Glycogen storage disease due to acid maltase deficiency, late-onset

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This early-stage trial is testing a new drug called DNL952 in 32 adults with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. Participants will receive the drug through an IV infusion at different doses.

This study tests whether taking a daily fiber and magnesium supplement (WellBiome) for 6-8 weeks before heart surgery can improve recovery. Researchers will compare ICU time, complications, and hospital stay between 80 patients who get the supplement or a placebo. The goal is to …

This study tests an experimental oral drug called S-606001 in 45 adults with late-onset Pompe disease. Participants take the drug or a placebo on top of their standard enzyme replacement therapy. The main goal is to see if the drug improves lung function and walking ability over …

This large phase 3 trial is testing whether adding vicadrostat to the existing drug empagliflozin can reduce the risk of heart problems in adults with type 2 diabetes, high blood pressure, and heart disease. About 11,800 participants will take either the combination or a placebo …

This study looks at the long-term safety of the drug pelacarsen in people with high lipoprotein(a) and heart disease. About 5,700 participants who finished a previous pelacarsen trial will receive the drug and be monitored for side effects and heart events. The goal is to see if …

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the tre…

This study is testing a digital app called VIRTUES that provides educational resources and treatment recommendations for heart conditions like atrial fibrillation and heart failure. Researchers will enroll 2000 patients across 11 modules to see if the app is easy to use and helps…

This phase 3 trial is testing whether adding baxdrostat to the standard drug dapagliflozin can better prevent heart failure and cardiovascular death in people with type 2 diabetes who already have heart disease and high blood pressure. About 11,300 participants will receive eithe…

This study tests whether a special activity score, calculated from heart rate data, can help people who have completed cardiac rehab stick to exercise guidelines. About 318 participants will wear a smartwatch for 4 months and do an exercise test at the end. Researchers will compa…

This study compares two methods for closing the breastbone after open-heart surgery: rigid plate fixation (using a metal plate and screws) versus traditional wire cerclage (using stainless steel wires). The trial will enroll 250 adults at high risk for wound complications, such a…

This study tests whether text messages can help people who have finished cardiac rehab keep up healthy habits like weight management, physical activity, and taking medications. Researchers will enroll 400 people from groups often left out of heart research. The study compares dif…

This study tests a device called Efferon LPS that filters harmful substances from the blood during and after heart surgery. The goal is to see if it can reduce the risk of multiple organ failure, a serious complication. Researchers will enroll 60 adults having heart surgery with …

This study tests whether genetic testing can help veterans at high risk for heart disease start and stick with statin medications. About 410 veterans aged 40-75 with diabetes or heart disease who are not currently taking statins will receive either a genetic report on their stati…

This study tests a new method called decrement evoked potential (DeEP) mapping to guide catheter ablation for ventricular tachycardia (VT) in people with structural heart disease. VT is a fast, dangerous heart rhythm that can cause sudden cardiac arrest. The standard ablation pro…

This study tests whether adding digital coaching to home-based cardiac rehabilitation helps veterans complete more rehab sessions and improve their heart health. 150 veterans with heart disease will be randomly assigned to standard home rehab or home rehab plus a private social m…

This study looks at whether people at moderate risk for heart problems should continue or stop taking aspirin before a colonoscopy. About 2,500 participants will be randomly assigned to take either aspirin or a placebo for a few days around the procedure. The goal is to see which…

This study tests a noninvasive radiation treatment (SBRT) for people with ventricular tachycardia, a dangerous heart rhythm that can cause sudden death. The 12 participants have structural heart disease and have not been helped by standard treatments like drugs or catheter ablati…

This study compares a single dose of targeted radiation (SBRT) to the standard catheter ablation procedure for reducing dangerous heart rhythms in people with advanced heart disease. Sixty participants will be randomly assigned to one of the two treatments. The main goal is to se…

This study tests a program that uses an AI chatbot, health coach, and home monitoring devices to help 300 adults with heart disease, diabetes, or kidney disease manage their conditions. The goal is to see if this approach reduces hospital visits, complications, and deaths over 6 …

