Amyloidosis

This early-phase trial tested a combination of three drugs—daratumumab, ixazomib, and dexamethasone—in 21 people with AL amyloidosis, a rare disease where abnormal protein builds up in organs. The main goal was to find the safest dose and check for side effects. Researchers also …

This study tested a drug called ALXN2060 (acoramidis) in 25 Japanese patients with a rare heart condition called ATTR-CM, which causes heart failure. The drug is taken as a tablet twice daily. Researchers measured how far patients could walk in 6 minutes and tracked heart-related…

This study tested the drug patisiran in 10 adults with a rare condition called ATTR amyloidosis that causes nerve damage. Participants received an IV infusion every 21 days for 24 months. Researchers measured changes in nerve function, autonomic symptoms, and quality of life to s…

This study looked at how safe and effective Vyndaqel is for people with a rare heart condition called transthyretin amyloid cardiomyopathy. Over 1,200 patients in Japan were followed while taking the drug in regular medical practice. The main goal was to track side effects and su…

This study looked at medical records of 50 people with a rare heart condition called ATTR-CM to see if high-dose tafamidis (Vyndamax) slows nerve damage. Researchers compared how fast nerve problems got worse before and after starting the drug. The goal was to see if the medicati…

This phase 3 trial tested the drug patisiran in 360 people with ATTR amyloidosis with cardiomyopathy, a condition where abnormal protein builds up in the heart, making it stiff and weak. Participants received either patisiran or a placebo intravenously. The study measured how far…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This early-stage study tested a new drug called ALN-TTRSC04 in 96 healthy people to see if it is safe and how the body processes it. The drug aims to lower a harmful protein that causes transthyretin amyloidosis, a serious disease affecting nerves and the heart. Participants rece…

This study tested an experimental drug called NNC6019-0001 in 105 people with transthyretin amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart, causing heart failure. Participants received either the drug or a placebo by infusion every 4 weeks for a…

This study tested an automated insulin delivery system (a smart pump) in 15 adults with type 1 or type 2 diabetes who also had advanced kidney disease or were on dialysis. Participants used the system for 8 weeks, then switched to their usual care for another 8 weeks. The goal wa…

This early-stage trial tests NTLA-2001, a CRISPR-based gene editing therapy, in 72 adults with hereditary ATTR amyloidosis—a condition where abnormal protein builds up in nerves or the heart. The treatment aims to turn off the faulty gene to reduce protein levels. The study focus…

This study tested a new drug called vutrisiran in 164 people with a rare inherited disease that damages nerves and organs. Participants received either vutrisiran as a shot every 3 months or the standard treatment patisiran as an IV infusion every 3 weeks. The goal was to see if …

This study tested a drug called MCI-196 in 245 adults on dialysis who have high phosphate levels, a common problem in advanced kidney disease. After a 4-week washout and 12-week dose adjustment, patients were randomly assigned to continue MCI-196 or switch to a placebo for 4 week…

This study tested the long-term safety of a drug called MCI-196 (Colestilan) in 116 adults with stage 5 chronic kidney disease who are on dialysis and have high phosphorus levels. Participants took flexible doses of the drug for 52 weeks. The main goal was to see how many side ef…

This Phase 3 trial tested MCI-196, a drug that binds phosphorus and lowers cholesterol, in 642 adults on dialysis with chronic kidney disease. Participants received either MCI-196 or a placebo for 12 weeks. The goal was to see if the drug could safely reduce both serum phosphorus…

This study tested the long-term safety of a drug called MCI-196 in 632 adults with chronic kidney disease on dialysis who have high phosphorus levels. Participants took the drug for up to 52 weeks to see how it affected their phosphorus and cholesterol. The goal was to help contr…

This study tested whether adding daratumumab to standard chemotherapy (CyBorD) helps people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. About 416 adults took part. The goal was to see if the combination leads to better blood res…

This study looked at how well the drug tafamidis 61mg works for Korean patients with a rare heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). Researchers tracked 13 patients who were already prescribed the drug in real-world clinics. They measured changes in …

