Restrictive cardiomyopathy

This study tests an experimental drug called CAEL-101 in 125 people with a rare disease called AL amyloidosis, where abnormal proteins build up in organs like the heart and kidneys. The drug is designed to remove those protein deposits. The goal is to see if it helps people live …

This study tests whether benralizumab can improve lung structure and function in people with severe eosinophilic asthma who still have symptoms despite standard treatments. About 75 adults aged 18-70 will receive either the drug or a placebo for 48 weeks, followed by a 4-week che…

This phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…

This study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…

This phase 3 trial tests whether CAEL-101, an antibody that removes abnormal protein deposits from organs, can help people with AL amyloidosis live longer and avoid heart-related hospital stays. About 281 participants who have not yet received treatment for their plasma cell diso…

This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for…

This phase 2 trial tests an oral drug called venglustat, given alongside the standard enzyme therapy Cerezyme, in adults with Gaucher disease type 3 (a rare genetic disorder affecting the body and brain). The study has four parts: first, it checks spinal fluid biomarkers to disti…

This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…

This early-stage trial tests a combination of three drugs—venetoclax, ixazomib, and dexamethasone—in 24 patients with light chain amyloidosis that has returned or not responded to prior treatment. The study focuses on finding the safest dose and checking side effects. All partici…

This study tests a drug called eplontersen in Chinese adults with a heart condition caused by abnormal protein buildup. The goal is to see if the drug lowers that protein and is safe over time. Participants will be randomly assigned to receive the drug or a placebo for 24 weeks, …

This study tests whether the drug tafamidis can safely stop the progression of ATTR amyloidosis in people who have already received a heart transplant. About 25 stable patients will take tafamidis daily for 12 months. Researchers will measure changes in blood protein levels, nerv…

This phase 2 trial tests a drug combination including daratumumab for people with AL amyloidosis, a rare disease where abnormal proteins damage organs, especially the heart. The study has two groups: one gets the full combo right away, the other starts some drugs later. Researche…

This Phase 3 study tests a weekly injection called retatrutide in about 1,946 people with severe obesity (BMI ≥35) and established heart disease. The goal is to see if it helps with weight loss and improves heart-related health measures over 113 weeks compared to a placebo. Parti…

This study tests whether a weekly injection of CagriSema (a combination of two drugs) can reduce the risk of heart attacks, strokes, and other heart-related events in people with established cardiovascular disease. About 7,100 participants will receive either CagriSema or a place…

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 study tests a technique called acute normovolemic hemodilution (ANH) in 2000 high-risk heart surgery patients. Before the heart-lung machine is used, doctors remove some blood and replace it with fluids. The goal is to see if this reduces the number of patients who need a bl…

This study is for people with systemic amyloidosis, a disease where abnormal proteins build up in organs. It tests the long-term safety of an experimental drug called AT-02. About 120 adults will receive the drug and be monitored for side effects and how the drug behaves in the b…

This phase 3 trial tests whether a monthly injection of pelacarsen can reduce major heart problems like heart attacks and strokes in people with high levels of Lp(a), a genetic risk factor. Over 8,000 participants with existing heart disease are being followed for several years. …

This trial aimed to test an experimental drug called CM-336 for people with AL amyloidosis that has returned, not responded to treatment, or only partially responded after initial therapy. The drug works by helping the immune system attack and destroy harmful cells. However, the …

This study is checking the long-term safety of a drug called eplontersen in people with ATTR-CM, a heart condition caused by abnormal protein buildup. About 1400 adults who completed earlier studies will receive eplontersen injections. Researchers will monitor side effects, kidne…

This study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. …

This Phase 3 study tests whether benralizumab, a biologic injection given every 4 weeks, can reduce flares in people with hypereosinophilic syndrome (HES), a rare condition where too many eosinophils cause organ damage. About 134 patients aged 12 and older will receive either ben…

This study is monitoring 110 adults in Korea who are taking Vyndamax (tafamidis) for a rare heart condition called ATTR-CM. The goal is to track side effects and see how well the drug works over 10 years in everyday medical practice. Researchers will check heart function and symp…