This study tests a program called CVD-FIT for African American adults who struggle to afford food and are at risk for heart disease. Participants get monthly income support, weekly incentives to buy healthy food, and phone-based education on heart health. The goal is to see if th…

This study is testing whether giving air conditioners and a $100 electricity subsidy to older adults in Boston neighborhoods can reduce heat stress, heat strain, and related health problems. One hundred participants aged 55-95 who don't have AC will be randomly assigned to receiv…

This study tests whether bempedoic acid, a cholesterol-lowering drug, can reduce artery inflammation and improve heart health in people with HIV. About 121 adults aged 40 and older with well-controlled HIV and at least one heart disease risk factor will receive either the drug or…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study is testing a special mattress that can measure heart rate, breathing, temperature, and body position without touching the patient. It is designed for people with heart disease who are in the hospital. The goal is to see if this wireless system works as well as standard…

This study checks if a quick, non-invasive finger test (QuantaFlo HD) can detect early signs of heart dysfunction. Researchers will compare the finger test results with standard heart ultrasound measurements in 600 adults who have risk factors like high blood pressure or diabetes…

This study is testing whether a machine learning algorithm can accurately detect heart murmurs that point to structural heart disease. Researchers will record heart sounds from 125 adults using two different digital stethoscopes and compare the algorithm's findings to standard ec…

This study tests a smartphone app designed to help people prepare for heart surgery through exercise, nutrition tips, and telehealth check-ins. About 40 adults awaiting surgery will either use the app or receive standard care. Researchers want to see if the app is easy to use, ac…

This study tests a 6-session program called BE-FIT that helps cardiac rehabilitation patients reduce anxiety about exercise. The program uses gradual exposure to feared sensations, prevents safety behaviors, and uses activity monitors for feedback. Researchers will enroll 146 adu…

This study looks at whether doing breathing exercises before orthopedic surgery (like knee, hip, or fracture repair) can help your lungs work better after the operation. It involves 36 adults who have used tobacco and are at risk for breathing problems after surgery. Participants…

This study tests whether a daily movement program led by a kinesiologist can reduce frailty in 60 hospitalized heart patients. Frailty makes people weaker and more likely to have longer hospital stays or be readmitted. The program encourages simple activities like standing, walki…

This study tests whether whole-body electrical muscle stimulation (WB-EMS) can help adults with neuromuscular diseases like ALS, SMA, and muscular dystrophy exercise safely. Because these conditions weaken the nerves that control muscles, traditional exercise is often too hard. W…

This study tests whether adding a program that targets sleep and stress (called RESST) to standard cardiac rehabilitation helps heart patients recover better. About 200 adults with heart conditions who also have poor sleep or high stress will take part. Researchers will measure s…

This study tests whether a program of exercise and ergonomics training can reduce muscle and joint pain in teachers. About 80 teachers who have had pain for at least three months will be randomly assigned to receive the training or not. Researchers will measure changes in pain, d…

This study tests a web-based app called IAMABLE that provides evidence-based rehabilitation strategies for people with chronic conditions like heart disease, arthritis, or neurological disorders. Fifty adults aged 45 to 75 will use the app for 4 months to set goals and learn abou…

This study tests whether a type of talk therapy called Behavioral Activation, delivered via home-based telehealth, can improve social functioning and mood in veterans recently discharged from the hospital for a heart condition. About 132 veterans with depression will receive eith…

This study compares a women-only cardiac rehabilitation program to the standard mixed-gender program for women with heart disease. Sixty women aged 40-80 will attend 36 sessions over 12 weeks, with extra women-focused education. Researchers will measure attendance, completion rat…

This study tests whether extra support for case managers helps veterans leaving the military better manage pain and reduce risky substance use. About 1800 veterans will take part. The program focuses on non-drug pain treatments and counseling.

