Intrinsic cardiomyopathy

This trial is testing an experimental treatment called FKC289 for people with primary AL amyloidosis that has relapsed or not responded to standard care. FKC289 uses a patient's own immune cells, engineered to recognize and attack the abnormal plasma cells that produce the harmfu…

This study tests a minimally invasive procedure called SESAME for people with thickened heart muscle that blocks blood flow from the heart. The procedure uses a catheter to make a controlled slice in the muscle, aiming to relieve symptoms like shortness of breath. Researchers wil…

Researchers are studying depemokimab, an injected biologic drug, in adults aged 18 to 75 who have allergic bronchopulmonary aspergillosis (ABPA) along with severe eosinophilic asthma. The study tracks how many asthma attacks need systemic steroids and whether participants' qualit…

Researchers are testing whether trametinib, an FDA-approved cancer drug, can help infants with RASopathies who have a severe, life-threatening heart condition called hypertrophic cardiomyopathy. The trial enrolls about 25 babies with a confirmed genetic diagnosis. Participants re…

Researchers are testing whether adults with obstructive hypertrophic cardiomyopathy can safely taper off beta-blockers after their heart condition has stabilized on mavacamten. The trial enrolls 286 people aged 18 to 75 who have been on a stable dose of mavacamten and one beta-bl…

This phase 3 trial is testing whether an experimental drug called BHB-1893 works better than the standard beta-blocker metoprolol for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), a condition where the heart muscle thickens and blocks blood flow. The stu…

This study tests whether adjusting the dose of mavacamten based on heart pumping strength can further reduce the blockage of blood flow in people with obstructive hypertrophic cardiomyopathy. Participants are adults who have been on a stable mavacamten dose for at least 12 weeks …

This phase 3 trial compares an investigational drug called etentamig against a standard combination therapy (daratumumab, cyclophosphamide, bortezomib, and dexamethasone) in people newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Abo…

This study tests the long-term safety and effectiveness of an experimental drug called ALXN2220 in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart and impair its function. Participants who completed an earlier …

This early-stage trial is testing an experimental oral medication called S-606001 in adults with late-onset Pompe disease, a condition that causes progressive muscle weakness and breathing problems. The main goal is to see if the drug is safe and tolerable. Researchers will also …

This early study tests whether the drug CAEL-101/anselamimab can bind to and shrink abnormal protein deposits (amyloid) in people with AL amyloidoma. Five adults with measurable amyloid deposits will receive the drug and be monitored for up to 72 weeks. The goal is to see if the …

This phase II trial is testing whether a combination of selinexor, daratumumab, and dexamethasone (called XDd) can help people with advanced light-chain cardiac amyloidosis, a condition where abnormal proteins build up in the heart. The study aims to see if the regimen can achiev…

This study tests the long-term safety and tolerability of acoramidis, an oral medication that stabilizes the transthyretin protein, in people newly diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition that can lead to heart failure. Participants who complete…

This phase 2 trial is testing an investigational drug called QLS4131 in people newly diagnosed with AL amyloidosis, a condition where abnormal proteins build up in organs and cause damage. QLS4131 is a trispecific antibody designed to target and eliminate the plasma cells produci…

This study tests the long-term safety and effectiveness of an experimental oral drug called S-606001 in adults with late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Participants who completed a prior S-606001 study can join, and they will rec…

This study tests an experimental drug called elranatamab in people newly diagnosed with AL amyloidosis who have not yet had treatment. The drug helps the body's immune cells find and destroy the abnormal cells causing the disease. Researchers want to see if it can produce deep re…

This trial explores whether starting transdermal estradiol (a skin gel or patch) soon after menopause can protect the heart and brain in women who have hot flashes and at least one cardiovascular risk factor. One hundred women will be randomly assigned to receive the hormone ther…

This study tests a new pacing method called anodal biphasic pacing (ABP) in 108 adults who need a heart procedure or have a pacemaker. ABP uses a different electrical waveform that may improve how the heart pumps and reduce problems from standard pacing. Researchers will compare …

This trial tests whether a mechanical, spring-driven device called FreedomEDGE® can safely and effectively deliver daratumumab, a standard treatment for multiple myeloma and amyloidosis. Currently, nurses give these injections by hand, which can be physically demanding. About 100…

This phase 3 trial compares an experimental CAR-T cell therapy, NXC-201, with a standard drug combination (daratumumab plus CyBorD) in people newly diagnosed with AL amyloidosis, a rare condition where abnormal proteins damage organs. Participants receive either a single infusion…

This study tests whether pelvic floor muscle training (PFMT) with a handheld biofeedback device can improve erectile function and sexual quality of life in men with functional hypogonadism (low testosterone) and erectile dysfunction. Participants are randomly assigned to PFMT wit…

