Noonan syndrome

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to…

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 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 Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…

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 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 vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one…

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 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 new type of ultrasound called super-resolution ultrasound (SRUS) to see if it can better tell the difference between benign (non-cancerous) and malignant (cancerous) swollen lymph nodes. About 779 adults with abnormal lymph nodes will receive an injection of ul…

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 tests two different stitch materials (PDS and Vicryl) for closing groin incisions after planned vascular surgery. About 259 adults will be randomly assigned to one stitch type or the other. The goal is to see which material leads to fewer wound problems.

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 a supplement called MyCondro can help people with knee osteoarthritis move better and feel less joint discomfort. Researchers will give two different doses to 240 adults aged 45 and older who have had knee problems for at least six months. The main goal i…

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 whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

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 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 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 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 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 natural history study aims to collect data from 1,200 people with lymphatic anomalies—rare conditions affecting the body's lymph system—over many years. Researchers will track symptoms, perform genetic testing, and gather biospecimens to better understand why these condition…

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 is collecting medical data and biological samples from people with genetic conditions affecting the heart and blood vessels, their relatives, and healthy volunteers. The goal is to better understand these diseases, improve diagnosis, and lay the groundwork for future t…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

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 aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…

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 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 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 looks at DNA methylation patterns (chemical tags on DNA) in fetuses with rare genetic diseases. Researchers will analyze DNA from amniotic fluid and tissue samples to create reference signatures. The goal is to improve diagnosis of these conditions before birth. The st…

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 aims to create a collection of blood and urine samples from 100 people with Noonan syndrome. Researchers will use these samples to study how the disease works and look for factors that predict how it will progress. The study does not test any treatment, but may help gu…

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 looks at cholesterol and blood sugar levels in 200 people with Noonan syndrome and related conditions, aged 2 to 35. Researchers want to see if these levels differ by age, gender, or genetic type. The goal is to improve long-term care by identifying who might be at hig…

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 is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

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

This study is reviewing medical records from 5,000 heart and blood vessel surgery patients to find factors that influence recovery and survival. Researchers are looking at data from 2001 to 2013 to identify what leads to complications or death after surgery. The goal is to improv…

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 is testing advanced heart MRI scans to better diagnose and predict risks for people with rare heart muscle diseases. Researchers will scan 1000 participants to see if these new imaging methods can identify conditions like Fabry disease and cardiac amyloidosis more accu…

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 is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…

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 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 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.

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 is testing whether using rapid genetic sequencing can help doctors diagnose and treat birth defects in newborns in intensive care. Researchers will enroll 2,000 babies and compare death rates, disability rates, and genetic findings. The goal is to see if personalized t…