This study tests a program called One-Mind One-Heart that combines mindfulness and behavior change skills to help older adults with HIV reduce stress and improve heart health. The program includes text messages and covers topics like physical activity, diet, and substance use. Th…

This study tests whether a standardized, step-by-step protocol for giving blood thinner injections (low-molecular-weight heparin) can reduce bruising and pain in heart patients. About 180 patients in cardiac care units will be randomly assigned to receive injections either by the…

This study aims to better understand and treat people with certain inherited metabolic or genetic disorders. Researchers will use standard medical tests like blood work and imaging to diagnose and care for participants, who may also join other related studies. The goal is to expa…

This registry study will enroll 200,000 high-risk cardiac patients to see how well an AI platform called Willem can detect heart problems from standard ECGs. The AI's readings will be compared to expert cardiologist diagnoses, but the AI results won't be shared with doctors, so p…

This study looks at children with suspected or confirmed genetic and metabolic disorders to find new disease-causing gene mutations. Researchers will analyze blood samples for DNA and metabolites, and in some cases take a small skin sample. The goal is to better understand these …

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study aims to learn more about heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect samples and perform tests like imaging and stress tests on up to 5,000 participants, including healthy volunteers and relatives of affect…

This study at Necker Hospital in Paris is checking how well patients with inherited metabolic diseases follow their daily oral medication routines. About 200 patients (children aged 7 and up, teens, and adults) will fill out a questionnaire during a regular visit. The goal is to …

This study creates a large database of about 12,000 people with and without heart disease. Researchers collect medical information and blood samples to analyze factors that influence heart conditions like coronary artery disease, heart failure, and stroke. The goal is to better u…

This study looks at medical records of 1,000 older heart patients in Russia to see if they were prescribed potentially harmful medications during the COVID-19 pandemic. Researchers will use a medical database to count how often these risky prescriptions happened and check for dan…

This study enrolls up to 15,000 children with known or suspected endocrine or metabolic disorders, along with their family members. Researchers will collect medical records, blood, saliva, and other samples to identify genetic changes linked to these conditions. The goal is to be…

This study is creating a large collection of blood, urine, stool, and heart tissue samples from 10,000 people with and without heart or metabolic conditions. The goal is to store these samples along with medical information to speed up future research into what causes these disea…

This study is testing whether simple nutritional checks before heart surgery can predict how well patients recover. Researchers will give 250 patients questionnaires, blood and urine tests, and measure grip strength and body composition. They will then see if these measures are l…

This study looks at whether people with type 2 diabetes, COPD, or heart disease who join a health program in Halsnaes Municipality improve their physical function and well-being. About 194 adults will be tracked before and after the program using tests and questionnaires. The goa…

This study looks at how people with ME/CFS (chronic fatigue syndrome) and people with heart conditions feel after a bike exercise test. Researchers want to see if the type and timing of symptoms after exercise are different between the two groups. 80 adults will take part, and th…

This study translates and tests several health questionnaires—covering pain, arm and leg function, mood, and daily activities—for use with Albanian-speaking people. Researchers will check if the translated versions are reliable and accurate by giving them to about 300 participant…

This study tests a new tool called the PRISM Dashboard, which gives physiotherapists feedback on how their decisions compare to their peers. The goal is to see if this feedback helps reduce differences in care for people with muscle and joint pain. About 60 physiotherapists and t…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study follows 300 people who had hospital-acquired pneumonia to see how it affects their heart and lungs over 18 months. Researchers will take blood samples, swabs, and perform heart and lung tests to understand the link between pneumonia and later cardiovascular problems li…

This study is testing a comprehensive MRI scan to see if it can better diagnose different heart conditions. Researchers will scan 2,000 adults with suspected or confirmed heart disease. The goal is to create a standard imaging method that improves early detection and risk predict…

This study is testing whether artificial intelligence can predict if someone will develop one of five common diseases: diabetes, heart disease, breast cancer, back pain, or arthritis. Researchers will collect health data from 1,000 adults aged 30 to 60, including medical records,…

This study uses advanced heart MRI to see how bariatric surgery and cardiac rehabilitation change the heart's structure and function. Researchers will scan 150 adults before and 6-12 months after these metabolic treatments. The goal is to understand how the heart adapts to these …

This study will use a new type of cardiac MRI to look at the heart's microscopic structure in 1,000 people—both healthy volunteers and those with various heart conditions. The goal is to see if this advanced imaging can detect early signs of heart damage better than standard test…

