Intrinsic cardiomyopathy

This early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must…

This study tests a new growth hormone medicine called somapacitan, given once a week, in 47 children who are very short due to being born small for gestational age, or having Turner syndrome, Noonan syndrome, or idiopathic short stature. The main goal is to see if it is safe and …

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 tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

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 study tests a new device that uses extreme cold (cryoablation) to treat a fast, dangerous heart rhythm called sustained monomorphic ventricular tachycardia. About 206 adults with this condition will receive the freezing treatment. The goal is to see if it safely stops the ab…

This phase 4 study tests the drug mavacamten in 63 adults with a type of heart muscle disease called obstructive hypertrophic cardiomyopathy. The goal is to see if the drug can improve heart structure, measured by MRI, and ease symptoms like shortness of breath. Participants take…

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 study tests a new drug called EDG-7500 in 100 adults with hypertrophic cardiomyopathy, a condition where the heart muscle is abnormally thick. The goal is to check the drug's safety and how it affects heart function. Participants receive single or multiple doses, and researc…

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 phase 2 trial is testing whether combining daratumumab, venetoclax, and dexamethasone can produce a deep and rapid response in people newly diagnosed with light-chain (AL) amyloidosis who carry a specific genetic marker called t(11;14). The study enrolls 36 participants and …

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 trial tests whether an 8-week course of the immunosuppressive drug prednisone can reduce episodes of ventricular tachycardia (a potentially fatal fast heart rhythm) and improve heart function in people with non-ischemic cardiomyopathy and confirmed heart inflammation. Partic…

This phase 3 study is testing the long-term safety and effectiveness of an experimental drug called AOC 1001 (del-desiran) for people with myotonic dystrophy type 1 (DM1), a genetic muscle disorder. The trial enrolls 230 adults who have completed a prior AOC 1001 study and will r…

This study tests a drug called teclistamab in 30 people with AL amyloidosis, a rare disease where abnormal proteins damage organs. Participants have already had other treatments. The goal is to see if teclistamab can reduce or eliminate the harmful proteins in the blood. The stud…

This study tests whether benralizumab, an injected drug, can reduce asthma attacks in children aged 6 to 17 with severe eosinophilic asthma. About 200 participants will receive either benralizumab or a placebo, and researchers will track how long it takes for their first asthma f…

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 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 study tests a new treatment called NXC-201 CAR-T for people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. The therapy uses a patient's own immune cells, modified to target and destroy the cells producing these proteins. The trial is open to …

This phase 2b trial tests whether adding ALXN2220 to eplontersen works better than eplontersen alone for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal proteins build up in the heart. About 326 participants will receive either the combinati…

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 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 study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find th…

This trial investigates whether intravenous infusions of tiny particles (extracellular vesicles) from heart progenitor cells can safely improve heart function in people with severe dilated cardiomyopathy and heart failure. Participants receive three infusions and are monitored fo…

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 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 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 phase 3 trial is testing a drug called aficamten in 36 Japanese adults with symptomatic obstructive hypertrophic cardiomyopathy, a condition where the heart muscle thickens and makes it hard to pump blood. The drug aims to reduce over-contraction of the heart muscle. Researc…

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 study tests if a new growth hormone medicine (somapacitan) given once a week works as well as the standard daily growth hormone (Norditropin) for children who are very short due to being born small, or having Turner syndrome, Noonan syndrome, or unknown causes. About 412 chi…

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 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 study tests a device called AccuCinch that is placed around the heart to help it pump more effectively. It is for people with heart failure and a weakened heart muscle. The goal is to see if the device improves quality of life, exercise ability, and reduces serious heart eve…

This study tests a new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tu…

This study tests whether a special heart pacing technique (left bundle branch pacing) combined with standard heart failure drugs can improve heart function better than drugs alone. It includes 50 adults with non-ischemic heart failure and a specific heart block. The main goal is …

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 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 tests the drug mavacamten in 50 adults in India who have a condition where the heart muscle is too thick and blocks blood flow, causing symptoms like shortness of breath and chest pain. The goal is to see if the drug is safe and works well. Participants will take the d…

