AMYLOIDOSIS

This program provides early access to venetoclax, an investigational drug, for people with certain blood cancers like chronic lymphocytic leukemia (CLL), acute myeloid leukemia (AML), and multiple myeloma. It is for patients who have no other suitable treatment options and cannot…

This study 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. The treatment aims to lower a harmful protein (TTR) to slow or stop disease prog…

This study tests a new treatment called AZD0120 (GC012F) for people with AL amyloidosis that has come back or not responded to other therapies. The treatment uses specially engineered immune cells (CAR T-cells) to target and destroy harmful cells. The study aims to see if it is s…

This study tests whether adding the experimental drug SCTC21C to standard chemotherapy (VCd) works better than standard chemotherapy alone for people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 90 adults with measurable di…

This study is for people newly diagnosed with multiple myeloma or amyloidosis. It tests whether a very sensitive genetic test can identify patients who respond so well to initial drug therapy that they can safely postpone a stem cell transplant. The goal is to see if avoiding or …

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 special radioactive tracer used during PET/CT scans to find protein clumps (amyloidosis) in organs of people with CAPS who developed this condition from the drug anakinra. Thirty adults will get scans every 6 months for 2 years to see if the tracer can track ch…

This study is testing a new radioactive tracer called [18F]FT8 for PET/CT scans to see if it can safely and clearly show amyloid deposits in organs, especially the heart. Researchers will compare the scans to standard tests like echocardiograms and MRIs. The study involves 20 peo…

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 study tests an experimental drug called acoramidis in 587 adults who carry a gene that puts them at high risk for transthyretin amyloidosis (ATTR), a condition where sticky plaques damage the heart and nerves. The goal is to see if taking acoramidis early can prevent or dela…

This study compares giving cancer treatment at home versus in a clinic for 27 adults with various cancers. The goal is to see if home treatment is preferred and just as safe. Participants will receive their usual cancer drugs at home from a specialized team, and researchers will …

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 building a registry of 500 people with amyloidosis to better understand the disease. Researchers will collect information on risk factors, diagnosis, treatment, and survival over time. The goal is to describe how amyloidosis affects patients in Buenos Aires and impr…

This study looks at how often amyloidosis, a rare disease where abnormal proteins build up in organs, occurs in people having planned trauma surgery. Researchers will check 246 patients for certain warning signs called red flags, like age over 65 or heart problems. The goal is to…

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 using surveys and physical tests. The goal is to better understand this group's …

This study looks at whether generative AI can help doctors find complications of rheumatoid arthritis (RA), such as lung disease or heart problems. Researchers will use AI to analyze patient records and suggest possible complications. Doctors will then decide if the AI's suggesti…

This study is building a registry of up to 2,000 adults with plasma cell disorders, such as multiple myeloma and amyloidosis. Participants complete health assessments and may give a blood sample to study aging markers. The goal is to gather information to support future research,…

This study follows 200 adults with ATTR amyloidosis (a heart condition) who are taking the drug tafamidis. Researchers will check every six months for heart rhythm issues that might need a pacemaker or defibrillator. The goal is to better understand and monitor these risks in rou…

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 is creating a detailed registry of people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will collect information from medical records and patient reports to learn how these diseases progress, how they are treated, and what outcomes pati…

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…