This study looked at the safety of a drug called Tafamidis (brand name VyndaMx) in 6 people in India with a rare heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). In ATTR-CM, faulty proteins build up in the heart and make it harder to pump blood. Researchers …

This Phase 3 trial tested a new drug called AP301 in 474 Chinese adults on dialysis who had high phosphorus levels. Participants took AP301, a low dose of AP301, or the standard drug sevelamer carbonate with meals. The main goal was to see how well AP301 lowers phosphorus compare…

This phase II trial tested the drug isatuximab in 43 patients with AL amyloidosis that had come back or stopped responding to treatment. Isatuximab is a monoclonal antibody that may help stop abnormal protein buildup. The study measured how well the drug reduced disease markers a…

This phase 2 study tested a drug called belantamab mafodotin in 35 people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Participants had already tried other treatments without success. The goal was to see if the drug could reduce or eliminate tho…

This study tested a new genetic screening approach to find cases of a rare inherited nerve disease called TTR-FAP in 130 patients with chronic neuropathy of unknown cause. The goal was to see how many of these patients actually have TTR-FAP, which is often missed due to varied sy…

This completed Phase 3 study tested whether a radioactive tracer called [18F]Florbetaben, used in PET scans, can accurately diagnose cardiac AL amyloidosis—a condition where abnormal proteins build up in the heart. 244 adults with suspected cardiac amyloidosis received a single P…

This study looked at 15 patients with a rare heart condition called wild-type transthyretin amyloid cardiomyopathy (ATTRwt) who also had signs of aortic stenosis. Researchers used dobutamine stress echocardiography—a test that uses a drug to make the heart pump harder while imagi…

This study tested a new imaging drug called I-124 evuzamitide to see if it can accurately diagnose cardiac amyloidosis, a condition where abnormal proteins build up in the heart. 204 adults with suspected cardiac amyloidosis received a single dose followed by a PET/CT scan. The g…

This study tested whether a special type of heart MRI, called T1 mapping, can accurately diagnose cardiac amyloidosis—a condition where abnormal proteins build up in the heart muscle. Researchers studied 112 older adults with heart failure and thickened heart walls. They compared…

This study tested whether a special PET/MR scan using a radioactive tracer called F-18 florbetapir can detect amyloid protein buildup in the nerves of people with amyloidosis. Eight adults with confirmed nerve amyloidosis or other nerve diseases took part. The goal was to see if …

This study compared different ways to detect early nerve damage in people with hereditary TTR amyloidosis, a condition that can cause progressive nerve problems. Researchers used blood tests, skin imaging, and standard nerve exams in 47 participants. The goal was to see which too…

This study tested whether an artificial intelligence (AI) tool could help doctors detect cardiac amyloidosis, a rare but serious heart condition, earlier using standard ECG tests. Over 20,000 patients were involved, with some doctors using the AI-enhanced ECG and others continuin…

This study tested a computer model that uses a patient's age, sex, medical history, and heart tests (ECG and echocardiogram) to estimate their risk of having cardiac amyloidosis, a serious but often undiagnosed heart condition. Researchers invited 50 high-risk patients for furthe…

This pilot study tested whether a radioactive tracer called F-18 florbetapir (Amyvid) can detect amyloid protein buildup in the heart using a PET scan. Researchers enrolled 23 people with known cardiac amyloidosis and a control group without the condition. The goal was to see if …

This study screened 646 elderly Black and Hispanic patients with heart failure to find out how many have cardiac amyloidosis, a condition where abnormal proteins build up in the heart. Researchers used a special heart scan and blood tests to detect the disease. The goal was to le…

This phase 2 trial tested a radioactive tracer called 124I-AT-01 to see how well it can measure amyloid buildup in organs like the heart, kidneys, liver, and spleen using PET/CT scans. 33 adults with systemic amyloidosis received the tracer twice, six weeks apart. The goal was to…

This trial investigates whether applying a heated pack to the arm for 5 or 10 minutes before inserting a dialysis needle can reduce pain in people with an arteriovenous fistula. About 51 adults on hemodialysis will be randomly assigned to receive heat for 5 minutes, 10 minutes, o…

