AMYLOIDOSIS

This phase 2 trial is testing whether a powerful four-drug combination can make multiple myeloma or amyloidosis disappear at a genetic level, allowing patients to safely postpone or avoid a stem cell transplant. About 40 newly diagnosed adults will receive the drug therapy first;…

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 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 program offers early access to venetoclax, a targeted cancer pill, for people with certain blood cancers like CLL, AML, and multiple myeloma. It is for patients who have no other suitable treatment options and cannot join ongoing clinical trials. A doctor must decide if the …

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 1 study tests a radioactive tracer called 124I-AT-01 during PET/CT scans to see if it can detect amyloid deposits in organs of people with CAPS who developed amyloidosis from anakinra injections. Thirty adults will be scanned every 6 months for 2 years. The goal is to …

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 is testing a special imaging agent called Iodine-124 evuzumitide to see if it can more accurately diagnose cardiac amyloidosis, a condition where abnormal proteins build up in the heart and other organs. About 150 adults with known or suspected amyloidosis will receive…

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 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 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 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 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 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 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 looks at whether a generative AI tool can help doctors find complications of rheumatoid arthritis (RA), such as lung disease or heart problems. Researchers will review past patient records and also observe how doctors use AI suggestions in real time. About 100 RA patie…

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 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. About 20 adults with this condition will un…

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…