National Heart, Lung, And Blood Institute (nhlbi)

This clinical trial tests a partial stem cell transplant for people with severe sickle cell disease or beta-thalassemia who need regular blood transfusions. The transplant uses a lower-intensity conditioning regimen with alemtuzumab, low-dose radiation, and abatacept to help dono…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This early-stage trial is testing whether a skin gel made from curcumin (a compound in turmeric) can be safely absorbed by adults with stable sickle cell disease. Researchers will apply the gel to the forearms twice a week for six weeks and monitor for side effects. The goal is t…

This study tests whether a supplement called nicotinamide riboside (NR) can reduce inflammation and improve immune cell function in women with mild-to-moderate lupus. About 78 participants will receive either NR or a placebo for 12 weeks. Researchers will measure changes in immun…

This study tests whether mobile health technology, like smartphone apps and activity trackers, can help overweight or obese African American women increase their physical activity. Participants will wear a wrist device to track steps and sleep, use a study app, and may receive co…

This study tests whether baricitinib, a drug already approved for arthritis, can help people with Kohlmeier-Degos (KD) disease that affects the brain and spine. KD causes inflammation and blood clots that damage small blood vessels, and there is currently no treatment. The trial …

This study tests whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study looks at whether taking omega-7 oil (palmitoleic acid) can improve cholesterol levels and help control appetite in healthy adults. About 110 participants will take omega-7 capsules daily for 24 weeks and have their blood and health checked. The goal is to see if this n…

This phase 1 trial tests the safety of a new drug called ZMA001 in 96 healthy adults. ZMA001 is a monoclonal antibody designed to reduce lung damage in pulmonary arterial hypertension (PAH), a serious condition. Participants receive a single dose of the drug or placebo and are mo…

This early-stage trial is testing the safety of fostamatinib, a drug already used for arthritis, in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with close monitoring for side effects. The goal is to see if the drug is safe …

This study tests whether starting two oral medications (cyclosporine and eltrombopag) earlier than usual is safe and feasible for people with severe aplastic anemia, a rare condition where the immune system attacks bone marrow. Participants aged 3 and older will take these pills …

This study offers a second stem cell transplant to people with sickle cell disease or beta-thalassemia whose first transplant is losing its effect. Participants receive donor stem cells along with drugs to help their body accept them. The goal is to restore donor cell levels and …

This study is testing new, lower-radiation CT scan methods for taking pictures of the heart and blood vessels. Researchers want to see if these scans are accurate at finding blocked arteries and predicting future heart problems like heart attacks. About 5,000 adults who need a he…

This study tests whether a nebulizer or a standard inhaler delivers albuterol better to improve breathing in women with LAM, a rare lung disease. About 100 women with reduced lung function will stay overnight for 3 days to try each method. The goal is to see which approach opens …

This study tests a mobile app called OurSleepKit that coaches couples to work together on CPAP therapy for obstructive sleep apnea. 360 newly diagnosed patients and their partners will use the app for 6 months. Researchers will measure how many hours per night the patient uses CP…

This study watches people with blood cancers like CLL who take BTK inhibitors or venetoclax to see how these drugs affect heart rhythm. Researchers will use EKGs, stress tests, and heart monitors to track abnormal rhythms and sudden death risk. The goal is to better understand an…

This study tests whether new CT imaging methods that use less radiation can produce pictures as clear as standard CT scans. About 215 healthy adults scheduled for a chest, abdomen, and pelvis CT will get one extra low-radiation scan. Researchers will compare image quality between…

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 is testing a new type of MRI scanner that uses less energy than standard machines. The goal is to see if it can still take clear pictures of the heart, which could help people with metal devices in their body. Healthy volunteers and people with heart disease will have …

This study follows up to 1,000 adults with certain slow-growing blood cancers (CLL, SLL, LPL, WM) to track how the diseases change over time. Participants have regular check-ups and blood draws every 6 to 24 months, and may also have optional biopsies or scans for research. No tr…

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 collects blood stem cells from healthy volunteers after they receive a growth factor injection. Researchers aim to develop better methods to remove the white blood cells that cause graft-versus-host disease, a serious complication of bone marrow transplants. Up to 500 …

This study aims to learn more about lipid disorders—conditions that affect fats like cholesterol in the blood. Researchers will observe up to 2,000 people with abnormal cholesterol levels to better understand how these disorders develop and how to diagnose them. Participants will…

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine up to 400 adults, perform skin exams, and take small tissue samples (biopsies) from tumors. The goal is to identify the cells and genetic …

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

This study is testing a non-invasive blood test that looks for donor DNA in the blood of heart and lung transplant patients. Higher levels of this DNA may signal early rejection before permanent damage occurs. About 991 participants aged 14 and older will provide blood samples ov…

This study follows up to 3,500 people with sickle cell disease to learn more about how the condition progresses and what complications arise. Participants receive their usual medical care outside the hospital and visit the clinic for check-ups and research tests. No experimental …

This study tests whether a new drug called Fx-5A is safe in healthy adults. Researchers will give a single infusion of the drug and monitor participants for side effects. The goal is to see if Fx-5A can be used in future studies for heart disease and inflammation.