This study looked at 157 women with breast cancer who had a mastectomy. It compared two ways to drain fluid after surgery: active drainage (using suction) and passive drainage (gravity). The goal was to see which method led to less fluid buildup (seroma), shorter drain time, and …

This study tested whether adding acupuncture to standard care can slow down memory and thinking problems in people with mild Alzheimer's disease. Researchers compared real acupuncture to a fake (sham) procedure in 160 participants to see if the effects were real or just a placebo…

This pilot study tested whether applying methylene blue (a dye) to skin lesions and then shining red light on them could improve skin roughness in people with lichen amyloidosis, a condition causing itchy, bumpy patches. Fifteen Asian adults with the condition received the treatm…

This pilot study tested a 308-nm excimer laser on 15 Asian adults with lichen amyloidosis, a condition that causes itchy, rough patches on the skin. The goal was to see if the laser could improve skin roughness. The study is small and early, so results are not conclusive.

This study tested the drug dapagliflozin (a diabetes medicine) in 50 adults with heart failure due to amyloid cardiomyopathy, a rare condition where abnormal proteins stiffen the heart. Researchers measured changes in quality of life, walking distance, and heart stress markers ov…

This study tested whether lowering the temperature of the dialysis fluid from 36.5°C to 36°C could reduce the tiredness patients often feel after dialysis. Fifty-seven adults on maintenance hemodialysis received three sessions of 'cold dialysis' over one week. Researchers measure…

This study tested whether a program called Strategy Training can help older adults with mild cognitive impairment (MCI) manage everyday tasks like shopping, paying bills, and taking medication. The training, delivered by occupational therapists, teaches goal-setting, scheduling, …

This completed study followed 945 people, mostly children and young adults with severe eczema or related allergic conditions, along with their relatives. Researchers observed how these diseases progress over up to a year, using tests like allergy skin pricks, blood draws, and lun…

This study uses advanced heart imaging to understand how blood flows inside the heart in people with cardiac amyloidosis, a condition where abnormal proteins build up in heart tissue. Researchers will compare these images with those from healthy volunteers to find unique patterns…

This study looks at whether a specific gene variant (ApoE4) is more common in people with certain brain MRI patterns that suggest cerebral amyloid angiopathy (CAA), a condition where proteins build up in brain blood vessels. Researchers will analyze genetic samples from 100 patie…

This completed study in Russia checked how many people with carpal tunnel syndrome also have a rare inherited nerve condition called hereditary transthyretin amyloidosis. Researchers reviewed medical records of 721 patients and will test some for the genetic mutation. The goal is…

This study tested whether a special MRI scan of the wrist can detect amyloid deposits in people with systemic amyloidosis, a condition where abnormal proteins build up in organs. Researchers enrolled 128 adults, including patients with cardiac amyloidosis and healthy volunteers. …

This study checked how often nerve damage (neuropathy) occurs in people with a rare heart condition called wild-type TTR cardiac amyloidosis. Researchers examined 65 patients using interviews, physical exams, and nerve tests (electromyograms). The goal was to find out if nerve pr…

This study looked at MRI scans from 72 people with two common types of small vessel disease in the brain: hypertensive arteriopathy and cerebral amyloid angiopathy. Researchers focused on tiny fluid-filled spaces in a part of the brain called the dentate nucleus. The goal was to …

This study looked at how often cardiac amyloidosis occurs in older Tunisian patients with a type of heart failure called HFpEF. Researchers used heart scans and imaging to find cases and identify early warning signs. The goal is to make diagnosis easier and faster for this seriou…

This completed study involved 250 adults with AL amyloidosis, a rare disease where abnormal proteins damage organs. Researchers analyzed patient samples and data to find biological markers that predict how well someone will respond to initial treatments. The goal is to help docto…

This study looked at whether advanced MRI scans and a nerve test called MUNIX can detect the earliest signs of nerve damage in people who carry a gene mutation for familial amyloid neuropathy. Researchers studied 60 adults with the TTR mutation, comparing those with and without s…