This large study is testing whether a lower blood pressure target can reduce major heart problems like heart attacks, strokes, and heart failure in people with type 2 diabetes. About 9,500 participants with high blood pressure and high cardiovascular risk are being randomly assig…

This trial tests whether a procedure called catheter ablation works better than anti-arrhythmic drugs for people with structural heart disease who have had a dangerous fast heart rhythm (ventricular tachycardia). About 162 participants will be randomly assigned to either ablation…

This study tests whether the drug imatinib (Gleevec) can safely lower high levels of eosinophils (a type of white blood cell) in people with hypereosinophilic syndrome (HES), a rare condition that can damage organs. About 70 adults and children (age 2 and up) with a specific form…

This early-phase trial tests a combination of three drugs—azacitidine, venetoclax, and pevonedistat—in adults newly diagnosed with a challenging form of acute myeloid leukemia (AML). The goal is to find the best dose and see if the combo can improve remission rates. About 40 part…

This study tests whether adding daratumumab back to pomalidomide and dexamethasone can improve complete remission rates in 15 adults with relapsed or refractory AL amyloidosis who have already received daratumumab. Participants receive the three-drug combination to control the di…

This Phase 3 trial tests an experimental drug called ALXN2220 in about 1,180 adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein builds up in the heart, causing heart failure. The drug is designed to remove those protein deposits. Partic…

This study is for people with a rare heart condition called ATTR amyloidosis with cardiomyopathy, where abnormal protein builds up in the heart. It tests the long-term safety and effectiveness of a drug called vutrisiran, given as an injection every three months. About 700 adults…

This study aimed to test a monthly injection called ION-682884 in people with TTR amyloid cardiomyopathy, a condition where abnormal protein deposits damage the heart. Only patients who had already completed a 2-year study of a similar drug (inotersen) were eligible. The goal was…

This early-phase trial tests whether adding the drug isatuximab to standard chemotherapy can safely treat people with high-risk AL amyloidosis, a rare disease where abnormal proteins damage organs. Eleven participants will receive the combination to see if it reduces toxicity and…

This early-phase trial tests a single intravenous dose of LY-M001 gene therapy in 9 children (ages 6 to 17) with type 1 Gaucher disease. The goal is to see if it is safe and can improve key symptoms like liver size and blood markers. Researchers will monitor participants for side…

This phase 2 trial tests a new enzyme replacement therapy called avalglucosidase alfa (Nexviazyme) in 22 children with infantile-onset Pompe disease who are not responding well to standard treatment. The study aims to see if the new drug is safe and can improve outcomes. Particip…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests a drug called vutrisiran in 655 adults with a rare heart condition caused by abnormal protein buildup (ATTR amyloidosis with cardiomyopathy). The drug is given as a shot every 3 months and aims to reduce deaths and heart-related hospital stays. The goal is to see…

This study tests whether giving patients capsules containing gut bacteria (called Probacine) before and after heart surgery can reduce gut injury and organ failure. Researchers will enroll 500 adults undergoing cardiovascular surgery. The goal is to see if changing the gut microb…

This study tests whether colchicine, a drug used for gout, can improve blood vessel stiffness and heart function in people with high blood pressure. About 150 adults with hypertension and additional risks like diabetes or high cholesterol will take colchicine or a placebo daily f…

This study tests whether a second round of treatment with ALXN2220 is safe and can improve heart function in people with transthyretin amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. About 35 adults who previously received the drug will get it a…

This phase 2 trial tests whether daratumumab, an antibody that targets cancer cells, can improve outcomes for people with multiple myeloma after they receive a stem cell transplant. The study involves 31 participants and is currently active but not recruiting. The main goal is to…

This phase 2 trial is testing whether the oral chemotherapy drug ixazomib can help control AL amyloidosis after initial treatment. Seventeen adults with the disease will take ixazomib along with low-dose dexamethasone to see if it delays organ progression or the need for more the…

This study looks at whether the drug eplontersen can reduce abnormal protein buildup in the heart for people with a condition called ATTR-CM. About 150 participants from a larger trial will get heart scans after 140 weeks of treatment. The goal is to see if the drug helps clear t…

This study tests the long-term safety of an experimental drug called NNC6019-0001 in people with transthyretin amyloid cardiomyopathy, a rare heart disease that can lead to heart failure. All 80 participants have already completed an earlier study and will receive the drug every …

