the Florida Panhandle. The goal is to see if home treatment is preferred and improves satisfaction, while also checking safety. Participants will receive their usual cance…

This study tests whether raising the heart rate with a pacemaker can improve heart failure symptoms and exercise ability in 10 people with cardiac amyloidosis who already have a pacemaker. Participants will get a personalized pacing protocol based on their height and heart functi…

This study tests a new device that uses focused ultrasound waves on the brain to see if it can safely improve movement problems in people with Parkinson's disease. About 60 adults with Parkinson's will receive either real or fake (sham) ultrasound treatments. Researchers will mea…

This study looks at whether a simple device that makes you breathe in a little extra carbon dioxide (CO2) can help raise blood pressure in people with a condition called neurogenic orthostatic hypotension (nOH). People with nOH get dizzy or even faint when they stand up because t…

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 whether adding electroconvulsive therapy (ECT) to usual care can safely reduce severe agitation in people with moderate to severe dementia, including Alzheimer's and other types. Fifty participants will receive ECT sessions with anesthesia, and researchers will m…

This study tests whether a 6-week swallowing rehabilitation program can improve swallowing function in people with multiple system atrophy (MSA) who have trouble swallowing. 24 adults with MSA and confirmed swallowing problems will receive either the full rehab program or a singl…

This study tests a safe, non-invasive brain stimulation technique called tDCS to see if it can improve movement and balance in people with a rare neurological condition called Multiple System Atrophy - Cerebellar type (MSA-C). About 30 participants will receive either real or sha…

This study looks at whether using a CPAP machine (a device that gently blows air through a mask) can safely lower high blood pressure when lying down at night and help reduce dizziness upon standing in people with autonomic failure. About 59 adults aged 40-80 with conditions like…

This pilot study tests a personalized music therapy program to prevent delirium (sudden confusion) in hospitalized people with neurodegenerative diseases like Alzheimer's or Parkinson's. Thirty participants will receive up to five live music sessions tailored to their preferences…

This study tests whether applying an ice pack or a warm gel pad to the drain site before removal can lower anxiety, improve comfort, and stabilize vital signs in patients recovering from open urological surgery. 120 adults will be randomly assigned to ice, warm gel, or standard c…

This study tests whether a non-invasive brain stimulation technique called patterned repetitive transcranial magnetic stimulation (prTMS) can reduce bradykinesia—slowness of movement—in people with Parkinson's disease. Researchers will apply the stimulation over a brain area invo…

This study looks at language difficulties in people with Lewy body disease (like Parkinson's or dementia with Lewy bodies) who also have mild cognitive impairment. Researchers will first compare sentence understanding between patients and healthy older adults. Then they will test…

This study looks at whether changing the ratio of carbidopa to levodopa in standard Parkinson's medication can reduce orthostatic hypotension—a sudden drop in blood pressure when standing that causes dizziness and fainting. Researchers will give 36 patients with Parkinson's disea…

This study tests whether a safe, non-invasive brain stimulation technique called tACS can improve cognitive abilities in people with Lewy body dementia. Forty participants will receive either real or sham stimulation over two weeks, and their thinking, memory, and daily function …

This study tests whether applying gentle heat to the abdomen overnight can safely lower high blood pressure that occurs when lying down in people with autonomic failure. About 20 adults with conditions like Parkinson's or Multiple System Atrophy who have this nighttime hypertensi…

This study explores whether combining a safe, low-level electrical brain stimulation (tDCS) with online chair yoga can help manage chronic knee pain in older adults with Alzheimer's or related dementias. Forty participants, along with their caregivers, will do 14 supervised sessi…

This study looks at whether raising the head of the bed can help lower high blood pressure that happens when people with autonomic failure lie down. About 44 adults aged 18-85 with this condition will be studied overnight. The goal is to see if gravity reducing blood return to th…

This study looks at whether using a CPAP machine at night can lower dangerously high blood pressure that happens when people with autonomic failure lie down. About 12 participants will use CPAP or a placebo pill/patch. The main goal is to see if CPAP reduces blood pressure while …

This study tests whether giving the drug daratumumab as a shot in the upper thigh works as well as the usual shot in the abdomen for people with plasma cell disorders like multiple myeloma. The goal is to find a less painful or more accessible injection spot. About 30 adults will…