This study explores whether a non-invasive brain-computer interface (BCI) can help people with motor disorders, such as spinal cord injury or stroke, control assistive devices using their thoughts. Researchers will record brain signals with EEG and use machine learning to interpr…

This study is building a huge database of up to 800,000 Korean patients with heart or brain vascular disease. By tracking their health over many years, researchers hope to understand the unique patterns and risk factors in this population. The goal is to create a platform that ca…

This study is creating a large biobank of blood, DNA, plasma, and tissue samples from 15,000 people with and without heart and aortic diseases. Researchers will use this information to better understand conditions like aortic aneurysm, heart failure, and bicuspid aortic valve. Th…

This study aims to develop better MRI methods for imaging the heart and brain, and to describe heart diseases using these new techniques. It will also investigate whether gadolinium, a common MRI contrast agent, stays in the body long after use. Up to 5,000 healthy volunteers and…

This study tests whether a machine-learning model can predict serious heart events in children admitted to the hospital. The model alerts doctors when a child may need a palliative care consultation. Researchers will compare outcomes before and after the model is used, involving …

This study will use advanced heart MRI scans on up to 100,000 people with known or suspected heart disease. The goal is to learn how these scans can help doctors better understand, diagnose, and treat different heart conditions. Researchers will track participants' health over ti…

This study follows pregnant women with Pompe disease to see how the condition and its treatments affect pregnancy and infant growth. Researchers will track complications and monitor babies for up to three years after birth. No new drugs are being tested; participants receive thei…

This study creates a large collection of blood, tissue, and medical information from up to 5,000 people with or at risk for pediatric heart disease. Researchers will use these samples to better understand the disease and find new ways to diagnose or treat it in the future. Partic…

This study measures how hard children with conditions like neuromuscular disease, scoliosis, or heart problems work to breathe. Researchers use a thin tube placed through the nose into the esophagus to measure breathing muscle strength and effort. The goal is to better understand…

This registry is collecting information from 8,000 people undergoing heart or chest surgery. The goal is to see how factors before and during surgery relate to serious complications like death, shock, or infection. By analyzing this data, researchers hope to find ways to improve …

This study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

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 is testing whether giving probiotics after heart surgery can improve recovery by changing the bacteria in the gut. Researchers will measure certain chemicals in the blood that come from gut bacteria. The trial involves 30 adults scheduled for heart surgery, who will re…

This study looks at how the brain, nerves, and blood vessels interact in 80 adults having heart surgery. Researchers will measure brain activity, blood flow, and heart reflexes to better understand the link between heart disease and depression. The goal is to develop new tools fo…

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 follows 252 adults with muscle, bone, or joint problems (like arthritis, back pain, or after surgery) to see how well they do their prescribed home exercises. Researchers will track who sticks with the program and why, looking at factors like pain, mood, and support. T…

This study collects blood samples from 1500 adults at risk for heart disease to discover new biomarkers that can predict future heart attacks, strokes, or blocked leg arteries. Participants are seen at a specialized prevention center and provide samples and health data at the sta…

This study is a global registry that will follow about 500 people with Pompe disease over time. It includes both treated and untreated patients, and aims to collect real-world data on treatment safety, effectiveness, and quality of life. No new treatments are being tested—this is…

This study tests whether a single PET tracer (FDG) can measure both blood flow and sugar use in the heart, instead of needing two separate tracers. Researchers will compare results from FDG with a dedicated flow tracer in 60 healthy adults and people with heart disease. The goal …

This study tests whether using a virtual reality (VR) headset during shoulder exercises helps patients feel more engaged and motivated. Fifty adults with shoulder problems will do rehab exercises in a VR environment and then answer questionnaires about how easy and immersive the …

This study tests a program called Heart-to-Heart, where community health workers help low-income patients make informed choices about cardiac rehab after a heart event. About 50 adults from Johns Hopkins Hospital will take part. The goal is to see if the program is practical and …

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 uses MRI scans to look for brain differences in people with metabolic diseases compared to healthy volunteers. Researchers will track changes over time and link them to body fat and other health measures. About 126 adults will take part at Ulm University Hospital. No d…