This study tests two chemotherapy-free treatments for people newly diagnosed with AL amyloidosis, a rare blood disorder. The choice of treatment depends on whether the patient has a specific genetic change called t(11;14). Those with t(11;14) receive a daily pill called sonrotocl…

This phase 2 trial tests an oral drug called Aom0304 in adults with hypertrophic cardiomyopathy, a condition where the heart muscle is abnormally thick. The study includes people with both obstructive and non-obstructive forms. Participants receive different doses of Aom0304 for …

This Phase 2 trial tests a new drug called QLS4131, given under the skin, in combination with other medications for people with malignant plasma cell neoplasms like multiple myeloma. The study will compare different drug combinations to see which works best at shrinking tumors an…

This study will test whether sildenafil, a drug often used for erectile dysfunction, can improve heart function in children aged 1-18 with dilated cardiomyopathy, a condition where the heart is enlarged and weak. Researchers will measure changes in the heart's pumping ability ove…

This study tests whether a 12-month digital program with health coaching and a wearable device can reduce scarring in the heart muscle caused by high blood pressure. Researchers will enroll 200 adults aged 40-70 with early signs of heart thickening. The goal is to see if lifestyl…

This early-phase study tests a new treatment called CAR19-BCMA CAR-T for people with certain blood cancers (plasma cell neoplasms or lymphomas/leukemias) that have returned or not responded to at least three prior treatments. The therapy uses a patient's own immune cells, modifie…

This study tests a new treatment for people with a rare disease called light-chain cardiac amyloidosis that has come back or not responded to other treatments. The treatment uses specially engineered immune cells (CAR-NK cells) from donated umbilical cord blood to target and atta…

This study tests whether routinely placing a small tube in the leg artery (distal perfusion catheter) is better than waiting until a problem arises in patients on mechanical heart pumps. About 500 adults with severe heart failure or shock will be randomly assigned to one of the t…

This phase 2b trial tests AVTX-801, a D-galactose supplement, in 8 adults with PGM1-CDG, a rare genetic disorder affecting sugar metabolism. Participants currently on D-galactose will receive either the study drug or a placebo to see if it reduces disease-related events like low …

This study looks at whether an implantable defibrillator (ICD) can reduce the risk of sudden cardiac death in people with non-ischemic dilated cardiomyopathy, a type of heart muscle disease not caused by blocked arteries. Researchers will use MRI scans to find heart scarring (fib…

This early trial tests whether baricitinib, an immune-calming drug already used for arthritis, can reduce heart inflammation in people with cardiac sarcoidosis. Ten adults will take the drug for 16 weeks alongside their usual steroid-sparing medication. Researchers will use PET s…

This Phase 3 trial tests whether the anti-inflammatory drug anakinra can help children with acute myocarditis (heart inflammation) recover heart function faster. About 110 children in intensive care will receive either anakinra or a placebo for 7 days, alongside standard care. Th…

This study tests whether a herbal medicine called Danhong injection can help protect the heart in people with sepsis, a life-threatening infection. Researchers will give 140 patients either the injection or a placebo for 7 days and check heart function and survival rates. The goa…

This early-phase trial tests a new type of immune cell therapy called GPRC5D CAR-T in 18 people with relapsed or treatment-resistant plasma cell disorders, such as multiple myeloma. The therapy involves giving patients a single infusion of their own modified immune cells designed…

This early-phase trial will test whether injecting lab-grown heart muscle cells (made from stem cells) into the heart can safely help people with severe heart failure. Fourteen participants with weak hearts will receive the cells via a catheter. The main goal is to check for side…

This study tests whether adding melatonin (a natural sleep hormone) to standard heart failure treatment can improve heart function in women with peripartum cardiomyopathy, a rare heart condition that develops during or after pregnancy. 25 women will receive either melatonin, sele…

This study tests whether henagliflozin, a drug currently used for diabetes, can reduce scarring in the heart muscle of people with a type of hypertrophic cardiomyopathy (non-obstructive HCM). The trial will enroll 150 adults and use special PET scans to measure changes in scar ti…

This early study tests whether high-dose vitamin B3 (nicotinamide) can help preserve or improve vision in people with Leber's hereditary optic neuropathy (LHON), a rare genetic disease that causes sudden vision loss. Researchers will give 13 participants 2 grams of vitamin B3 dai…

This Phase 2 trial tests AD17002, a nasal spray that aims to calm the immune system in people with moderate to severe eosinophilic asthma that is not well controlled by current treatments. About 126 adults will receive either the spray or a placebo for 3 months. Researchers will …

This study compares two heart failure medications—sacubitril/valsartan (a newer combo drug) and captopril (an older drug)—in 70 children aged 1 month to 18 years with dilated cardiomyopathy (an enlarged, weak heart). The goal is to see which drug better improves the heart's pumpi…

