Restrictive cardiomyopathy

This Phase 3b study is testing whether the drug benralizumab (Fasenra) can allow people with severe eosinophilic asthma to safely reduce their daily maintenance medications. About 504 participants aged 12-75 in China will try to step down from drugs like inhaled steroids or long-…

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 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 the drug mepolizumab in 16 children aged 6 to 17 with hypereosinophilic syndrome (HES), a rare condition causing too many eosinophils (a type of white blood cell). The goal was to see if mepolizumab could reduce disease flares and lower the need for steroids ove…

This study tested the safety and effectiveness of eliglustat, an oral medication, in 57 children aged 2 to 17 with Gaucher disease types 1 and 3. Some children also received the standard enzyme therapy imiglucerase. Researchers measured how the drug moves through the body, side e…

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 early-stage trial tested a new gene therapy called FLT201 in 10 adults with Gaucher disease type 1. The therapy uses a harmless virus to deliver a working copy of the gene that produces a missing enzyme. The main goal was to check safety and see if the treatment can boost en…

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 early-phase study tested a new drug called ABX1100 in 46 people, including healthy volunteers and patients with late-onset Pompe disease. The main goal was to check safety and how the drug moves through the body. Researchers gave single or multiple doses to find the right am…

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 study tested whether a drug called mepolizumab, given after a severe COPD flare-up that required hospital care, could prevent future hospital stays or death. It involved 238 adults with eosinophilic COPD (a type with high levels of certain white blood cells). Participants re…

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 study followed 152 adults in Greece with severe eosinophilic asthma who were starting benralizumab, a medication that helps control severe asthma. Researchers measured not only standard asthma control but also quality of life, sleep, mood, and physical activity using wearabl…

This early-phase study tested a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The main goal was to check safety and side effects. Participants received a single intravenous dose, and researchers monitored their health over time.

This study looked at how well benralizumab (Fasenra) works for people with severe eosinophilic asthma in real-world clinics across the Gulf, Latin America, and India. Researchers tracked 291 patients to see if their asthma control improved over time using a standard questionnaire…

This study looked at whether using a HEPA air purifier at home can improve lung health in adults with a type of COPD called eosinophilic COPD. 178 people took part, with half getting real purifiers and half getting fake ones. The main goal was to see if the purifiers helped with …

This study tested the long-term safety of a combination therapy (ATB200 and AT2221) in 119 adults with late-onset Pompe disease who had completed a prior study. Participants received the drugs for an extended period, and researchers tracked side effects, walking distance, and lun…

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 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 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 followed 106 adults with severe eosinophilic asthma in Poland who received mepolizumab injections every 4 weeks for at least a year. Researchers tracked asthma attacks and the need for oral steroids before and during treatment. The goal was to see if the drug helps con…

This study looked at whether dupilumab, a drug that blocks certain immune signals, can reduce signs of autoimmunity in the airways of people with severe asthma. 30 patients were followed for 4 months. The goal was to see if this approach could help those who don't get better with…

This study tested the drug dasatinib in 68 adults with several types of blood and bone marrow disorders, including certain leukemias, myelofibrosis, and mastocytosis. The goal was to see if dasatinib could help control the disease and to check its safety. Participants took dasati…

This study tested whether using cardiac MRI to guide ablation (a procedure that destroys small areas of heart tissue) can improve outcomes for people with ventricular tachycardia, a dangerous fast heart rhythm. 104 adults with structural heart disease were randomly assigned to on…

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 study tested whether a new combination of two drugs (ATB200 and AT2221) is safe for adults with Pompe disease, a rare genetic disorder that causes muscle weakness. 29 participants received the drugs through an IV and by mouth. The main goal was to check for side effects and …

This phase 3 trial tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. Participants received either the experimental combo (cipaglucosidase alfa plus miglustat) or the current standard therapy (alglucosidase…

This study tested an experimental nasal spray called AD17002 in 40 adults with poorly controlled moderate-to-severe eosinophilic asthma. Participants self-administered the spray twice a week for six weeks. The goal was to see if it could improve lung function and reduce the need …

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 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 explores whether a cardiac MRI can safely and effectively gather the same heart pressure and flow measurements normally obtained through right heart catheterization, a more invasive procedure. Researchers will attempt to perform the catheterization inside the MRI machi…