This study aims to create a secure national database for muscle and joint (musculoskeletal) conditions by collecting routine data from community clinics and GP practices. About 22,000 adults will have their information anonymously gathered to help develop a dashboard that tracks …

This study collects small heart tissue samples from 400 adults undergoing heart procedures. Researchers will analyze these samples to find new markers that could help diagnose heart diseases earlier and better understand how the heart works in health and disease. The goal is to i…

This study watches 2000 people having hip or knee replacement surgery to see how long spinal anaesthesia lasts. The goal is to learn how the amount of anaesthetic affects timing, helping doctors decide if patients can safely go home the same day. No new treatments are tested—just…

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 uses a special eye scan called OCTA to measure the density of tiny blood vessels in the retina. Researchers will compare results from 64 people—half with cardiovascular disease and half healthy—to see if the scan can reliably detect vessel changes linked to heart probl…

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 is testing whether adding genetic and imaging information to standard risk assessments can better predict heart disease in 30,000 healthy Italian adults aged 40-80. Participants will have their risk calculated using traditional methods and then again with the added dat…

This study is testing a virtual patient navigation program for 260 English, Chinese, or Spanish speakers with breast cancer or heart disease. The goal is to see if a phone-based portal can help underserved people get the support they need, even though it won't fully replace in-pe…

This study looks at how changing the heel cushion on an ankle-foot brace (AFO) affects walking in people who use one daily. Forty adults with leg injuries or nerve problems will try four different heel wedges—tall or short, soft or firm—while walking at controlled speeds. Researc…

This study looks at how 3D computer simulation and printing can help surgeons better plan and perform bone correction surgeries. Researchers will compare the planned corrections to the actual results in 100 children and young adults with rare bone deformities. The goal is to make…

This study follows over 10,000 Moroccan adults for 10 years after a heart screening. Researchers will track heart attacks, strokes, and heart-related deaths to build a risk score tailored to the Moroccan population. The goal is to better predict and prevent heart disease in Moroc…

This study is building a biobank of blood and tissue samples from 500 patients undergoing cardiac surgery. Researchers will analyze these samples to better understand the causes and progression of heart disease. The goal is to improve future diagnosis and treatment, but no new th…

This study tracks 150 adults in cardiac rehab to see if men and women differ in how much they move during and up to 6 months after the program. Participants wear activity trackers and complete fitness tests at three visits over 10 months. The goal is to understand barriers to sta…

This study follows 4,000 children who receive orthopedic implants (like rods or screws) during standard care for bone fractures, deformities, or hip problems. Researchers will track how long the implants last, any complications, and how well they work over time. The goal is to ga…

This study looks at how often young children (ages 1 to 6) with inherited metabolic diseases develop eating disorders when they are on special diets. Researchers will use a feeding scale to measure eating problems in 200 children. The goal is to understand the link between these …

This study follows 144 adults with muscle or bone problems who are already scheduled to receive treatment with Chattanooga Intelect devices (like TENS or ultrasound). Researchers will measure pain, muscle strength, and movement range to confirm the devices are safe and work as ex…

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 aims to see if voice recordings from a smartphone can help detect heart disease. Researchers will collect voice samples and health data from up to 1 million adults using the Mayo Clinic app. No treatment is given; the goal is to gather information for future AI tools.

The SMILE study is tracking 10,000 adults aged 18 and older to see how sarcopenia (age-related muscle loss) and metabolic diseases like diabetes influence overall health and lifespan. Researchers will collect data from medical records, tests, and surveys over time. This is an obs…

This study is testing whether a smartwatch, combined with artificial intelligence and remote doctor check-ins, can help detect heart issues like atrial fibrillation and unstable angina earlier than usual. Researchers at the University of Sao Paulo will enroll 520 adults with hear…

This study is observing 900 adults in Greece who have both chronic obstructive pulmonary disease (COPD) and at least one heart condition. Researchers will track their health for two years, recording lung flare-ups, heart complications, hospital visits, and quality of life. The go…

This study follows 100 children under 18 who are already on a ketogenic diet for conditions like drug-resistant epilepsy, autism, chronic migraine, or brain tumors. Researchers want to see if the diet supports healthy growth, improves symptoms, and boosts quality of life. The die…