This study will test whether adding a special breathing exercise (inspiratory muscle training) to standard cardiac rehab helps heart patients recover better. About 80 adults who have been hospitalized for a heart condition will take part. The main goal is to see if the training i…

Researchers at Turku University Hospital are testing a PET imaging tracer called Al[18F]F-NOTA-folate to see how well it detects two heart conditions: cardiac sarcoidosis, an inflammatory disease of the heart muscle, and infective endocarditis, an infection of the heart valves. T…

This study tests whether a special PET-CT scan using a drug called evuzamitide can detect signs of cardiac amyloidosis in people whose standard heart scans were unclear but who have high levels of a blood marker called TAD1. About 25 adults with heart failure or a genetic risk fo…

This study looks at whether taking a tiny tissue sample from the heart during a pacemaker or defibrillator implant can help diagnose transthyretin cardiac amyloidosis (ATTR-CM), a condition where abnormal proteins build up in the heart. The trial enrolls people aged 65 and older …

This early-phase study tests whether a special ultrasound with a targeted contrast agent (Sonazoid) can quickly diagnose heart inflammation caused by immune checkpoint inhibitor cancer drugs. About 30 adults who recently received these drugs and show signs of heart inflammation w…

This study explores whether a PET/MR scan using a radioactive tracer called 18F-Florbetaben can detect amyloid deposits—abnormal protein clumps that can damage organs—in people with certain blood disorders. Participants receive a single injection of the tracer followed by a whole…

This study will test whether a faster type of bone scan (CZT-SPECT) can accurately diagnose transthyretin cardiac amyloidosis, a serious heart condition. Researchers will compare the new scan with the standard method in 200 patients. If it works, it could make diagnosis quicker a…

This study tests whether a computer model can find signs of a heart condition called cardiac amyloidosis in bone scans that were done for other reasons. Researchers will look back at 57 patients' scans to see if the model can correctly identify those with the disease. If it works…

This early study tests a new radioactive tracer called 64Cu-GRIP B that targets a protein released by immune cells during heart inflammation. Researchers will scan 10 people with acute myocarditis to see if the tracer can clearly highlight inflamed areas of the heart. If it works…

This study tests whether a structured 5-week cardiac rehabilitation program can improve exercise ability and quality of life in 40 adults with cardiac amyloidosis. Participants will undergo supervised aerobic and strength training five days a week, with tests before and after the…

This trial investigates whether a structured, moderate-intensity cardiac rehabilitation program is safe for people with arrhythmogenic cardiomyopathy who carry high-risk genetic mutations. Participants will be randomly assigned to either the exercise program or standard physical …

This study tests whether increasing the pacemaker's lower rate from 60 to 80 beats per minute improves quality of life and daily function in people with ATTR cardiac amyloidosis. The heart in this condition is stiff and depends on a faster heart rate to pump blood. Thirty-four pa…

This study tests whether a home-based exercise program combined with nutritional supplements can improve walking ability and muscle strength in people aged 70 and older with transthyretin cardiac amyloidosis, a heart condition that causes weakness and frailty. About 102 participa…

This early-stage trial tests amlodipine, a calcium channel blocker used for high blood pressure, in 20 adults with myotonic dystrophy type 1. The goal is to see if the drug is safe and can improve muscle strength, reduce stiffness, and help with daily function. All participants w…

This pilot study will test a new method to see how two pain medicines, naproxen and pregabalin, work in healthy adults. Up to 25 participants will receive each medicine and a placebo in random order. They will undergo several short, controlled pain tests, like a cold water hand b…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This study looks at whether a digital tool called Luscii is practical and acceptable for people with a rare heart condition called ATTR-CM. About 60 adults in Austria, Germany, and Italy will use a smartphone app and home devices to share health information with their care team. …

This study is building a biobank of blood samples and imaging data from 300 people with suspected inflammatory heart or autoimmune rheumatic diseases. Researchers aim to identify immune biomarkers that could predict major heart events like heart failure, stroke, or atrial fibrill…

This study looks at whether biologic therapy can reverse long-term lung damage in adults with severe asthma. Researchers will follow 150 patients and 50 healthy volunteers for two years, using advanced imaging and lung function tests. The goal is to see if treatment leads to 'cli…

This study follows 100 adults with myotonic dystrophy (types 1 or 2) for two years to understand how muscle stiffness, daily function, and heart health change over time. Researchers will look back at up to 18 months of past medical records and then track participants with clinic …

This study looks at people with obstructive hypertrophic cardiomyopathy — a condition where the heart muscle is abnormally thick and blocks blood flow. Researchers want to see if certain abnormalities in the mitral valve or its supporting structures affect how well the drug mavac…

