AMYLOIDOSIS

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…