Agranulocytosis

This phase 2 trial tests whether empagliflozin, a diabetes drug also known as Jardiance, can help people with glycogen storage disease type Ib (GSD-1b). GSD-1b causes low neutrophil counts, leading to frequent infections and bowel inflammation. The study gives empagliflozin orall…

This study tests a donor stem cell transplant for people with severe immune system problems, like SCID and Wiskott-Aldrich syndrome. The goal is to help the body make healthy blood cells and fight infections. Participants receive donated stem cells to rebuild their immune system.…

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This study tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

This study tests a new approach for people aged 5 to 45 with primary immune deficiencies and end-stage lung disease. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combined procedure is safe and can…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This phase 3 trial tests a long-acting injection called PEG-G-CSF to prevent severe neutropenia (dangerously low white blood cell counts) after chemotherapy in 250 women with breast cancer. Participants receive either the test drug or the already-approved Neulasta. The goal is to…

This study tests whether a specific massage technique called Peng's Fenjin Tuina can reduce pain from superior cluneal nerve entrapment syndrome, a condition that causes pain in the lower back and buttocks. Researchers will compare it to standard massage in 70 adults aged 18 to 7…

This study screens up to 10,000 people with blood disorders or healthy volunteers to see if they can safely join other active research studies at the National Heart, Lung, and Blood Institute. Participants undergo clinical, imaging, and lab tests. If no suitable study is availabl…