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 2 trial is testing whether a patient's own bone marrow stem cells can improve heart function and ease symptoms in people with dilated cardiomyopathy and heart failure. Twenty participants will have stem cells collected from their hip and then infused into their heart a…

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 early-phase study tests a new drug called ERAS-601, alone or with other cancer treatments, in adults with advanced solid tumors that have not responded to standard therapies. The main goals are to find safe doses and check for side effects. About 90 participants will take pa…

This study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safe…

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 study tests a single dose of SRP-9003 gene therapy in 17 people with limb girdle muscular dystrophy 2E/R4, a genetic muscle-weakening disease. The goal is to restore a missing protein in muscle cells and improve muscle function. Both walkers and non-walkers can join, and the…

This expanded access program gave a single patient with Leber Hereditary Optic Neuropathy (a genetic cause of vision loss) an experimental gene therapy called GS010. The treatment was injected into both eyes to test safety. Only one person was involved, so the results are very li…

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 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 a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein …

This phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. T…

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 early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a…

This phase 3 trial tests an experimental drug called AOC 1001 in 159 adults with myotonic dystrophy type 1, a genetic condition that causes muscle weakness and stiffness. Participants receive either the drug or a placebo by intravenous infusion. The main goal is to see if the dru…

This study follows 900 people with hypertrophic cardiomyopathy who have already taken aficamten in earlier trials. Researchers want to see if the drug is safe and tolerable over a longer period. Participants take aficamten tablets daily, with regular clinic visits to monitor for …

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 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 a drug called mavacamten in 44 teenagers aged 12 to 18 who have a condition where the heart muscle is too thick and blocks blood flow. The goal is to see if the drug can reduce the blockage and improve symptoms. Participants are randomly assigned to receive eithe…

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 study tests whether a combination of anti-inflammatory and heart-protecting drugs can reduce symptoms and heart damage in people with Long COVID who have ongoing heart inflammation. About 279 adults with heart inflammation seen on MRI will receive either the drug combo or a …

This study tests a new surgery done on a beating heart to help people with a type of thick heart muscle disease that causes symptoms like chest pain and shortness of breath. The surgery aims to remove a small part of the thickened heart muscle to improve blood flow and heart func…

This study tests a one-time gene therapy called AB-1002, given directly into the heart arteries, for people with advanced heart failure (NYHA Class III). The goal is to see if it can improve heart function, reduce symptoms, and lower the risk of death or hospitalization. About 15…

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 early-stage trial tests a gene therapy called LX2020 for people with a heart condition called arrhythmogenic cardiomyopathy, caused by a change in the PKP2 gene. The study involves 10 adults who already have a defibrillator implanted. The main goal is to see if the treatment…

This study follows 10 people who already received LX2020 gene therapy for arrhythmogenic cardiomyopathy caused by a PKP2 gene mutation. Researchers will monitor them for years to see if the treatment remains safe and continues to help control the disease. The goal is to understan…

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 enrolls about 4,000 adults aged 60 and older with heart failure who are at high risk for ATTR amyloidosis, a rare disease that stiffens the heart. Doctors receive special training on how to spot and diagnose the condition using standard tests. The goal is to see if thi…

This study is testing whether artificial intelligence can detect transthyretin amyloid cardiomyopathy (ATTR-CM), a serious and often underdiagnosed heart condition, earlier than usual. Researchers will use AI algorithms to analyze routine ECGs and heart ultrasounds from up to 1.5…

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 looks at whether adding just 3 minutes to a cardiac MRI can help doctors find heart inflammation (myocarditis) early in cancer patients on immunotherapy. Myocarditis is a rare but often deadly side effect of these drugs. The researchers will scan 200 patients who alrea…

This study tests whether an artificial intelligence (AI) program can detect hypertrophic cardiomyopathy (HCM), a serious heart condition, using ECGs from standard machines, the Apple Watch, and the KardiaMobile device. Researchers will compare the AI's accuracy across these devic…

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 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 phase 3 trial tests whether aficamten, an oral drug, can improve quality of life and exercise capacity in 500 adults with symptomatic non-obstructive hypertrophic cardiomyopathy, a heart condition that makes the heart muscle thick and stiff. Participants receive either afica…