This small pilot study tests whether a non-invasive brain stimulation technique called tDCS can reduce psychotic symptoms like hallucinations and agitation in people with Lewy body dementia. Thirty participants will receive either real or sham stimulation over two weeks, and rese…

This study compares two antipsychotic drugs, pimavanserin and quetiapine, to see which one better reduces hallucinations and delusions in people with Parkinson's disease or dementia with Lewy bodies. About 94 participants will be randomly assigned to one of the two medications, a…

This study tests whether a mild electrical brain stimulation technique called tDCS can improve swallowing difficulties in people with Parkinson's disease. 58 participants will receive either real or sham tDCS alongside standard swallowing therapy. Researchers will measure changes…

This study tests whether a gentle, non-invasive brain stimulation technique called tACS can improve memory and thinking in people with Alzheimer's disease, frontotemporal dementia, Lewy body dementia, and related conditions. Two hundred participants will receive either real or sh…

This study explores whether a small accelerometer worn on the thigh can objectively measure how well treatments work for neurogenic orthostatic hypotension—a condition causing dizziness and fainting upon standing. Participants wear the device for one week on placebo and one week …

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 is looking for early signs of heart amyloidosis in Black adults who carry a specific gene change (V122I TTR) that raises their risk. Researchers will use heart MRI scans and blood tests to detect protein buildup before symptoms appear. The goal is to find ways to diagn…

This study tests a new PET scan technique to better track heart damage in people with a condition called ATTR-CM, a progressive heart muscle disease. Researchers will scan 140 participants to see if the imaging can show how well treatments are working. The goal is to improve moni…

This pilot trial is testing whether amyloidosis patients can use the MyFitnessPal smartphone app to track their daily food and supplement intake for 8 weeks. The goal is to see if this low-cost method is feasible for studying malnutrition and weight loss in this condition. Forty …

This study will follow about 1,850 people with ATTR amyloidosis, a rare disease that affects the heart and nerves. Researchers will collect information on patients' health, treatments, and outcomes over time to better understand the disease and how current therapies work in every…

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 follows up to 10 pregnant or breastfeeding individuals with transthyretin amyloidosis who have taken eplontersen, along with their infants. Researchers will track pregnancy complications, birth defects, and infant health during the first year of life. The goal is to de…

This study is testing how to safely perform skin allergy tests in people receiving biotherapies for cancers and blood disorders. Researchers will find the highest concentration of each biotherapy that does not cause a skin reaction in 9 out of 10 patients. The goal is to establis…

This study follows 100 obese patients starting dialysis to understand why they are less likely to receive a kidney transplant. Researchers will track health changes over two years and compare them to non-obese patients. The goal is to identify barriers and improve access to trans…

This study follows 214 people in China with multiple system atrophy, a rare and fast-worsening brain disease that affects movement and automatic body functions. Researchers will track symptoms over time to learn how the disease progresses and what factors influence it. The goal i…

This study follows 800 adults with synucleinopathies—diseases like Parkinson's, Lewy body dementia, and multiple system atrophy—to learn how they develop and change over time. Researchers will track symptoms, biological markers, and early signs like dizziness on standing or dream…

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 is testing a new radioactive tracer called [18F]MODAG-009 in 6 healthy adults. The goal is to see how the tracer moves through the body and how much radiation exposure it causes. Participants get one injection and then a full-body PET scan. This is an early-phase study…

This observational study is recruiting 600 participants to improve detection and understanding of dementia with Lewy bodies (DLB). Researchers will collect clinical data, brain scans, and spinal fluid samples from people with early symptoms like hallucinations or movement problem…

This early-stage trial is studying how the drug dobutamine affects the heart in 15 people with a rare condition called wild-type transthyretin amyloid cardiomyopathy (ATTRwt). Participants will receive increasing doses of dobutamine while doctors measure heart function using ultr…

This study is examining heart valve tissue from 100 people undergoing surgery for aortic stenosis to see how often ATTR amyloid deposits are present. Researchers will use mass spectrometry to identify the type of amyloid. The goal is to better understand the link between amyloido…

