Dilated cardiomyopathy

This study tests a small device called C-MIC that is implanted without open-heart surgery to help people with heart failure. It includes 22 adults with weak heart pumping (ejection fraction between 25% and 50%) despite standard medications. The device delivers microcurrents to th…

This study tests whether bromocriptine, a drug that stops breastfeeding, can improve heart function in women newly diagnosed with peripartum cardiomyopathy (a type of heart failure after childbirth). 200 women will receive either bromocriptine or a placebo, plus standard heart fa…

This early-stage trial tests a single dose of a gene therapy called NVC-001 in 21 adults with a genetic form of dilated cardiomyopathy (LMNA mutation). The therapy uses a harmless virus to deliver a modified gene into heart cells, aiming to protect the heart muscle. The main goal…

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This study tests an experimental daily pill, danicamtiv, in 332 people with genetic or familial dilated cardiomyopathy (a weakened, enlarged heart). Participants take the drug or a placebo for about 6 months to see if it improves heart function and exercise ability. The goal is t…

This early-stage study tests a new medicine called AZD4063 in 31 adults with a specific genetic form of dilated cardiomyopathy (PLN R14del). The main goal is to check the drug's safety and how the body processes it. Participants receive the drug as an injection under the skin and…

This early-stage trial tested a new cell therapy called CAR-DC in 8 people with end-stage dilated cardiomyopathy, a condition that weakens the heart. The therapy targets a protein called FAP to calm the immune system and improve heart function. The main goal was to check safety, …

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

This early-stage trial tests a single intravenous dose of AFTX-201, a gene therapy designed to deliver a working copy of the BAG3 gene to heart cells. It enrolls 22 adults aged 18–55 with dilated cardiomyopathy and a BAG3 mutation. The study primarily checks safety and tolerabili…

This trial investigates whether empagliflozin, a drug originally used for diabetes, can help children with heart failure. About 60 children aged 1 month to 18 years with confirmed heart failure will receive either standard care plus empagliflozin or standard care alone for 90 day…

This early study tests whether a second infusion of a patient's own modified immune cells (CAR-DC) is safe and can improve heart function in 5 adults with end-stage dilated cardiomyopathy. Participants had already received one dose but saw their heart function worsen again. The g…

This early-stage trial tests a new cell therapy for people with heart failure caused by blocked arteries. The treatment uses the patient's own immune cells, engineered to target scar tissue in the heart. The study will check safety and whether it can improve heart function in 15 …

This early-stage trial tests a one-time gene therapy called RP-A701 in 8 adults with dilated cardiomyopathy caused by a faulty BAG3 gene. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to improve heart function. The main goal is to check safety, b…

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This early-stage trial tests whether infusions of stem cells from donated newborn tissue are safe for young people aged 4 to 39 with dilated cardiomyopathy, a condition where the heart becomes enlarged and weak. Up to 36 participants will receive three intravenous doses over 30 d…

This phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…

This early-stage study tests a one-time gene therapy called ALXN2350 for adults with a specific genetic heart condition (BAG3-related dilated cardiomyopathy). The heart muscle becomes weak and enlarged, making it hard to pump blood. The treatment aims to fix the faulty gene to im…

This study tests whether a carefully controlled high-fat diet can improve heart function in people with a specific type of heart failure (non-ischemic dilated cardiomyopathy). Researchers believe that switching the heart's fuel source back to fats may help it pump more effectivel…

This study tests whether adding empagliflozin (a diabetes drug) to standard carvedilol treatment can prevent liver decompensation events and death in 400 adults with cirrhosis and left ventricular diastolic dysfunction (stiff heart). Participants will receive either empagliflozin…

This study compares two approaches for people with ischemic cardiomyopathy (weak heart muscle due to blocked arteries) and multiple blocked vessels. One group gets optimal medical therapy alone, while the other also gets a heart artery procedure (PCI) guided by imaging and pressu…

This study tests whether injecting donor stem cells (from healthy volunteers) can safely improve heart function in people with a weakened heart muscle not caused by blocked arteries. About 136 adults aged 18-80 with low pumping ability will receive either stem cells or a placebo.…