This Phase 3 study tests whether the drug sotagliflozin can improve symptoms and daily function in 500 adults with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Participants take either the drug or a placebo pill once daily for 26 weeks. The main…

This study explores whether weekly music and movement sessions are practical and enjoyable for children aged 6 to 18 with myotonic dystrophy type 1 (DM1). Over 10 weeks, participants attend 45-minute music classes, undergo physical and cognitive tests, and provide biological samp…

This study tests whether empagliflozin, a drug typically used for diabetes, can help people with hypertrophic cardiomyopathy (a thickened heart muscle) who do not have diabetes. About 250 adults will receive either the drug or a placebo daily for 12 months. The main goals are to …

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …

This study is testing the long-term safety and effectiveness of a drug called mexiletine PR for people with myotonic dystrophy types 1 and 2. The drug is taken once daily as a liquid to help reduce muscle stiffness (myotonia). The study includes 176 adults and teens who have alre…

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 is developing a digital twin—a virtual model of the heart and blood vessels—to better understand and manage hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will gather detailed health data from 2500 people with HCM or the gen…

This study follows people who previously received NTLA-2001, an experimental gene-editing therapy, for a type of amyloidosis that can affect nerves or the heart. The goal is to monitor long-term safety and see if the therapy's effects on disease markers persist over time. Partici…

This study examines genetic test results from over 600 people who had acute myocarditis (heart muscle inflammation) confirmed by MRI. Researchers want to find out how common certain gene variants are in this group and whether they are linked to worse outcomes like heart failure o…

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 looks at how myotonic dystrophy types 1 and 2 affect the brain. About 100 adults aged 30-65 will have MRI scans, thinking and movement tests, and blood draws. Some will also have a spinal tap. The goal is to find brain changes that could be used as markers in future tr…

This study looks at whether detailed analysis of heart MRI images (called radiomics) can better predict life-threatening heart rhythms in people with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will also see if the drug mavacamten, …

This study tested whether a special PET imaging probe called [68Ga]CBP8 can measure scarring (fibrosis) in the hearts of people with cardiac amyloidosis, a rare condition where abnormal proteins build up in the heart. Researchers compared 18 participants with cardiac amyloidosis …

This study follows about 35 people with dilated cardiomyopathy caused by a BAG3 gene mutation, a condition that weakens the heart muscle. Over three years, researchers will monitor heart function, symptoms, and quality of life using tests like imaging and blood work. The goal is …

This study follows 1000 Chinese patients with myotonic dystrophy type 1 (DM1) to understand how the disease affects multiple body systems and leads to disability. Researchers will collect health data and blood samples every 3 to 6 months for several years. The goal is to identify…

This study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …

This study looks at whether diabetes or blood sugar problems can worsen thinking and memory issues in people with myotonic dystrophy type 1. Researchers will use MRI brain scans and thinking tests over 4 years to track changes. The goal is to understand why some patients have mor…

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 aims to make genetic testing for inherited heart muscle disease available directly in heart failure clinics, instead of requiring a separate referral to a genetics specialist that can take years. Researchers will train heart doctors to order a simple blood or saliva te…

This study follows 319 people under 21 who developed heart inflammation (myocarditis or pericarditis) within 21 days of receiving the Comirnaty (Pfizer) COVID-19 vaccine. Researchers will monitor their heart function with imaging tests for up to 5 years to see if there are lastin…

This study follows 331 adults with hypertrophic cardiomyopathy (HCM) to see how they are treated in everyday medical practice, especially with the drug mavacamten. Researchers will collect data on diagnosis, symptoms, treatments, and outcomes over time. The goal is to understand …

This study looks at over 1,200 adults with type 2 diabetes and heart muscle problems to see if diabetic cardiomyopathy is a unique condition. Researchers will use detailed medical tests, imaging, and biological data to find patterns. The goal is to better understand and diagnose …

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 looks at 57 people who received a COVID-19 vaccine to understand why some develop heart injury or myocarditis. Researchers use advanced heart scans (PET/MRI) and blood tests to find early warning signs. The goal is to better identify those at risk and improve follow-up…