This study monitors pregnancy outcomes in women who took patisiran-LNP for hereditary ATTR amyloidosis. Researchers will track birth defects, pregnancy complications, and infant health. The goal is to better understand the drug's safety during pregnancy, not to test a new treatme…

This observational study will follow 1,500 people with ATTR amyloidosis, a rare disease that damages the heart and nerves, for many years. Researchers will track how the disease progresses, how patients are treated in real-world settings, and the safety of two approved drugs, pat…

This study looks at why people with Alzheimer's disease may move more slowly. Researchers will use brain MRI scans and simple reaction time tests in 155 participants with mild cognitive impairment or early dementia. The goal is to link brain lesions to attention-related motor slo…

This study follows 200 people with Multiple System Atrophy (MSA) to see if receiving care from a team of specialists every four months improves their quality of life and eases the burden on their caregivers. Participants and their doctors will complete questionnaires about daily …

This observational study will analyze medical records from over 52,000 people with ATTR amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers want to learn about patients' symptoms, treatments, and outcomes in everyday medical practice. No new treatm…

This study aims to find genes that increase the risk of developing Alzheimer's disease. Researchers are recruiting 10,000 people, including those with Alzheimer's and their family members, from all ethnic backgrounds. By analyzing DNA, they hope to discover new genetic clues that…

This study looks at whether new, quick tests that measure how fast people can move can help doctors diagnose brain diseases like Alzheimer's and predict recovery after a stroke. Researchers will test 129 adults aged 40-85 with stroke or memory problems. The goal is to see if thes…

This study is a worldwide registry that follows 5,000 adults with breast cancer, blood cancers, or those on immune checkpoint inhibitors. Researchers collect medical records, lab results, and imaging to identify who is at risk for heart problems during or after cancer treatment. …

This study looks at a specific gene (IGLV1-44) to understand why it causes two different diseases: AL amyloidosis and POEMS syndrome. Researchers will analyze blood and bone marrow samples from 100 adults with these conditions or multiple myeloma. The goal is to find genetic patt…

This study is testing whether a non-invasive electrical stimulation technique can improve working memory, attention, and thinking skills in healthy older adults and those with mild cognitive impairment. The stimulation targets deep brain structures without surgery, using special …

This study will follow about 2000 adults with a heart condition called ATTR-CM, where abnormal proteins build up and weaken the heart. Researchers want to see how patients do on a drug called vutrisiran in everyday medical care, not just in a controlled trial. They will measure s…

This study follows 150 people with early-stage Parkinson's disease to see if the condition starts in the brain or the body first. Researchers will track symptoms like walking problems and thinking changes over 5 years using tests, scans, and questionnaires. The goal is to better …

This study looks back at the last 10 years in Denmark to understand how many people use breathing machines at home through a tube in their windpipe. Researchers will track which diseases lead to this need and how many patients survive one year after starting. The goal is to spot …

This study looks at how often cardiac amyloidosis—a condition where abnormal proteins build up in the heart—occurs in people aged 80 and older who have been hospitalized for heart failure. Researchers will use bone scans to detect the condition in 637 participants. The goal is to…

This study observes 280 people with Parkinson's disease or multiple system atrophy to understand how pain and automatic body functions (like blood pressure and digestion) affect their lives. Participants will undergo standard rehabilitation and be assessed with questionnaires. Th…

This study is building a large registry of 5,000 people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect real-world data on how the disease progresses and how current treatments affect it. No new drug is being tested; the go…

This study is creating a large European registry of 400 newly diagnosed AL amyloidosis patients. Researchers will collect medical data and blood samples to study the disease using advanced technology. The goal is to better understand how AL amyloidosis develops and progresses, wh…

This study is creating a network of hospitals in Italy to find and treat AL amyloidosis earlier. Researchers will screen people with certain blood conditions (MGUS or smoldering myeloma) using special tests. The goal is to speed up diagnosis, connect patients to the right care, a…

This study follows 200 adults with ATTRwt amyloidosis who are taking tafamidis to see how often they develop heart rhythm problems that need a pacemaker or defibrillator. Researchers will also track changes in diuretic use. Participants are checked every six months as part of rou…