This study follows 75 people with Gaucher disease type 1 who previously received FLT201 gene therapy. Researchers will monitor them for 5 years to see if the treatment remains safe and if its effects last. The goal is to understand whether a single dose can provide long-term dise…

This study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the dru…

This study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least…

This study is checking the long-term safety of a drug called acoramidis in people with a rare heart condition called transthyretin amyloid cardiomyopathy. About 389 participants who finished a previous 30-month trial will take acoramidis twice daily for up to 5 years. Researchers…

This study tests a new, more stable version of the chemotherapy drug melphalan in people with multiple myeloma or AL amyloidosis who are getting a stem cell transplant. The goal is to find the best dose that reaches a target level in the body, while possibly causing fewer side ef…

This Phase 3 trial tests dexpramipexole, an oral tablet, as an add-on treatment for people with eosinophilic asthma that is not well controlled. About 600 participants aged 12 and older will receive either the drug or a placebo for 24 weeks. The study measures improvements in lun…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study is testing a way to find cardiac amyloidosis—a rare but serious heart condition—earlier in people aged 65 and older. Researchers in Turkey will screen 800 patients at family medicine clinics using simple blood tests and medical history. The goal is to see if this appro…

This study tests whether a new, more powerful 7 Tesla MRI can better detect scarring and swelling in the heart muscle of people with cardiomyopathy. Researchers will scan 13 adults aged 20-70 to see if the higher-resolution images improve diagnosis. The goal is to see if this adv…

This study explores whether a simple, non-invasive leg movement test can measure blood vessel health in older adults. Researchers will use ultrasound to track blood flow changes after a passive leg movement, comparing young healthy people and older patients undergoing heart tests…

This study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …

This large study tested whether taking daily vitamin D3 and omega-3 (fish oil) supplements can lower the risk of developing cancer, heart disease, or stroke. Over 25,000 healthy older adults (men 50+, women 55+) took the supplements or placebos for about 5 years. The goal is to s…

This study tests a nurse-led education program designed to help women in cardiac rehab address sexual concerns after a heart event. Sixty-six women will be randomly assigned to either the program or usual care. The goal is to see if the program improves sexual function, mood, and…

This study tests whether adding blood flow restriction (BFR) to standard resistance training helps heart rehab patients get stronger and healthier. Twenty adults with stable heart disease will do exercises with and without a special cuff that limits blood flow to the arms and leg…

This study tests whether a nerve block given before heart surgery can reduce pain and the need for opioid painkillers afterward. About 150 adults having heart surgery will receive either a long-acting or standard form of the numbing drug bupivacaine via ultrasound-guided injectio…

This study tests a short program that helps heart patients and their families explore what gives life meaning during cardiac rehab. About 80 people in Denmark will try one of three formats: one-on-one, with a relative, or in a group. The goal is to see if this approach can reduce…

This study tests whether a drug called siltuximab can reduce symptoms like weakness, fatigue, nausea, and pain after a stem cell transplant in patients aged 60-75 with multiple myeloma or AL amyloidosis. The drug blocks a protein linked to inflammation, which may help patients re…

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 tracks 120 adults with heart disease and at least two other long-term illnesses to see how often dangerous drug interactions happen. Researchers check medications, including over-the-counter and herbal products, and follow participants for a year. The goal is to unders…

This study looked at whether having more frequent contact with a clinician (at least once a week) improves satisfaction for people with long-lasting musculoskeletal conditions. Participants were split into two groups: one with standard contact and one with extra check-ins via tex…

This study uses anonymous health insurance records from Germany to learn more about a rare heart condition called ATTR-CM, where abnormal protein builds up in the heart. Researchers will look at survival rates, hospital visits, and how common the disease is. No new treatments are…

This study tracks over 1,700 heart patients who had hip or knee replacement surgery. Researchers want to see how often bleeding or blood clots happen in the months after surgery. The goal is to find better ways to prevent these complications.