This study tests a gene therapy called GS010 for people with Leber hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. Researchers will give the treatment as an injection into the eye at two different doses to see if it improves vision and mitoc…

This pilot trial tests whether a patient's own stem cells can help the heart muscle recover in people with advanced chronic heart failure who already have a mechanical heart pump (LVAD). Ten patients will receive their own CD34+ stem cells injected into a heart artery. Researcher…

This study tests whether injecting stem cells from umbilical cords into the heart during bypass surgery can help repair heart muscle and improve function in people with chronic heart failure. About 51 adults with weak hearts (ejection fraction 40% or less) will take part. The goa…

This study tests a special pacing technique called high-intensity his bundle pacing in 34 adults with heart failure and a narrow QRS pattern on their ECG. Standard pacing doesn't help these patients, but this new approach may improve heart function and quality of life. Participan…

This study tests whether a newer pacing method called left bundle branch pacing (LBBP) works better than the standard biventricular pacing for people with chronic heart failure and reduced pumping ability. About 60 adults with heart failure and a specific electrical delay in the …

This phase 2 study tests a new radioactive imaging agent, 18F-mFBG, to measure nerve damage in the hearts of 20 people with stable heart failure and implantable defibrillators (ICDs). The goal is to see if the agent can reveal differences in nerve function between those who have …

This study tests an artificial intelligence program that analyzes heart rhythm data from a small, wearable patch to estimate how well the heart pumps blood. Researchers will compare the AI's results to standard ultrasound in 2,000 adults. If accurate, this could offer a simpler, …

This study explores whether a quick, painless cheek swab can spot protein changes linked to inherited heart conditions that cause sudden cardiac death in young people. Researchers will collect samples from 26 patients with arrhythmic disorders or family members at risk. If the te…

This study will enroll 5,000 adults with chronic heart conditions like heart failure, high blood pressure, or aortic stenosis. Participants will wear a Prolaio digital health patch that monitors heart rate, breathing, and other vital signs. The goal is to observe how these condit…

This study is looking at whether advanced heart imaging, especially cardiac MRI, can help identify the cause of cardiomyopathy and predict risks like death or heart failure in a diverse group of patients. The research focuses on adults in the Bronx, including many from Hispanic a…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study aims to understand how heart problems develop in people with Duchenne and Becker muscular dystrophy, as well as in carriers. Researchers will collect cardiac MRI scans and clinical data from 1,000 participants to build a registry. Using advanced image analysis and deep…

This study looks at a heart condition called cirrhotic cardiomyopathy that can affect people with advanced liver disease (cirrhosis). Researchers want to learn how common it is, what increases the risk, and whether bile acids in the blood play a role. About 440 adults with decomp…

This study aims to uncover the genetic causes of cardiomyopathy in children by analyzing DNA from affected individuals and their families. Researchers hope to identify mutations that lead to different types of cardiomyopathy, which could improve genetic counseling and deepen unde…

This study will collect electronic health records from up to 2500 people with Duchenne or Becker muscular dystrophy, including female carriers, across U.S. clinics. The data will be combined with patient-reported information to give researchers a fuller picture of the diseases. T…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study is building a registry of 1,211 people in Brazil who have inherited heart conditions like cardiomyopathy, arrhythmias, and Marfan syndrome. Researchers will collect DNA samples and medical information to discover which genes are most commonly affected and how often the…

This study is building a worldwide online registry for people with Duchenne or Becker muscular dystrophy, including female carriers. Participants share their health information to help researchers learn more about the disease and to match patients with clinical trials. The goal i…

This study is looking for new genes and blood markers linked to arrhythmogenic cardiomyopathy (AVC), a genetic heart condition that can lead to heart failure and sudden cardiac arrest. Researchers will enroll 1,000 people, including patients with AVC or unexplained cardiac arrest…

This study looks at whether brain wave tests (EEG) done before and after heart surgery in babies under 1 year old can predict later learning or behavior problems, such as autism or ADHD. About 50 infants will be followed to age 2. The goal is to find early warning signs so that c…

This study aims to create a tool that uses stress heart MRI and routine medical data to predict which patients with dilated cardiomyopathy (a weakened, enlarged heart) are at highest risk for serious problems like heart failure or sudden cardiac death. Researchers will follow 2,0…