This study follows 650 people with early Parkinson's disease or a sleep condition called idiopathic REM sleep behavior disorder (iRBD). Researchers use brain scans, heart tests, and fluid samples to track changes over time. The goal is to better understand how these conditions de…

This study follows nearly 3,000 people, including healthy adults and those with memory or movement problems, to learn how brain diseases like Alzheimer's and Parkinson's develop. Researchers use advanced brain scans and tests to track changes over time. The goal is to improve ear…

This study uses a new ultra-high-resolution PET scanner to take detailed pictures of the brain and spinal cord in 300 healthy volunteers and people with conditions like Alzheimer's, Parkinson's, ALS, and psychotic disorders. The goal is to understand how small brain regions chang…

This study looks at 57 people aged 20 to 70 who carry the Val50Met gene variant linked to hereditary ATTR amyloidosis, a condition that can damage nerves and the heart. Researchers want to describe the different symptoms people experience, such as heart problems, nerve issues, or…

This study is collecting medical data and brain scans from 500 people with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain and memory problems in older adults. Researchers want to better understand how the disease progresses and find markers that …

This small study tests a smartphone app that creates a 'digital twin' of the patient to help manage health after a stroke, moyamoya disease, or cerebral amyloid angiopathy. Twenty adults will either use the app plus standard care, or standard care alone. The goal is to see if pat…

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 large database of 1,000 people with ATTR amyloidosis, a rare disease that causes abnormal protein buildup in organs. Researchers will collect health information over time to better understand how the disease progresses and to develop tools that help docto…

This observational study will follow 20 adults with a confirmed genetic mutation for hereditary transthyretin amyloidosis (ATTR), a rare and serious condition that causes abnormal protein buildup in organs. Researchers will conduct thorough medical exams, heart tests, and neurolo…

This study tests a communication program called COACH-Cog for older adults with both dementia and cancer. It includes a training video for doctors and a coaching session for patients and their care partners. The goal is to see if this helps everyone work together better when maki…

This study is creating a database of up to 5,000 people in Ohio with plasma cell disorders like multiple myeloma and amyloidosis. Researchers will track treatments, outcomes, and quality of life, while also offering patients access to expert consultations and information about su…

This study uses a special PET scan with a radioactive tracer called [18F]F-DOPA to measure dopamine production in the brains of people with autonomic failure (problems with automatic body functions like blood pressure). The goal is to see if changes in dopamine levels can predict…

This study is observing 1,000 critically ill adults in Latin American ICUs who need dialysis for severe acute kidney injury. Researchers will track treatments, outcomes, and country differences to understand what works best. The goal is to gather data that can improve care and gu…

This study is gathering information from 700 people with Parkinson's disease or similar conditions who experience freezing of gait—a sudden inability to move their feet while walking. Researchers will track symptoms, walking patterns, and falls over three years using questionnair…

This study looks at whether a rapid drop in a blood marker called dFLC can predict a complete response in people with AL amyloidosis treated with daratumumab-based therapy. Researchers will follow 50 patients to see if early changes in dFLC levels match up with later treatment su…

This study looks at why people with Lewy body dementia have sudden changes in attention and thinking, called cognitive fluctuations. Researchers will use skin biopsies, brain scans, and EEG monitoring in 120 participants to link these symptoms to damage in a key brain chemical sy…

This study is following 700 people with Parkinson's disease or multiple system atrophy in Shanghai. Researchers will collect medical history, brain scans, blood samples, and other data every six months. The goal is to find early signs of these diseases and better predict how they…

This study investigates how a sugar modification called N-glycosylation affects AL amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will analyze blood and bone marrow samples from 100 adults with related conditions to better understand the disea…

This study tests whether a non-invasive brain stimulation technique called temporal interference stimulation (TIS) can temporarily improve language processing in people with mild cognitive impairment due to Parkinson's disease, Lewy body disease, or amnestic MCI. Seventy particip…

This study is creating a biobank and data registry for people with amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers will collect blood, urine, tissue samples, and medical information from 505 participants over time. The goal is to store these ma…

This study is for men over 65 who have chest pain but no blocked heart arteries. It checks if a hidden condition called cardiac amyloidosis might be the cause. Participants will have blood tests, heart scans, and other exams. The goal is to find out how common this hidden disease…

