National Heart, Lung, And Blood Institute (nhlbi)

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (like a parent or sibling) for people with severe aplastic anemia, a related bone marrow disorder, or a rare blood disease called PNH. Participants receive chemotherapy and a one-time …

This study is for people with sickle cell disease or beta-thalassemia whose first stem cell transplant didn't fully work and their disease came back. It offers a second transplant to try to get more donor cells into the bone marrow. Participants will receive drugs to prepare thei…

This study tests whether starting two oral drugs (cyclosporine and eltrombopag) early, before the standard treatment, is safe and practical for people with severe aplastic anemia. Participants take the pills for 6 months and may later receive standard therapy. The goal is to see …

This study tests a drug called ruxolitinib in adults with immune bone marrow failure, where the immune system attacks the bone marrow. Participants take the drug twice daily for up to 6 months to see if it can control the disease and improve blood counts. The goal is to find a be…

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

This study tests baricitinib, an FDA-approved drug for other conditions, in 12 adults with Kohlmeier-Degos disease affecting the brain and spine. The goal is to see if the drug can stop new lesions from forming and keep existing ones stable. Participants take baricitinib daily fo…

This study tests whether nicotinamide riboside (a form of vitamin B3) can reduce harmful inflammation in women with mild-to-moderate lupus. About 78 women will receive either the supplement or a placebo for 12 weeks. Researchers will measure changes in immune cell activity and bl…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia and other bone marrow failure diseases. The goal is to lower the risk of serious complications, like graft-versus-host disease, by giving fewer donor white blood cells. Participants rece…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or a related bone marrow disease called MDS. It combines stem cells from a family member with specially grown cord blood cells from an unrelated donor to help the new cells grow faster a…

This study tests whether a low dose of the hormone drug danazol can safely slow the rate at which telomeres shorten in people with telomere diseases. Telomeres are protective caps on our DNA that get shorter as we age; people with certain gene changes have very short telomeres, w…

This study looks at whether taking omega-7 oil (found in fish and macadamia nuts) can improve cholesterol levels and help with appetite control. Researchers will give healthy adults omega-7 supplements for 24 weeks and track changes in their blood fats and eating habits. The goal…

This study tests whether a nebulizer or a standard inhaler (MDI) delivers albuterol better to improve lung function in women with LAM, a rare progressive lung disease. About 100 women with LAM and breathing problems will stay overnight for 3 days to try each method. The goal is t…

This early-phase study tests the safety of a drug called fostamatinib in 25 adults with stable sickle cell disease. Participants take the pill twice daily for up to 6 weeks, with regular checkups to monitor side effects. The goal is to see if the drug is safe enough for larger st…

This study tests a mobile health program for couples to help people with sleep apnea stick with their CPAP machine. About 360 newly diagnosed patients and their partners will get tips and resources on their phones for 6 months. The goal is to see if this support improves nightly …

This study follows 155 adults with CADASIL, a genetic condition that narrows small blood vessels and can lead to strokes and dementia, over 9 years. Researchers will track changes in blood flow, brain structure, and thinking abilities through regular tests and samples. The goal i…

This study collects blood stem cells from 500 healthy volunteers to develop better ways to process them for transplants. The goal is to remove the white blood cells that can cause a serious complication called graft-versus-host disease while keeping the stem cells needed for reco…

This study aims to develop better MRI methods for imaging the heart and brain, and to understand how heart disease relates to other organs. Researchers will also check if gadolinium, a contrast dye used in some MRIs, stays in the body long-term. Healthy volunteers and people with…

This early-stage study tests a new drug called ZMA001 in 96 healthy adults aged 18 to 60. The goal is to check if it is safe and how the body processes it before testing it in people with a serious lung condition called pulmonary arterial hypertension (PAH). Participants receive …

This study collects fluid and tissue samples from the lungs and nose of healthy volunteers and people with a history of lung infections. Researchers will compare the samples to find differences that might explain why some people are more prone to infections. Up to 550 adults aged…