This study looks back at the medical records of 300 people with arrhythmogenic cardiomyopathy who have an implantable cardioverter-defibrillator (ICD) and have had dangerous heart rhythms. Researchers want to count how often ICD-related problems occur, such as lead failure or ina…

This study investigates whether measuring blood flow in the heart's smallest blood vessels (microcirculation) can help predict serious health events within a year after a person is hospitalized for acute myocarditis. Researchers will use a special technique during a standard hear…

This study will collect health information from 3000 people with hypertrophic cardiomyopathy (HCM) across multiple hospitals in China. The goal is to build a comprehensive database to understand how the disease is currently diagnosed and treated, and to identify factors that affe…

This study will follow 200 people with suspected or confirmed cardiac amyloidosis, a condition where abnormal proteins build up in the heart and cause heart failure. Over two years, participants will undergo heart MRI scans, exercise tests, and blood work at regular visits. The g…

This study will measure hormone levels in 100 children aged 1 to 12 who have dilated cardiomyopathy, a condition where the heart is enlarged and weakened. Researchers will collect blood samples to check hormones like thyroid, cortisol, and growth factors, and compare them to heal…

This observational study will enroll 1,000 pregnant women and their fetuses to establish normal ranges for heart sounds and ECGs. The goal is to develop early warning systems for serious heart events in mothers, progression of fetal heart defects, and autoimmune-related fetal hea…

This study will check children under 18 with chronic liver disease for hidden heart problems. Doctors will use simple tests like ECG and ultrasound to look for issues such as abnormal heart rhythms or weakened heart muscle. The goal is to better understand how liver disease affec…

This study looks back at medical records of 1500 adults hospitalized for acute myocarditis (heart muscle inflammation). The goal is to find out how often dangerous heart rhythms occur and what factors predict them. No new treatments are tested—researchers will use existing data t…

This study aims to better understand a condition called multilocus imprinting disorder (MLID), where multiple genes are affected by abnormal chemical marks. Researchers will test a new technique to detect these marks in 96 people, including those with known imprinting disorders a…

This study watches how dilated cardiomyopathy (a weakened heart muscle) progresses in adults with specific BAG3 gene changes. Researchers will track heart function and health events over time in 30 participants. No treatment is given—the goal is to better understand the disease.…

This study looks at how a person's genes affect their response to the heart drug mavacamten for obstructive hypertrophic cardiomyopathy. Researchers will collect DNA samples from 300 patients to see which genetic types respond best. The goal is to improve dosing safety and effect…

This study gathers information from 5,000 adults with hypertrophic cardiomyopathy, a condition where the heart muscle is too thick. Researchers will use heart MRI scans to look for scarring and build a better way to predict who is at risk of dying from the disease. The goal is to…

This study will follow 1,500 people with lupus or other autoimmune diseases to see if advanced heart scans can predict heart attacks, strokes, or other heart problems. Participants will have one of three types of heart imaging. The goal is to learn if these scans can help doctors…

This study is creating a registry for people with Duchenne and Becker muscular dystrophy, as well as symptomatic female carriers. The goal is to collect health data and quality-of-life information to monitor how new therapies work in real-world settings. Up to 1,500 participants …

This study aims to find out why the heart medication mavacamten works better for some people with hypertrophic cardiomyopathy (a condition where the heart muscle is too thick) than for others. Researchers will look at participants' genes, including the specific gene causing their…

This study will collect data from 5,000 adults with acute myocarditis (heart muscle inflammation) across France. Researchers will analyze heart MRI scans to see if certain patterns can predict who is at risk for serious complications like death, heart failure, or dangerous heart …

This study will observe 143 older adults with severe aortic stenosis who are scheduled for a minimally invasive valve replacement procedure called TAVR. Researchers want to find out how many also have cardiac amyloidosis, a condition where abnormal proteins build up in the heart.…

This study looks at whether a new medication (mavacamten) can improve the energy levels of the heart muscle in people with obstructive hypertrophic cardiomyopathy, a condition where the heart wall thickens and blocks blood flow. Researchers will use a special MRI scan to measure …

This study looks at why people with Noonan syndrome often bleed easily, especially from the skin, mouth, or nose. Researchers will compare a simple questionnaire about bleeding history with blood tests in 100 patients. The goal is to find better ways to predict serious bleeding, …

This study will follow 100 children aged 1 to 18 with myocarditis (inflammation of the heart muscle) to see how different types of the disease progress. Researchers will track symptoms, treatments, and outcomes over one year. The goal is to better understand which children recove…

This study looks at 100 adults with frequent extra heartbeats (PVCs) to see if hidden heart inflammation (myocarditis) is the cause. Participants will get advanced heart scans and lab tests. The goal is to better understand the link and see if anti-inflammatory therapy helps thos…