National Heart, Lung, And Blood Institute (nhlbi)

This study tested a drug called bitopertin in 15 adults with Diamond-Blackfan anemia (DBA), a rare inherited condition that causes severe anemia. Participants took the pill daily for 8 months, with doses possibly increasing over time. The goal was to see if the drug could raise h…

This study tested an artificial version of the LCAT enzyme (ACP-501) in one person with familial LCAT deficiency, a rare genetic condition that leads to low 'good' cholesterol and kidney failure. The participant received several infusions of the drug over weeks, with regular bloo…

This early-phase trial tested whether giving patients their own natural killer (NK) cells, boosted by the drug bortezomib, is safe for treating advanced cancers like leukemia, lung, and pancreatic cancer. Thirty-five adults whose cancers had not responded to standard treatments r…

This study tested whether low doses of the hormone drug danazol can slow the shortening of telomeres—the protective caps on DNA—in people with telomere diseases. These conditions can cause bone marrow failure, lung scarring, and liver damage. Eighteen participants took danazol fo…

This early study tested a new, less invasive procedure to treat functional mitral regurgitation, a condition where a leaky heart valve causes heart failure symptoms like shortness of breath and fatigue. Nineteen adults with this condition received a permanently implanted device t…

This study tested the drug eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Eltrombopag is a pill that mimics a natural protein to stimulate blood cell production. Researchers measured whether it safely imp…

This study tested a drug called ruxolitinib in one adult with Kohlmeier-Degos disease affecting the brain and spinal cord. The goal was to see if the drug could stop or slow the growth of lesions in the central nervous system. The participant took the drug by mouth twice daily fo…

This study tested a new tool called TELLTALE designed to safely cut a heart tissue flap during a valve replacement procedure (TAVR). In some cases, this flap can block blood flow to the heart, which is often fatal. The trial involved 90 adults at high risk of this complication. R…

This phase II study tested a combination of three drugs—ibrutinib, fludarabine, and pembrolizumab—in 15 people with high-risk or relapsed/refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The goal was to see if the combo could produce a complete r…

This study looks at people with a rare inherited condition that causes extremely high cholesterol and early heart disease. Researchers used special tests to measure artery hardening before it becomes life-threatening. The goal was to gather information to help develop future trea…

This study looked at how stretchy the skin and blood vessels are in people with Williams syndrome, a genetic condition that can cause heart problems. Researchers measured skin and pulse speed in 43 participants aged 5 to 70. The goal was to learn how these tissues change over tim…

This study aimed to understand how the immune system responds to seasonal and H1N1 flu vaccines in healthy adults. Researchers collected blood and nasal samples from 203 NIH employees before and after vaccination. The goal was to analyze immune responses to improve future vaccine…

This study aimed to find new gene changes that cause rare lipid disorders. Researchers used advanced genetic testing to look at many genes at once in 140 people and their relatives. The goal was to discover causes that older methods missed.

This study looked at what happens in the blood of people with sickle cell disease during painful attacks. Researchers compared blood samples taken during a pain crisis to samples taken when participants were feeling well. The goal was to learn more about the role of inflammation …

This completed study looked at how inflammation might connect heart disease and diabetes. Researchers used PET scans and blood tests to measure inflammation in 150 adults, including healthy volunteers. The goal was to better understand these links, not to test a new treatment.

This study measured how common pulmonary hypertension (high blood pressure in the lungs) is in adults with sickle cell anemia. Nearly 1,000 participants had heart ultrasounds and blood tests, then were contacted by phone for up to 3 years to track their health. The goal was to un…

This study looked at 20 people with CADASIL, a rare genetic disease that damages brain arteries and causes migraines, strokes, and dementia. Researchers used blood tests, skin biopsies, eye exams, and brain scans to learn more about how the disease works. The goal was to better u…

This study looked at whether new computer methods can create higher-resolution images from a standard CT scan. Researchers compared the quality of regular CT images with enhanced versions from the same scan. 148 adults scheduled for a CT scan participated. The goal was to see if …

This completed study looked at 670 African American adults to understand how genetics, lifestyle, and social factors like stress and neighborhood affect heart disease risk. Researchers collected blood samples, medical tests, and surveys to find links between ancestry-related DNA …

This study tests whether special low-energy MRI settings can make it safe to use guidewires during heart catheterization. Guidewires help position catheters but can heat up in standard MRI. Researchers will enroll 22 adults who need a right heart catheterization to see if this ap…