This study aims to learn more about rare genetic disorders that affect fat levels in the blood, such as too much or too little cholesterol or triglycerides. Researchers will follow 1,500 people aged 10 and older for up to 20 years to see how these conditions change and respond to…

This study follows 1,000 people aged 2 and older with bone marrow failure diseases (like severe aplastic anemia) for up to 20 years. Researchers want to understand how these diseases and their treatments affect the body over time. Participants have regular check-ups, blood tests,…

This study looks at how the heart, brain, kidneys, liver, and lungs change in people with sickle cell disease after they receive treatments meant to cure it, such as a bone marrow transplant or gene therapy. Researchers will compare these changes to those in people who continue w…

This study looks at how stress from your neighborhood might affect heart health in women. Researchers will track 250 healthy women in Washington, DC for about 2 weeks using a wrist device, GPS, and daily phone surveys. The goal is to understand how things like crime or poverty in…

This study collects blood and tissue samples from people with rare genetic heart or blood vessel diseases, as well as from healthy volunteers. The goal is to find the genetic causes and understand how these diseases work. No treatment is given, but the samples help researchers de…

This study follows people with sickle cell disease to learn more about how the condition changes over time and what health problems may arise. It does not offer any experimental treatments. Participants must be at least 2 years old and receive their regular medical care outside t…

This study looks at how small differences in the PKLR gene might make sickle cell disease symptoms better or worse for different people. Researchers will collect blood samples from 800 adults of African descent, both with and without sickle cell disease, to measure certain protei…

This study provides yearly follow-up care for people who received a donor stem cell transplant at least three years ago. Researchers monitor for late side effects, disease return, and overall health through exams, blood tests, and imaging. The goal is to improve long-term care an…

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 produce clear pictures of the heart, especially for people with metal implants or during procedures using metal tools. About 2,950 healthy adults and p…

This study collects blood samples from 1000 healthy adults aged 18 and older to help researchers study blood vessel and immune system diseases. Participants give about 10 tablespoons of blood per visit and can return for up to 10 years. The blood is used to develop lab tests and …

This study collects blood, bone marrow, and cheek cell samples from healthy volunteers ages 8 and older. Researchers use these samples to study blood cell formation, viral blood diseases, immune system problems, and genetic risks for conditions like aplastic anemia. The goal is t…

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 had or are waiting for a lung transplant. Participants will have regular check-ups, blood tests, lung function …

This study looks at people with lipid disorders—conditions where fats like cholesterol are too high or too low in the blood. Researchers want to learn how these fats cause diseases like hardening of the arteries. Up to 2,000 adults and children (ages 2 and up) will have their blo…

This study watches how certain blood cancer drugs (BTK inhibitors or venetoclax) affect the heart's rhythm. About 135 adults with cancers like CLL or mantle cell lymphoma will have heart tests before and during treatment. The goal is to spot dangerous heart rhythms early and unde…

This study aims to create and test new MRI methods for imaging the heart and blood vessels. It will involve up to 3,400 adults, including people with heart conditions and healthy volunteers. Participants will undergo MRI scans with possible contrast dye, stress tests, and follow-…

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 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and unrelated healthy v…

This study is for women with lymphangioleiomyomatosis (LAM), a rare lung disease that mainly affects young women. Researchers want to understand how the disease destroys lung tissue at the cellular and genetic level. Up to 2000 participants will help scientists identify the prote…

This study follows up to 1,000 adults with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), Waldenstrom macroglobulinemia, or related conditions to learn how these diseases progress over time. Participants receive no treatment as part of the study; instead, t…

This study measures how long red blood cells survive in people with sickle cell disease and other inherited blood disorders. Researchers will label participants' own red blood cells with biotin (a vitamin) and track them over time. The goal is to compare cell survival between tho…

