Amyloidosis

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…

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 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…