Agammaglobulinemia

This trial tests a one-time gene therapy for WHIM syndrome, a rare inherited immune disorder that causes severe infections. Doctors collect a person's own blood stem cells, use base editing to correct the faulty CXCR4 gene, and return the edited cells to the body. The goal is to …

This study is testing the long-term safety of a drug called leniolisib in people with primary immunodeficiency disorders linked to PI3K signaling. Participants are 12 people who already took leniolisib in a previous study and may benefit from continuing it. The goal is to see if …

This phase 3 trial is testing a new medicine called Newnorm, a concentrated antibody solution given as a shot under the skin, in 50 people with primary immunodeficiency (a condition where the immune system doesn't work properly). The goal is to see if Newnorm can prevent serious …

This phase 2 trial tests whether the supplement fisetin can improve lung disease in people with common variable immunodeficiency (CVID). Twenty participants will receive either fisetin or a placebo on four days over a month. The study measures changes in immune cells and lung fun…

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 study tests a drug called leniolisib in children aged 1 to 6 years who have a rare genetic immune disorder called APDS. The drug aims to control the overactive immune system and reduce swollen lymph nodes. Researchers will monitor safety and how well the drug works over time…

This study tests a medicine called Kedrion IVIG 10% in 30 children with primary immunodeficiency, a condition where the immune system doesn't work properly. The treatment is made from donated antibodies and is given through a vein to help prevent serious bacterial infections. Res…

This study looks at whether giving Cutaquig® (a medicine that helps fight infections) by a faster manual push method is safe and easy for adults with primary or secondary immunodeficiency. About 36 participants will use the rapid push method at home. The goal is to see if this ap…

This study tests a drug called leniolisib in children aged 4 to 11 with APDS, a rare genetic condition that causes a faulty immune system. The goal is to see if the drug is safe and can control the disease by blocking an overactive immune signal. About 15 children will take the d…

This study tests the long-term safety of TAK-881, a subcutaneous immunoglobulin treatment, in 53 adults and children with primary immunodeficiency diseases (PIDD) who completed a prior study. Participants receive regular infusions every 2-4 weeks and are monitored for side effect…

This study tests whether a stem cell transplant from a healthy donor can safely treat people with severe T-cell problems. Participants receive chemotherapy and antibody therapy before the transplant, followed by donor stem cells. The goal is to see if the new immune system can co…

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 drug called leniolisib in 12 people aged 12 to 75 with certain primary immunodeficiency disorders. The goal is to see if the drug is safe and can help control immune system problems. Participants will receive three different doses of the drug, and researchers w…

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 collects small skin samples from people with primary immunodeficiency or immune regulation disorders, as well as from healthy volunteers. Researchers will use these cells to create cell lines for laboratory research. The goal is to better understand immune diseases and…