This study tests whether a Norwegian clinic (NSAC) helps people with common mental health issues or muscle pain return to work sooner. 2500 adults are split into three groups: one gets treatment quickly, one waits 10-14 weeks, and one gets a basic check-up. Researchers will track…

This study will observe 200,000 Chinese adults with heart disease to see how their heart and lung function changes over 5 years. Participants will take a special exercise test to measure their fitness. Researchers hope to find better ways to predict future heart problems like hea…

This study looks at whether mepolizumab, a drug already used to reduce asthma attacks, can also improve physical activity and exercise tolerance in people with severe asthma. Researchers will track daily steps and activity levels in 62 adults over six months. The goal is to see i…

This study tracks 40 people with nasal polyps and severe asthma who are taking mepolizumab, a drug that reduces inflammation. Researchers collect nose, blood, and sputum samples before treatment and at 3, 6, and 12 months to see how the drug affects symptoms, nose bacteria, and g…

This study is a large registry that collects medical data and tissue samples from 30,000 people with heart disease and healthy volunteers. Researchers will analyze this information to better understand risk factors, treatments, and outcomes for various heart conditions. The goal …

This study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years…

This study follows 2000 people with dilated cardiomyopathy (a weakened, enlarged heart) over several years to learn how genetics and heart scarring affect the disease. Participants give blood for genetic testing and have heart MRI scans. The goal is to improve diagnosis and pave …

This study looks back at the medical records of about 4 million people who had physical or occupational therapy for muscle and joint problems. Researchers want to see if different ways of giving therapy lead to different results. No new treatments are tested—the goal is to learn …

This study aimed to see if a mobile monitoring tool could accurately identify surgical site infections (SSIs) in orthopedic surgery patients, compared to the usual manual review. The study was withdrawn before enrolling any participants, so no results are available. It was design…

This study is a disease registry that collects information from routine medical care of about 350 adults diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM) in Taiwan, Hong Kong, and Malaysia. Researchers will look at patient characteristics, treatments received, and ho…

This study looks at how being in a hospital intensive care unit (ICU) after heart surgery affects your body's natural sleep-wake cycle. Researchers will track sleep patterns and thinking ability in 15 adults to see if disrupted rhythms slow down recovery. The goal is to understan…

This study aims to find biological markers in blood, saliva, and sputum that can predict how well patients with EGPA (a rare inflammatory disease) and severe asthma respond to the drug mepolizumab. Researchers will analyze proteins in these samples before and after treatment to u…

This study creates a database of 476 patients in the Bronx who were tested for transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often missed heart condition. Researchers collected health records, imaging results, and outcomes like hospitalizations and deaths. The goa…

This study looks at 50 people with TTR amyloid cardiomyopathy, a condition where abnormal proteins build up in the heart. Researchers want to see if a special SPECT scan can measure blood flow in the heart's tiny vessels, which may be impaired even without blocked arteries. Parti…

This study aimed to see if using a special pad along with a standard closure device could help patients get out of bed sooner after a heart catheterization. It was planned for adults having the procedure through the groin. However, the study was withdrawn before any participants …

This study uses MRI scans to measure amyloid protein buildup in the hearts of up to 150 people with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Participants are already enrolled in a larger treatment trial. The goal is to see how amyloid levels change over time, not …

This study compares two types of radiation therapy for left-sided breast cancer: proton beam therapy and conventional photon therapy. The goal is to see which one causes fewer changes to the heart, measured by MRI scans. About 60 adults with non-metastatic breast cancer will part…

This registry follows 75 pregnant women with heart disease to better understand the risks and outcomes for both mother and baby. Researchers will track health data for one year and check vital status at five years. The goal is to fill knowledge gaps and improve future care for th…

This study follows 347 heart surgery patients to understand why some develop swallowing problems (dysphagia). Researchers will use special camera tests to watch how patients swallow and look for risk factors. The goal is to create simple tools that doctors can use at the bedside …

This study is following 10,000 adults who are having a heart MRI for medical reasons. Researchers want to see if the MRI results can help predict who will have heart problems in the future, like heart attacks or heart failure. The goal is to better understand how MRI scans can gu…

This study follows 20 newborns and children diagnosed with late-onset Pompe disease through newborn screening. Researchers will track their health for up to 4.5 years to document early muscle and joint symptoms, biomarkers, and quality of life. The goal is to learn when to start …