Amyloidosis

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 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 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 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 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 the drug acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart, weakening it. Over 36 months, researchers measure whether acoramidis improves heart function and structure using cardiac …

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

This study tests 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 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 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 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 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 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 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 trial tests whether PET/CT scans using special radioactive tracers can detect amyloid protein buildup in various diseases, including cardiac amyloidosis. Researchers will give each participant a single injection of one of several tracers, then perform imaging to measure how …

This study tests whether PET/CT scans using two radioactive tracers, 18F-92/AV45 and 11C-PIB, can detect beta amyloid buildup in the brain. Researchers will enroll 500 adults with suspected or known neurodegenerative diseases linked to amyloid. Each participant receives one injec…

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 study tests whether a cardiac imaging technique called a PYP scan can detect transthyretin amyloidosis in people aged 65 and older who have heart failure with preserved ejection fraction, also known as diastolic heart failure. The researchers will scan 515 participants to se…

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 Phase 1 trial is testing a new radioactive tracer called 99mTc-p5+14 to see how it spreads through the body and how much radiation it delivers. The study includes healthy volunteers and people with AL or ATTR systemic amyloidosis, a condition where abnormal protein deposits …

This trial tests whether an artificial intelligence algorithm, called EchoNet-LVH, can help identify patients who may have cardiac amyloidosis — a rare but serious condition where abnormal protein builds up in the heart. The AI analyzes routine echocardiograms for subtle signs th…

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 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 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 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 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 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 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 a supervised cardiac rehabilitation program is feasible and beneficial for people with cardiac amyloidosis, a condition where abnormal protein builds up in the heart. Participants attend three one-hour exercise sessions per week for 12 weeks. The study me…

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

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

Researchers at Peking University First Hospital are building a prospective cohort of 2,000 people with plasma cell disorders, including multiple myeloma, amyloidosis, and related conditions. Participants give blood, bone marrow, and urine samples before and after treatment, and t…

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 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 whether advanced imaging can detect a hidden heart condition called cardiac amyloidosis in people with severe aortic valve stenosis. The condition can complicate treatment and recovery. Researchers will use echocardiography, CT, and SPECT/CT scans to check for…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…