This study uses advanced heart MRI scans to look for the earliest signs of dilated cardiomyopathy (a weakened heart muscle) in close relatives of people already diagnosed. Researchers will follow 650 family members over time to see how genetic factors relate to heart changes. The…

This study looks at whether a smartwatch ECG can predict heart muscle disease (cardiomyopathy) in adult survivors of childhood cancer as well as a standard clinic ECG does. About 1,300 survivors will have both a standard 12-lead ECG and a 30-second Apple Smartwatch ECG. The goal …

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 looks at whether a new type of heart medication, called a cardiac myosin inhibitor, can improve blood flow in the small blood vessels of the heart in people with hypertrophic cardiomyopathy (HCM). Researchers will use special ultrasound imaging to measure blood flow be…

This study tests whether using an ultrasound measure of heart muscle coordination (mechanical dyssynchrony) can better select heart failure patients for cardiac resynchronization therapy (CRT) than current guidelines. About 500 patients will receive a CRT device, but pacing will …

This study follows 50 athletes who had acute myocarditis (heart muscle inflammation) to see how often they develop dangerous heart rhythms. Participants wear a portable ECG monitor and do a treadmill stress test at 3 months and 1 year after their illness. The goal is to learn mor…

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 measures how often people with myotonic dystrophy type 1 fall and what factors affect their balance. Researchers will also assess fear of falling and leg muscle strength. The goal is to better understand fall risk in this condition.

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 heart rhythm problems in people with hypertrophic cardiomyopathy, a condition where the heart muscle is thicker than normal. Researchers want to see how often new rhythm issues like atrial fibrillation happen, and whether sleep apnea plays a role. About 260 ad…

This study tests a digital tool called AsthmaOptimiser that helps doctors assess asthma control and find ways to improve treatment during a single visit. About 746 adults with asthma who have had recent flare-ups will take part. The goal is to see if the tool can identify better …

This observational study will enroll 10,000 adults with hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens. Researchers will combine genetic tests, advanced heart imaging, and blood markers to create better tools for early diagnosis and to predict risk…

This study is testing whether an artificial intelligence (AI) model can automatically write reports for cardiac MRI scans as accurately as experienced radiologists. Researchers will compare AI-generated reports with those written by experts using 20,000 patient scans. The goal is…

This study collects blood or skin samples from 100 adults with inherited heart rhythm disorders (like Long QT Syndrome or Brugada Syndrome) and healthy volunteers. Researchers will turn these samples into stem cells and then into heart cells to study how these diseases work and t…

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 will use advanced cardiac MRI scans to look at different patterns of heart inflammation in 5,000 people with myocarditis or inflammatory cardiomyopathy. The goal is to see if certain MRI findings can better predict who will have serious heart problems later, like heart…

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 observational study looks at 165 people with two different types of heart muscle thickening: syndromic/metabolic cardiomyopathy (SMCs) and hypertrophic cardiomyopathy (HCM). Researchers want to see if SMCs patients have worse heart outcomes like death or heart failure, and w…

This study looks at people with rare adrenal tumors (pheochromocytomas and paragangliomas) that do not produce excess hormones. Researchers want to see if these 'silent' tumors still cause heart problems, blood pressure changes, or metabolic issues like diabetes and high choleste…

This study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…

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 is creating a large database of 1400 adults and children with hypertrophic cardiomyopathy (HCM) in Russia. Researchers will track their health over time to learn how the disease progresses and affects daily life. The goal is to improve diagnosis and care for HCM patien…

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 study follows 205 people with four types of limb-girdle muscular dystrophy (LGMD) to understand how the disease changes over time. Participants will have their muscle strength, movement, and breathing tested regularly for up to 5 years. No treatment is given; the goal is to …

This study was designed to track the natural course of gamma-sarcoglycanopathy (LGMDR5), a rare muscle-weakening disease, over two years. Researchers planned to measure changes in muscle strength, walking ability, and daily function in patients aged 6 to 35. The goal was to bette…

This study is a registry that will follow up to 221 children with Noonan Syndrome who are already taking or starting Norditropin® for short stature. Researchers will collect information on growth, side effects, and quality of life over time. No new treatment is being tested—the g…

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 …