This early-phase study looks at how the drug pirtobrutinib changes immune cells in people with untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). About 30 adults will take pirtobrutinib daily and have blood and lymph node tests to track immune syste…

This study looks for better ways to find lung inflammation early in people who have received a stem cell transplant from a donor. Researchers will use special CT scans, lung washes, blood tests, and breathing tests before and for two years after the transplant. The goal is to see…

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 new techniques are accurate and can help predict future heart problems. About 5,000 adults who need a heart CT scan will participate. Th…

This study looks at how well vaccines work in adults with certain blood cancers like chronic lymphocytic leukemia (CLL) or non-Hodgkin lymphomas. Participants will receive one or more vaccines (e.g., COVID-19, hepatitis B, shingles) and give blood samples to measure their immune …

This study aims to understand the heart health of adults in Black church communities in Washington D.C. Researchers will measure things like blood pressure, cholesterol, and weight, and ask about lifestyle habits. Participants will also wear activity monitors for a month. The inf…

This study tests whether a synbiotic supplement (a powder containing a sugar from human milk plus probiotics) can increase helpful gut bacteria in healthy adults. One hundred participants will take the supplement twice daily for 8 weeks. Researchers will measure changes in gut ba…

This study aims to better understand heart and blood vessel diseases by observing people who have them or are at risk. Researchers will collect medical information, images, and samples from up to 5,000 participants, including healthy volunteers and family members. The goal is to …

This study aims to learn more about diseases of the heart and blood vessels by collecting medical information and samples from up to 1,000 people. Participants include those with known or suspected genetic vascular conditions, their relatives, and healthy volunteers aged 2 and ol…

This study is testing an experimental drug called Fx-5A in healthy volunteers to see if it is safe and how the body processes it. The goal is to gather information that could help treat heart disease and inflammation in the future. Participants receive a single infusion of the dr…

This study looks at how platelets—cells that help blood clot—work in healthy adults. Researchers will follow 400 volunteers for up to 10 years, taking blood samples and asking about diet, exercise, and health habits. The goal is to understand normal platelet function and how it c…

This study is testing a simple blood test that looks for tiny pieces of DNA from the donated organ in the patient's blood. Higher levels of this donor DNA may signal early rejection, allowing doctors to treat it before damage becomes permanent. The study involves nearly 1,000 hea…

This study aims to see if new CT scan methods that use less radiation can create pictures just as clear as traditional CT scans. Researchers will enroll 215 adults who already need a CT scan of their chest, abdomen, and pelvis. During their scheduled scan, one extra low-radiation…

This study looks at how genes play a role in lung diseases like cystic fibrosis, asthma, and pulmonary fibrosis. Researchers will analyze DNA from up to 3,500 people, including healthy volunteers, to find genetic differences linked to lung problems. A small optional part of the s…

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 will track whether participants develop blood cancers, heart disease, or other conditions. Participants give blood and u…

This study collects blood, bone marrow, urine, and tissue samples from people with cancer or blood disorders, as well as from their healthy family members who are stem cell donors. The samples are used in lab research to better understand diseases, improve treatments, and find ne…

This study follows 900 people with bronchiectasis (damaged and infected airways) to learn why they get repeated lung infections. Researchers will collect blood, spit, and genetic samples from patients and their family members. The goal is to find inherited or immune system proble…

This study looks at genetic changes in chronic lymphocytic leukemia (CLL) patients during treatment with the drug venetoclax. Researchers want to understand how the drug affects cancer cells and the immune system. The study involves 75 adults with relapsed or refractory CLL who w…

This study aims to uncover 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 up to 100 patients and their relatives to analyze DNA and other factors. The goal …

This study looks at why people with tuberous sclerosis, a rare genetic condition, develop non-cancerous skin tumors. Researchers will examine skin samples from up to 400 adults to find genetic and protein changes. The goal is to learn more about the disease, not to test a treatme…