This study looks at the gut microbiome (the bacteria living in the digestive system) of people with POEMS syndrome, multiple myeloma, MGUS, AL amyloidosis, and healthy household members. Participants provide stool samples at home using a collection kit. The goal is to understand …

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 uses special brain scans to measure damage to nerve connections in people with Multiple System Atrophy (MSA) and Progressive Supranuclear Palsy (PSP). Researchers will scan 36 adults aged 45-80 to see how the disease changes over time. The goal is to better understand …

This study looks at how the brain, memory, thinking, and movement change over time in people with Lewy body disease, Alzheimer disease, and healthy volunteers. Researchers will use brain scans and brain-wave tests to learn more about these disorders. The goal is to better underst…

This study uses advanced heart MRI scans to look at blood flow patterns in people with different types of hypertrophic cardiomyopathy (thick heart muscle), including rare forms like Anderson-Fabry disease and cardiac amyloidosis. Researchers will also study first-degree relatives…

This study uses non-invasive eye scans (OCT and OCTA) to look for changes in the retina that might be linked to brain diseases like Alzheimer's, Parkinson's, and multiple sclerosis. Researchers aim to find early markers that could help diagnose or track these conditions. The stud…

This study is testing whether artificial intelligence (AI) can analyze brain scans to help diagnose Parkinson's disease and related conditions like multiple system atrophy. Researchers will also see if the scans can predict how fast the disease will progress. The study involves 9…

This study collects information and samples from 1,000 people with or at risk for brain diseases like Alzheimer's, ALS, and frontotemporal degeneration. Researchers will track changes in thinking, language, and brain scans over time. The goal is to build a resource for future stu…

This study follows 1,573 adults aged 50 to 90 across Canada, including people with different types of dementia and those with healthy memory. Researchers will use memory tests, brain scans, and blood samples to track how thinking and memory change over time. The goal is to better…

This study is creating a registry of 500 people with amyloidosis at Hospital Italiano de Buenos Aires. Researchers will track risk factors, diagnosis, treatment, and survival over time to better understand the disease. No new treatments are being tested—this is purely an observat…

This study collects DNA from up to 1,000 adults with progressive supranuclear palsy (PSP), corticobasal syndrome (CBS), multiple system atrophy (MSA), or related conditions, plus their family members. Researchers will sequence participants' whole genomes to find genetic variants …

This study is creating a large database to collect information on people with rare diseases like amyloidosis, sarcoidosis, and Gaucher disease. Researchers will track patients' health over time, including their symptoms, treatments, and outcomes. The goal is to improve diagnosis …

This study looks at how flexible brain activity is in people with Lewy body disease, a condition that causes thinking and behavior problems and is often hard to diagnose. Researchers will analyze past EEG recordings from 40 adults to see if certain brain patterns are linked to th…

This study is tracking 50 adults with a heart condition called ATTR-CM to see if medications like tafamidis or acoramidis lower harmful amyloid proteins in the blood over time. Researchers will take blood samples at several visits to measure these proteins. The goal is to find ou…

This study is a large registry that will collect health information from up to 60,000 people with both heart and kidney disease. The goal is to better understand how these conditions interact and to find ways to predict and improve patient outcomes. No new treatments or procedure…

This study is testing whether new brain imaging techniques can better diagnose diseases like Parkinson's, multiple system atrophy, and frontotemporal dementia. Researchers will use MRI scans and neurological exams in about 94 adults aged 40-85. The goal is to improve how doctors …

This study looks at whether tiny particles released by cells into the blood, called extracellular vesicles, can help detect a type of heart disease called ATTR amyloidosis earlier. The disease often goes unnoticed until serious damage occurs. Researchers will compare these partic…

This early-phase study tests a new radioactive tracer called [11C]SY08 to see if it can detect clumps of alpha-synuclein protein in the brain using PET scans. Researchers will scan 40 volunteers, including healthy people and those with Parkinson's disease, multiple system atrophy…

This study tests a new, non-invasive heart scan called magnetocardiography (MCG) to see if it can detect and track amyloidosis, a rare disease where abnormal proteins build up in organs. Researchers at Mayo Clinic will compare MCG with standard ECG in 500 adults. This is an obser…

