Familial restrictive cardiomyopathy

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 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 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 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 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 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 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 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 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 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 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 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 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 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 compares three ways of delivering genetic counseling to adults with inherited heart conditions. Some participants get counseling before testing, others get it after, with or without a required phone call. The goal is to see which approach is most efficient and empoweri…

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 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 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 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 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 …