This study looked at whether being physically active over a lifetime is linked to developing a rare heart disease called wild-type transthyretin amyloid cardiomyopathy. Researchers compared the exercise histories of 180 people over age 60, including those with the disease, those …

This study tested a communication tool called 'Best Case/Worst Case' for older adults with advanced kidney disease. Doctors were trained to discuss best, worst, and most likely outcomes for treatment options. The goal was to see if this approach helps patients receive more pallia…

This study looked at 30 people newly diagnosed with cardiac AL amyloidosis, a rare disease where abnormal proteins damage the heart. Researchers used advanced MRI, echocardiogram, and PET scans to see if these images could predict survival and show how well chemotherapy works. Th…

This study looked at 70 people who had a brain hemorrhage (bleeding stroke) to see if a special PET scan could tell whether the bleeding was caused by amyloid buildup in blood vessels or by high blood pressure. Participants received an injection of a radioactive tracer called Flo…

This study tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …

This completed study looked at 200 people with cerebral amyloid angiopathy (CAA), a condition where protein builds up in brain blood vessels, raising the risk of bleeding and cognitive decline. Researchers collected clinical data, brain scans, and samples of spinal fluid and bloo…

This study looked at 60 adults on long-term dialysis to see if the schedule of dialysis sessions affects blood flow to the legs. Some patients did standard 4-hour sessions three times a week, while others did shorter 2-hour sessions six times a week. The goal was to find out whic…

This study followed 37 people with a genetic risk for hereditary ATTR amyloidosis over five years. Researchers measured levels of misfolded proteins in the blood to see if they could detect the earliest signs of the disease. The goal is to develop a way to catch the condition bef…

This study aimed to find out how often people in Reunion Island start dialysis as an emergency and what social or medical factors lead to that. Researchers followed 233 adult patients beginning dialysis at local centers. The goal was to gather information to improve care and redu…

This study checked how common smell and taste problems are in 51 people with cardiac amyloidosis, a rare disease where abnormal proteins build up in the heart. Researchers used a simple smell test called Sniffin' Sticks to measure these senses. The goal was to see if these issues…

This study looked at the gut microbiome (the bacteria living in the digestive tract) of 44 people with mild cognitive impairment, early Alzheimer's, or healthy memory. Researchers wanted to see if lifestyle changes could alter the gut microbiome and whether those changes might be…

This completed study looked at 110 people with cardiac amyloidosis, a condition where protein deposits build up in the heart. Researchers implanted a small device under the skin to continuously monitor heart rhythms for up to several years. The goal was to learn more about when a…

This study followed 170 older adults who had a type of stroke caused by cerebral amyloid angiopathy (CAA), a condition where protein builds up in brain blood vessels. Researchers wanted to see if a specific finding on MRI scans, called cortical superficial siderosis (cSS), could …

This study looked at 15 stroke patients with a condition called cerebral amyloid angiopathy (CAA), which makes blood vessels in the brain fragile and prone to bleeding. All patients received a clot-busting drug (thrombolysis) and were checked for brain bleeding 24 hours later. Re…

This study looked at brain scans from 315 people with two types of small vessel disease: cerebral amyloid angiopathy (CAA) and hypertensive arteriopathy (HA). Researchers wanted to see if the pattern of white matter changes around the brain's fluid-filled spaces could help tell t…

This study looked at 111 people with cerebral amyloid angiopathy (CAA), a condition that causes bleeding in the brain. Researchers used MRI scans to check for iron deposits in the cerebellum, a part of the brain that controls balance and coordination. The goal was to learn where …

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…

This study looked at 167 adults with chronic kidney disease on either hemodialysis or peritoneal dialysis to compare their blood levels of calcium, phosphorus, and other minerals linked to bone health. The goal was to see if one dialysis method is associated with fewer mineral an…

This study used special brain scans (PET imaging) to measure amyloid plaques in 60 people with Alzheimer's disease or related conditions like posterior cortical atrophy and logopenic progressive aphasia. The goal was to understand how age influences the amount of amyloid in the b…