This study tests whether spending less time in bed for four weeks can deepen sleep and boost memory in adults aged 65-85 who have poor sleep and are at risk for Alzheimer's. Researchers will measure brain activity, memory, and blood markers. The goal is to see if a simple behavio…

This study is observing 3000 adults with various types of heart failure to learn how the disease develops and progresses. Researchers will collect medical records, blood samples, and imaging data to identify distinct patient groups. The goal is to match the right treatments to th…

This study looks at why about one-third of people with a heart condition called cardiac amyloidosis continue to get worse even with medication. Researchers will use special heart scans (ultrasound, MRI, and PET) at the start and after one year to see what is happening inside the …

This study creates a registry of 5,000 adults aged 50 and older with blood cancers like multiple myeloma and lymphoma. Researchers will track frailty, muscle loss, and other age-related health issues to better understand this group's needs. The goal is to gather information that …

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 looks at how often amyloidosis, a rare disease where abnormal proteins build up in organs, occurs in people scheduled for trauma surgery. Researchers will use a checklist of warning signs (red flags) like age over 65, heart issues, or family history. About 246 adults w…

This study looks at whether a campaign called Choosing Wisely can safely reduce unnecessary tests and treatments in intensive care units (ICUs). About 10,000 adult patients in Swedish ICUs will take part. The goal is to see if this approach lowers costs and improves care without …

This study is creating a large collection of blood, urine, and tissue samples from up to 10,000 people with amyloid diseases (like multiple myeloma). The goal is to store these samples and health data for future research to better understand these conditions. Participants do not …

This study creates a registry for adults with plasma cell disorders, such as multiple myeloma and amyloidosis. Researchers will collect health information and optional blood samples to better understand these conditions. The goal is to gather data that could support future resear…

This study is looking for 500 people with early dementia or mild cognitive impairment to help develop a way to predict how their disease will progress. Researchers will use blood tests and other models to better understand each person's outlook. The goal is to give patients and d…

This study tests whether a special ultrasound treatment, combined with a contrast agent, can improve blood flow and heart function in people with cardiac amyloidosis. Researchers will measure changes using PET scans and echocardiograms. The trial includes 70 participants with and…

This study is for people with light chain amyloidosis, a rare disease where abnormal proteins build up in the heart and other organs. Researchers will use special PET and MRI scans to take detailed pictures of these protein deposits in the heart. The goal is to better understand …

This study is creating a large collection of blood samples, tissue, and health information from 2,500 people with plasma cell disorders like multiple myeloma. Researchers will use this repository to better understand how the disease develops and changes over time. No new treatmen…

This observational study tests a new device (eMyosound LYRA) that uses sound waves to measure stiffness in the heart and liver. Researchers will compare 150 people—some with heart failure, some with a rare heart condition called ATTR-CM, and some healthy volunteers. The goal is t…

This study looks at how midodrine and droxidopa, two drugs used for low blood pressure upon standing, affect the veins in the abdomen of people with autonomic failure. About 34 adults aged 40-80 with conditions like multiple system atrophy or Parkinson's disease will participate.…

This study is building a large database of health information from 250 people with Parkinson's disease and related conditions. Researchers will collect data on symptoms, treatments, genetics, and brain scans over time. The goal is to better understand how the disease progresses a…

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 is testing whether special eye scans can detect changes linked to memory disorders like Alzheimer's and Parkinson's. Researchers will compare eye images from 450 people with and without memory complaints. The goal is to see if these non-invasive scans can help diagnose…

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 looks at whether people with Alzheimer's disease also have amyloid deposits in their hearts, which can cause heart problems. Researchers will use special PET scans—normally used to see amyloid in the brain—to also scan the heart. The goal is to better understand if the…

This study is testing whether a special type of PET/MRI scan using a radioactive tracer called florbetaben can help predict major heart problems in people with cardiac amyloidosis. The researchers will follow 25 patients to see if certain scan results are linked to serious events…

This study looks at people with autoinflammatory diseases or AA amyloidosis who have genetic changes that are hard to interpret. Researchers want to understand if these changes actually cause the disease. The goal is to improve diagnosis and knowledge, not to test a new treatment…

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…