National Heart, Lung, And Blood Institute (nhlbi)

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 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 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 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 produce a complete resp…

This study tested a new daily pill, bitopertin, in 15 adults with Diamond-Blackfan anemia (DBA) that no longer responds to steroids. DBA is a rare inherited condition causing severe anemia, often requiring frequent blood transfusions. The goal was to see if bitopertin could boost…

This early study tested a new catheter-based device to treat functional mitral regurgitation, a condition where the heart's mitral valve leaks due to damaged heart muscle. Nineteen adults with heart failure symptoms took part. The device is implanted permanently to tighten the va…

This study tested an artificial version of a missing enzyme (ACP-501) in one person with familial LCAT deficiency, a rare genetic condition that causes low good cholesterol and kidney failure. The participant received several doses of the drug and was monitored with blood tests a…

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 8 am and 2 pm) can reduce inflammation in men with mild to moderate psoriasis. Participants stayed at the clinic for 5 days, followed the eating schedule, and had blood tests to measure immune ma…

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 275 adults with a lung condition called bronchiectasis, where airways are stretched and trap mucus, leading to infections. The goal was to better describe the physical traits, lung scans, and germs in the lungs of people with no known cause. Participants gave…

This study looked at 32 people with known genetic changes that affect elastic fibers in the skin, blood vessels, and lungs. Researchers used medical tests and exams to learn how these gene changes impact overall health. The goal was to gather information, not to test a treatment.

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

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 normal hemoglobin. Researchers enrolled 307 pregnant women to donate cord blood after delivery. The stored blood may be used in…

This study followed more than 2,100 people with myelodysplastic syndromes (MDS) or related blood disorders to learn how the disease naturally progresses. Researchers collected medical data and blood or bone marrow samples over time to build a biobank for future research. The goal…

This study looked at nearly 1,000 adults with sickle cell anemia to find out how many also develop pulmonary hypertension, a condition where blood pressure in the lungs is too high. Participants had heart ultrasounds and blood tests, and were followed by phone for up to 3 years. …

This study collected DNA, tissue, and medical history from 305 people with Williams syndrome or a related heart condition called supravalvar aortic stenosis (SVAS). The goal was to create a resource for future research into why symptoms vary so much from person to person. Partici…

This study aimed to find new gene changes that cause rare lipid disorders, like unusual cholesterol or fat levels in the blood. Researchers used advanced genetic testing (genomic sequencing) on 140 people aged 2 and older, including affected individuals and their relatives. The g…

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 measured the amount of radiation people receive during a common heart scan called CCTA. Researchers collected data from 66 adults at multiple hospitals worldwide to see how radiation doses vary and how often dose-saving techniques are used. The goal was to help make th…

This study looked at how immune cells in the lungs respond to a TB protein called PPD. Seventeen adults—some with latent TB (no active disease) and some never exposed—received PPD directly into the lung during a bronchoscopy. Researchers then collected lung cells and used PET-CT …

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 what causes airway inflammation in asthma. No treatment was given; the study was purely for research to f…

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 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 using gentle suction and a pencil-like probe. The goal w…

This study looked at how fat particles called lipoproteins travel in the blood and how they relate to artery hardening. Researchers studied 759 people, including healthy volunteers and those with high cholesterol or other lipid disorders. Participants received injections of label…

This study tested whether new computer methods can create higher resolution images from regular CT scans. 148 adults who already needed a CT scan took part. Researchers compared the standard images with new, sharper versions made from the same scan data to see if they provide mor…

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 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 causes of these painf…

This study looked at 70 adults with sickle cell disease to find the most accurate way to measure kidney function. Participants gave blood and urine samples and had special imaging tests. The goal was to see if a newer blood test (cystatin C) is better than the standard one (creat…

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 over two months. The goal was to learn more about the body's defens…

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…

This study looked at people with early-onset Parkinson's disease (starting before age 50) and their healthy 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 why some people ge…

This study looked at 73 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 became life-threatening. The goal was to gather information to help develop future treatm…

This study followed 171 adults with severe asthma, mild-to-moderate asthma, or no asthma over several years. Researchers collected health data, lung tests, and samples to understand how severe asthma progresses and what complications arise. The goal is to find new ways to treat p…

This study aimed to understand how blood vessel stiffness in people with Williams Syndrome and related conditions affects organs like the heart, brain, and kidneys over time. Researchers followed 159 participants, including healthy volunteers, for up to 10 years with yearly check…