National Heart, Lung, And Blood Institute (nhlbi)

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 new lesions from forming or shrink existing ones. The participant took the drug twice daily for up to 26 weeks and…

This 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 combination could achieve a complete resp…

This study tested an artificial enzyme called ACP-501 in one person with familial LCAT deficiency, a rare genetic condition that causes low good cholesterol and kidney failure. The participant received multiple infusions of the enzyme over weeks. Researchers measured whether the …

This study tested a new, less invasive way to fix a leaky mitral valve in people with heart failure. The procedure uses a special device threaded through a blood vessel to tighten the valve like a purse-string, without open-heart surgery. Nineteen adults with functional mitral re…

This study tested a new drug called bitopertin in 15 adults with Diamond-Blackfan anemia (DBA), a rare inherited condition that causes severe anemia. Participants took a daily pill for 8 months, starting at a low dose that could be increased. The goal was to see if the drug could…

This study tested a daily pill called eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The goal was to see if the drug could safely increase platelet and red blood cell counts and reduce the need for transf…

This study tested a new tool called TELLTALE designed to make heart valve replacement safer. In 90 adults at high risk of a serious complication where a heart flap blocks blood flow, doctors used the device to cut the flap during the procedure. The goal was to see if the tool wor…

This early-phase study tested whether giving patients their own specially grown natural killer (NK) cells, combined with the drug bortezomib, is safe for people with advanced solid tumors or leukemia that no longer respond to standard treatments. 35 adults received increasing dos…

This 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 5 days, following…

This study looked at how psoriasis, a skin condition caused by inflammation, might be linked to heart disease and other metabolic problems. Researchers followed 386 adults with psoriasis for up to 4 years, using blood tests, heart scans, and imaging to measure inflammation in blo…

This study looked at 31 people with early-onset Parkinson's disease (starting before age 50) and their healthy family members to find genetic mutations that cause the disease. Researchers collected blood and skin samples to grow cells in the lab and study how these genetic change…

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 find the best ways to collect, process, and store umbilical cord blood from newborns with sickle cell disease, sickle cell trait, or no disease. Cord blood is rich in stem cells that might one day be used for treatments like stem cell transplants or gene thera…

This study looked at 32 people with specific gene changes that affect elastic fibers in the body, which are important for skin, blood vessels, and lungs. Participants came to the NIH for 3-5 days of tests like lung function, heart stress tests, and imaging to see how their genes …

This study followed 171 adults with severe asthma, milder asthma, or no asthma over several years. Researchers collected medical history, lung function tests, and samples to understand how severe asthma progresses and what complications arise. The goal is to find new ways to trea…

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 into why symptoms vary so much from pe…

This completed study looked at how genes, lifestyle, and social factors like stress and neighborhood affect heart disease risk in 670 African American adults. Participants gave blood and urine samples, had heart scans, and answered surveys. The goal was to build a resource to bet…

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

This study looked at 275 adults with a chronic cough and a lung condition called bronchiectasis, where airways are stretched and trap mucus. The goal was to better describe the physical features, lung scans, and bacteria in the lungs of people with unexplained bronchiectasis. Par…

This study collected fluid and cells from the lungs of 574 people with and without asthma using a thin tube called a bronchoscope. The goal was to learn more about what causes airway inflammation in asthma. Findings may help identify new targets for future treatments.

This study tested whether new computer methods can make standard CT scan images sharper and more detailed. 148 adults who already needed a CT scan took part. Researchers compared the new high-resolution images to the usual ones to see if they were better.

This study collected information from 66 adults who had a heart CT scan to measure the amount of radiation used in everyday medical practice. The goal was to see how radiation doses vary between different hospitals and CT machines, and how often dose-saving techniques are used. T…

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 followed more than 2,100 people with myelodysplastic syndromes (MDS) or related blood disorders to learn how the disease develops and changes over time. Researchers collected medical information and blood and tissue samples to create a biobank for future studies. The g…

This study looked at nearly 1,000 adults with sickle cell anemia to find out how many also develop pulmonary hypertension (high blood pressure in the lungs). Participants had heart ultrasounds and blood tests, then were followed by phone for up to 3 years. The goal was to learn h…

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 looked at what happens in the blood during painful attacks in people with sickle cell disease. 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 causes of pain and co…

This study aimed to find new gene changes that cause rare lipid disorders. Researchers used advanced genomic sequencing to look at the DNA of 140 people with unusual lipid problems and their family members. The goal was to discover genetic causes that older methods missed.

This study looked at how fat particles called lipoproteins travel in the blood. Researchers wanted to better understand normal and abnormal fat metabolism and its link to hardening of the arteries. About 759 healthy volunteers and people with cholesterol disorders took part. Part…

This study looked at how immune cells in the lungs respond to a TB protein called PPD. Seventeen adults—some with latent TB and some without—received PPD directly into their lungs during a bronchoscopy. Researchers then collected lung cells and used scans to measure immune activi…

This study looked at how stiff blood vessels in people with Williams Syndrome or supravalvular aortic stenosis (SVAS) affect organs like the heart, brain, kidneys, and gut. Researchers followed 159 participants (ages 3-85) for up to 10 years using imaging, blood tests, and cognit…

This study aimed to find markers in blood and urine that could help doctors choose the right dose of sirolimus for women with LAM, a rare lung disease. Thirty-three women who were starting sirolimus gave samples before and after taking the drug for 3 and 9 months. The goal was to…

This study looked at 20 people with CADASIL, a rare genetic disease that damages arteries in the brain and can cause migraines, strokes, memory loss, and dementia. The goal was to learn more about how the disease works by doing detailed exams, brain scans, and lab tests over two …

This study looked at lipoproteins, which carry fats like cholesterol in the blood. Researchers measured these levels in over 1,000 patients and healthy volunteers to train medical staff. The goal was to improve skills for diagnosing and treating genetic fat disorders. Participant…

This study looked at whether inflammation is linked to heart disease and diabetes. Researchers compared 150 adults with these conditions to healthy volunteers using blood tests, heart scans, and tissue samples. The goal was to better understand the relationship, not to test a new…

This study looked at people with diseases that make it hard to clear mucus from the lungs, like cystic fibrosis and primary ciliary dyskinesia. Researchers did tests and genetic analysis to better understand why these patients get repeated lung infections and to find the genes re…

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