This study aims to collect blood and tissue samples from up to 10,000 people with rare genetic vascular or cardiovascular diseases, as well as their healthy relatives. Researchers will use these samples to identify genetic causes and understand how these diseases work. The goal i…

This study follows 155 adults with CADASIL, a genetic condition that narrows small blood vessels and raises the risk of strokes and dementia, over nine years. Researchers will use brain scans, blood tests, and other exams to track how the disease changes blood vessels and thinkin…

This study follows people with acquired or inherited bone marrow failure for up to 20 years to learn how the diseases and treatments affect the body over time. Participants, aged 2 and older, will have regular check-ups and tests every 1 to 3 years. The goal is to better understa…

This study aims to understand how lymphangioleiomyomatosis (LAM) damages the lungs at the cellular and genetic level. Researchers will follow up to 2,000 participants, including women with LAM and healthy volunteers, using advanced imaging and lab tests. The goal is to find the p…

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 collects blood, bone marrow, and cheek cell samples from healthy volunteers to help researchers understand blood diseases like aplastic anemia. Participants must be 8 or older (18 for bone marrow). The samples are used for lab research only, not to treat any condition.…

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 follows 900 people with bronchiectasis—a condition where airways are damaged and prone to infection—to learn why they get sick repeatedly. Researchers will collect medical history, lung function tests, and blood, urine, and sputum samples from patients and their family…

This study follows 306 adults with certain DNA changes in their blood cells (called CHIP or CCUS) to see how these changes affect health over time. Researchers want to learn if these changes increase the risk of blood cancers or heart problems. Participants will have yearly check…

This study aims to find the genetic causes of severe combined immunodeficiency (SCID), a rare condition where the immune system doesn't work properly. Researchers will collect blood samples from 100 patients and their relatives to analyze DNA and other factors. The goal is to bet…

This study collects blood samples from 1,000 healthy adults aged 18 and older to help researchers understand blood vessel and immune system diseases. Participants give about 10 tablespoons of blood at the NIH Clinical Center and may return for up to 10 years. The samples will be …

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will examine DNA from up to 3,500 people with and without lung disease to find genetic differences. The goal is to better understand what causes these cond…

This study tests whether a daily supplement called Cardiosyn can increase helpful gut bacteria in healthy adults who eat a typical Western diet. The supplement contains a natural sugar found in human milk along with probiotics. One hundred participants will take the powder for ab…

This study aims to understand why some people who get a lung transplant develop rejection while others do not. Researchers will follow 100 adults aged 18 to 75 who have progressive lung disease or have had or may get a lung transplant. Participants will have regular check-ups, bl…

This study looks at how differences in the PKLR gene might change sickle cell disease symptoms. Researchers will collect blood samples from 800 adults of African descent, with and without sickle cell disease, to measure certain proteins and energy molecules in red blood cells. Th…

This study works with Black churches in Washington, D.C. to understand heart health risks like obesity, high blood pressure, and diabetes. About 200 adults will get a health exam, wear an activity monitor for a month, and answer health questions. The results will help design a fu…

This study looks at how stress from your neighborhood environment might affect heart health in women. Researchers will track 250 healthy women in Washington, DC, using smartphone surveys and wearable devices to measure stress and exercise. They will also use brain scans to see ho…

This early-phase study is testing pirtobrutinib, a drug already approved for later-stage CLL/SLL, in 30 people who have not yet received any treatment. The main goal is to see how the drug changes immune cells in lymph nodes and blood, not to measure cure or symptom relief. Parti…

This study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study gathers fluid and tissue from the lungs and nose of healthy volunteers and people with a history of lung infections. Researchers will compare samples to find differences in immune function that might explain why some people are more prone to infections. Participants un…

This study looks at adults with sickle cell disease to see how their heart, brain, kidneys, liver, and lungs change after treatments meant to cure the disease. Researchers will compare people getting curative therapy (like a bone marrow transplant or gene therapy) with those who …

This study aims to develop and test new MRI methods for imaging the heart and blood vessels to improve diagnosis and management of heart disease. It will enroll up to 3,400 adults, including both patients with heart conditions and healthy volunteers. Participants will undergo MRI…

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 aims to learn how platelets—blood cells that help with clotting and healing—work in healthy people over time. Researchers will collect blood samples and health information from 400 volunteers aged 18 and older every 6 to 7 months for up to 10 years. The goal is to unde…

This natural history study tracks people aged 10 and older with rare genetic lipid disorders over up to 20 years. Researchers will monitor changes in cholesterol and fat levels, and how these conditions progress. The goal is to better understand these diseases and develop new dia…

This study tracks genetic changes in chronic lymphocytic leukemia (CLL) cells during treatment with the drug venetoclax. Researchers aim to understand how the cancer evolves as the drug is started. 75 adults with relapsed or refractory CLL will receive venetoclax and undergo regu…

This study aims to measure how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will take a blood sample, label the red cells with biotin (a vitamin), and infuse them back into the participant. Over up …

This study looks at how well vaccines for flu, COVID-19, shingles, hepatitis B, and pneumonia work in people with certain blood cancers like chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphomas. Researchers will measure immune responses through blood samples before and af…