This study is building a large online registry of 10,000 adults with cardiomyopathy or myocarditis, plus those with a strong family history. Researchers will track participants over time to see how many experience serious events like heart failure, dangerous heart rhythms, or dea…

This study aims to improve how doctors predict the risk of sudden cardiac death or heart failure in people with inherited heart diseases. Researchers will use artificial intelligence to analyze data from 1,000 participants, including medical history, ECGs, imaging, genetic tests,…

This study uses magnetic resonance spectroscopy, a special MRI technique, to measure the heart's energy levels without needles or biopsies. Researchers will compare healthy volunteers, people with heart failure, and those with other heart conditions to see if low energy metabolis…

This study aims to identify genetic factors that affect how severe muscle and heart problems become in people with LMNA gene mutations. Researchers will collect skin and muscle samples from 40 participants and use advanced DNA and RNA analysis to look for protective or aggravatin…

This study tests whether a wrist-worn device can help monitor pregnant women with congenital heart disease. Researchers will track heart rhythms and other data from 50 participants to see if the wearable can detect early signs of trouble. The goal is to improve care and reduce th…

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study is looking at 400 people with weak hearts who are getting an implantable defibrillator (ICD) to prevent sudden cardiac death. Researchers want to see if detailed heart MRI scans can find structural problems that better predict who will actually have a dangerous heart r…

This study follows 1500 people who already have an implantable cardioverter-defibrillator (ICD) to prevent sudden cardiac death. Researchers will track heart function, genetics, and blood markers to find better ways to predict who is at highest risk. The goal is to improve how do…

This study looks at fat tissue surrounding the heart arteries in 60 patients who had a heart transplant. Researchers want to see if the size of fat cells close to the artery differs from those farther away, and if that relates to artery disease. The goal is to better understand h…

This study looks at heart tissue samples from 216 adults who already need a biopsy as part of their care. Researchers want to find unique molecular patterns in different heart conditions, including after a heart transplant. The goal is to improve how these diseases are diagnosed …

This study will use cardiac MRI scans to measure heart tissue in 2000 people with various heart conditions, such as heart attacks, cardiomyopathies, and myocarditis. The goal is to create a reference map of normal and abnormal tissue values, which may help doctors diagnose diseas…

This pilot study is testing a new type of PET/CT scan that uses a radioactive tracer called 68Ga-FAPI to detect active scarring (fibrosis) in the hearts of 30 people with heart failure. The goal is to see if this scan can spot early or ongoing scarring better than current MRI met…

This study looks at how problems in the heart's tiny blood vessels (coronary microvascular dysfunction) affect long-term outcomes in people with dilated cardiomyopathy, a condition where the heart muscle is enlarged and weak. Researchers will follow 330 participants who have alre…

This study compares two ways to manage fluids in cirrhosis patients with severe sepsis or septic shock. One method uses a bedside heart ultrasound (POCUS) to guide fluid amounts, while the other follows standard sepsis guidelines. The goal is to see if ultrasound-guided care redu…

This study is testing a new non-invasive device that uses laser speckle to measure blood flow and structure inside the eye. Researchers will compare these images with standard vision tests in 500 people with various retinal conditions. The goal is to see if this technology can be…

This 24-month observational study will follow up to 1000 people with certain types of muscular dystrophy (LGMD, DM2, and late-onset Pompe disease) aged 6-50. Researchers want to see if specific physical tests, like the North Star Assessment and a 100-meter walk, are good ways to …

This study looks at people who carry genetic changes linked to Leber Hereditary Optic Neuropathy (LHON) but still have normal vision. Researchers want to see if certain chemicals in the blood and tears can signal early nerve damage in the eye, before vision loss occurs. The goal …

This study is for adults with a weak heart muscle and a leaky mitral valve (functional mitral regurgitation). Researchers will use advanced MRI scans to better predict which treatments—medication or a minimally invasive valve repair—will work best for each person. The goal is to …

This study tests an online clinic (eCG Family Clinic) that helps families with inherited heart conditions get genetic counseling and DNA testing from home. Researchers will see if more family members use the service and how satisfied they are compared to standard care. About 170 …