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 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 early-phase study tested whether losartan, a common blood pressure medication, could prevent early heart damage caused by radiation therapy in breast cancer patients. Five women with left-sided breast cancer who were scheduled for radiation took losartan. Researchers used he…

This study looks at whether older adults with a heart condition called cardiac amyloidosis feel better and can exercise more when they stop taking beta-blockers. Twenty participants will each go through two periods: one on their beta-blocker and one off it. Researchers will measu…

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 high-dose inspiratory muscle training (IMT) using a handheld device is safe and feasible for people with late-onset Pompe disease. 34 adults with stable Pompe disease did remote breathing exercises. Researchers measured changes in respiratory strength an…

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 looks at how mepolizumab (Nucala), a treatment for severe eosinophilic asthma, impacts emotions and mental well-being in patients and their partners. Researchers will track changes in feelings like anxiety, depression, and stress using questionnaires and interviews. Th…

This study investigates whether people with late-onset Pompe disease have a higher risk of severe blood vessel problems in the brain and aorta. Researchers will collect medical and genetic data from adults with Pompe disease and from those who have had a brain aneurysm or bleedin…

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 tested a decision algorithm to help doctors diagnose the cause of eosinophilia (high levels of a type of white blood cell). The algorithm guides which tests to do and in what order. Researchers enrolled 53 patients and measured how many followed the algorithm correctly…

This study looks at how inherited heart rhythm problems (like Long QT Syndrome) and heart muscle diseases (like Hypertrophic Cardiomyopathy) affect the quality of life and physical activity of children aged 6 to 18. Researchers will compare 107 children with these conditions to 1…

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 collected information from over 15,000 people hospitalized with cardiomyopathy, a heart muscle disease. Researchers looked at how much these hospital stays cost and what the money was spent on. The goal was to understand the real-world financial impact and help manage …

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 different types of immune cells (eosinophils) in the blood of people with asthma, chronic sinusitis with nasal polyps, or both, compared to healthy volunteers. The goal was to find patterns that could help doctors choose the best targeted therapy for each pat…

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 looked at 22 people with severe aortic stenosis who were scheduled for valve replacement. Before surgery, they had an ultrasound of the heart, and during surgery a tiny sample of heart muscle was taken to check for scarring (fibrosis). The goal was to see if ultrasound…

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 how the asthma drug mepolizumab affects gene activity in adults with severe eosinophilic asthma. Researchers took blood samples from 20 participants before and after 12 weeks of treatment to see which genes changed. The goal was to understand how the drug wor…

This study looked at 86 people with severe aortic valve stenosis who had valve replacement surgery. Researchers used special MRI scans to measure scarring in the heart muscle before and one year after surgery. The goal was to see if the scarring improves and whether MRI could hel…

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 create and test a new questionnaire called CARD-SARC that measures how cardiac sarcoidosis affects patients' daily lives and well-being. Researchers worked with 130 adults diagnosed with cardiac sarcoidosis at two NHS specialist centres. The goal was to develo…

This study measured levels of brain chemicals related to oxidative stress and inflammation in people with type 1 Gaucher disease and healthy volunteers. Researchers gave 33 participants with Gaucher disease the antioxidant N-acetylcysteine (NAC) for about 90 days to see if it cha…

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 looked for new biological markers to help diagnose hypereosinophilic syndrome (HES), a rare condition where too many eosinophils (a type of white blood cell) cause damage. Researchers compared blood samples from 41 people with HES, those with other causes of high eosin…

This study looked at how mepolizumab, a biologic drug, changes inflammation in people with severe eosinophilic asthma. Researchers measured immune and metabolic markers in the nose, lungs, and blood of 15 adults. The goal was to better understand the drug's full impact beyond jus…

This small pilot study looked at whether simple breath and urine tests can measure how well muscles produce energy in children with metabolic myopathies (rare muscle disorders). Three participants completed a 12-week at-home physiotherapy program. The goal was to see if these non…

This study looked at whether menopause makes the heart stiffer and reduces exercise ability in female endurance athletes. Researchers compared postmenopausal female athletes to premenopausal females and older male athletes using heart scans and exercise tests. The goal is to unde…

This study tested new MRI methods to detect bleeding inside the heart muscle after a heart attack. Researchers studied 14 patients to see if these scans could better identify damage that raises the risk of heart failure. The goal was to improve imaging, not to test a treatment.