Featured Studies

Severe aplastic anemia is a rare and serious blood disorder. Patients with severe aplastic anemia commonly receive Cyclosporine to help improve blood counts. When cyclosporine treatment stops, the disease may return in one in three people. Researchers at the National Institutes of Health (NIH) Clinical Center are studying if sirolimus, an immunosuppressant, can help prevent a relapse in patients diagnosed with severe aplastic anemia following cyclosporine treatment.

Researchers at the National Institutes of Health (NIH) seek patients with Williams Syndrome (WS) or Supravalvular Aortic Stenosis (SVAS) to join a research study. Researchers want to see how blood vessel elasticity and stiffness affect the heart, gut, kidneys, and the brain in people with Williams Syndrome.

Doctors at the National Institutes of Health (NIH) are enrolling people with LAM into a research study. LAM is a rare progressive lung disease that usually strikes women during their childbearing years, between the onset of puberty and menopause. One possible treatment to improve breathing in patients with LAM is inhaled albuterol. Albuterol can be given using a hand held inhaler or with a nebulizer. The study wants to compare these methods to see which method improves lung function in patients with LAM.

Researchers at the National Institutes of Health (NIH) are testing NX-2127, an oral drug that degrades a protein in cancer cells called Bruton's tyrosine kinase (BTK), instead of inhibiting or blocking it like other drugs. NX-2127 may also stimulate your immune T-cells to attack the cancer.

Heart disease is the leading cause of death and disability in the United States. Omega-7 fatty acids are found in the oil extracted from certain fish and nuts like macadamia. Palmitoleic acid is one of the most common omega-7 fatty acids. Many studies suggest that this oil is good for heart health. Researchers at the National Institutes of Health (NIH) want to find out more about these potential benefits.

Research Study 000444-H investigating how well the following vaccines work in patients with CLL/SLL malignancies: SHINGRIX and influenza vaccine. If you are currently receiving treatment for your CLL/SLL (e.g. ibrutinib, acalabrutinib or venetoclax), you may be able to participate.

After stem cell transplantation, some people develop hard-to-treat anemia (low red blood cells) or thrombocytopenia (low platelets). Cytopenia(s) or low blood cell counts make you weak, prone to bleeding and bruising, and dependent on blood product transfusions. Researchers at the National Institutes of Health (NIH) are testing the drug fostamatinib to see if it is safe to treat cytopenia(s) after stem cell transplantation. A physician referral is not required to participate.

Pharmacologic therapy with Bruton's tyrosine kinase (BTK) inhibitors is associated with an increased risk of heart problems like atrial fibrillation, bleeding, and high blood pressure (hypertension) which can lead to sudden death. There is currently no standard for cardiac screening or monitoring patients on BTK inhibitors. Researchers at the National Heart, Lung, and Blood Institute (NHLBI) are investigating the relationship between the development of heart problems in patients on BTKi as it relates to other variables.

Doctors at the National Institutes of Health (NIH) are conducting a research study to determine the viability and safety of early initiation of oral therapy with cyclosporine and eltrombopag in patients with severe aplastic anemia (SAA).