This study looked at 275 adults with a lung condition called bronchiectasis, where airways are stretched and trap mucus, leading to infections. The goal was to learn more about the physical traits, lung scans, and germs in the lungs of people with the unexplained form of the dise…

This study looked at how blood vessel stiffness affects organs like the heart, brain, kidneys, and gut in people with Williams Syndrome (WS) or supravalvular aortic stenosis (SVAS). Researchers compared 159 participants (ages 3–85) with these conditions to healthy volunteers over…

This completed study looked at 386 adults with psoriasis to understand how skin inflammation might be linked to heart and blood vessel diseases. Participants had up to seven visits over four years, including blood tests, heart scans, and optional skin biopsies. The goal was to se…

This study followed 171 adults with severe asthma, milder asthma, or no asthma over several years. Researchers collected health data, lung tests, and samples to understand how severe asthma progresses and differs from other forms. The goal is to find new ways to treat people whos…

This completed study looked at people with genetic conditions that affect how the lungs clear mucus, such as cystic fibrosis and primary ciliary dyskinesia. Researchers examined 87 participants, including healthy volunteers, to understand why these patients get repeated lung infe…

This study looked at 70 adults with sickle cell disease to see which lab test—cystatin C or creatinine—better measures kidney function. Participants gave blood and urine samples and had a special dye test to check how well their kidneys work. The goal is to help doctors catch kid…

This study examined blood samples from 119 adults with sickle cell disease, sickle cell trait, or no condition to understand why some develop dangerous blood clots. Participants gave blood and had health check-ins over two years. The goal was to find biomarkers that could lead to…

This study collected fluid and cells from the lungs of 574 people with asthma and healthy volunteers using a thin tube called a bronchoscope. The goal was to learn more about airway inflammation and what causes it. The findings may help identify new targets for future asthma trea…

This study looked at how fat-carrying particles called lipoproteins work in healthy people and those with high cholesterol or other lipid disorders. Researchers injected tiny, safe amounts of radioactive lipoproteins to track them in the body. The goal was to better understand ho…

This study collected DNA, tissue, and medical history from 305 people with Williams syndrome or supravalvar aortic stenosis (SVAS), as well as their family members and healthy volunteers. The goal was to create a resource for future research to understand why symptoms vary so muc…

This study looked at how immune cells in the lungs respond to tuberculosis (TB) proteins in 17 adults—some with latent TB (no symptoms) and some never exposed. Researchers used a thin tube to deliver the protein directly into the lung and collected cells to measure immune reactio…

This study looked at lipoproteins, which carry fats like cholesterol in the blood. Over 1,000 patients and healthy volunteers had their blood tested while on a balanced diet. The goal was to train medical staff to better diagnose and treat fat-related disorders. Participants did …

This pilot study tested whether time-restricted feeding (eating all daily calories between 8am and 2pm, then fasting for 18 hours) could reduce inflammation in men with mild to moderate psoriasis. 27 participants (including healthy volunteers) stayed at the clinic for 4.5 days, w…

This completed study looked at 31 people with early-onset Parkinson's disease (starting before age 50) and their family members to find genetic causes. Researchers collected blood and skin samples to study DNA and create cell lines. The goal was to better understand which gene mu…

This completed study looked at how much radiation people receive during heart CT scans (CCTA) in everyday hospital practice. Researchers enrolled 66 adults who needed a heart CT and recorded the radiation dose used, along with any dose-saving techniques. The goal was to understan…

This study looked at 33 women with LAM, a rare lung disease, to find blood and urine markers that show how the drug sirolimus is working. Participants took sirolimus daily for 9 months and gave samples at the start, 3 months, and 9 months. The goal was to identify markers that co…

This completed study enrolled over 2,100 people with or suspected of having myelodysplastic syndromes (MDS), a group of blood disorders. Researchers collected medical data, blood, and tissue samples over time to track how the disease develops and progresses. The goal was to creat…

This study collected umbilical cord blood from over 300 newborns with sickle cell disease, sickle cell trait, or no disease to find the best ways to process and store it. The blood is rich in stem cells that might one day be used in treatments like gene therapy or transplants. Th…

This study looked at data from over 1,600 African Americans aged 30-55 with severe high blood pressure. Researchers examined genetic, behavioral, and social factors to find out why this group is more affected by hypertension. No new participants were enrolled; the goal was to lea…

This completed study looked at 32 people with known genetic changes in connective tissue genes to better understand how these changes affect overall health. Participants underwent a range of medical tests and exams over 3-5 days. The goal was to gather information, not to test a …