Agammaglobulinemia

This study tested an extra dose of the HIPRA COVID-19 vaccine in 241 adults with conditions like HIV, kidney disease, or autoimmune disorders that weaken the immune system. The goal was to see if the booster raises antibody levels against virus variants and remains safe. Research…

This early-phase study tests whether Mozobil, a drug already used to boost stem cells for transplants, can safely raise white blood cell counts in people with WHIMS. WHIMS is a rare genetic condition that traps infection-fighting cells in the bone marrow, leading to frequent infe…

This study tests a drug called leniolisib in 20 people with common variable immunodeficiency (CVID), a condition that weakens the immune system. All participants will receive the drug for 6 months, starting at a low dose and increasing. The main goal is to check safety and tolera…

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This study tests a new intravenous immunoglobulin (IGIV) called PlasmaCap IG in adults and children aged 2 to 70 with primary immune deficiency diseases (PIDD). Participants receive regular infusions of the therapy for 12 months. The goal is to see if it prevents serious bacteria…

This study tested two drugs, plerixafor and G-CSF, in 20 people with WHIM syndrome, a rare genetic condition that causes frequent infections and warts. Participants received each drug for a year and tracked their infections. The goal was to see which drug better reduced infection…

This study tested whether regular infusions of Panzyga (a concentrated antibody solution from donated blood) could prevent serious infections in people with chronic lymphocytic leukemia (CLL) who also have low antibody levels. The trial included 247 adults with CLL who were recei…

This study checked the long-term safety of TAK-771 in 15 Japanese patients with primary immunodeficiency disease (PID). Participants received TAK-771 injections for up to 3 years. The main goal was to monitor side effects and immune reactions. The study is now complete.

This study tested a new intravenous immunoglobulin (IVIg) treatment called Kedrion IVIg 10% in 47 people with primary immunodeficiency, a condition where the immune system cannot make enough antibodies. The goal was to see if regular infusions of this antibody therapy could preve…

This study tested a new medicine called TAK-881, a concentrated antibody solution given under the skin, in 65 people with primary immunodeficiency diseases (PIDD). The goal was to see how well the body absorbs it and if it is safe compared to an existing treatment (HYQVIA). Parti…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This study analyzed DNA from 9 patients with primary immune deficiencies whose genetic cause was unknown. Researchers used whole exome or genome sequencing to look for new mutations that might explain their condition. The goal was to identify new inborn errors of immunity, which …

This study looked at whether ultrasound and stiffness measurements of the liver and spleen can predict a serious condition called portal hypertension in people with common variable immunodeficiency (CVID). Researchers examined 250 CVID patients during routine visits, using non-in…

This study looked for hidden immune system problems in adults who get frequent or severe bacterial infections, like pneumonia or meningitis. Researchers screened 120 adults aged 18-65 to see how many had an undiagnosed primary immunodeficiency. The goal was to find out if these c…

This observational study compared blood samples from 24 people with antibody deficiency (CVID) and healthy volunteers. Researchers used advanced technology to measure how different immune cells respond to various triggers. The goal was to find patterns that could improve diagnosi…

This study looked at 99 women with primary immune deficiency to learn more about their pregnancies and birth outcomes. Researchers tracked infections during pregnancy and after birth, as well as how well contraception worked. The goal is to help doctors give better advice to wome…

This study looked at how common variable immunodeficiency (CVID) changes the bacteria and immune activity in the respiratory tract. Researchers collected samples from 75 people with and without CVID to analyze microbes and gene activity. The goal was to understand why lung proble…

This study looked at 27 adults with a severe form of primary immunodeficiency, which causes frequent infections, enlarged lymph nodes, and low white blood cells. Researchers analyzed their DNA to find known and new genetic mutations linked to the condition. The goal was to better…

This study looked at how well the Pfizer COVID-19 vaccine triggers an immune response in 196 adults with weakened immune systems (due to conditions like kidney transplant, cancer, or HIV) compared to healthy adults. Researchers measured antibody levels and their ability to neutra…

This completed study in France looked at how common bronchiectasis (permanent lung damage) is in 46 adults with primary humoral immunodeficiency, a condition that weakens the immune system. Participants had a chest CT scan and lung function tests to check for bronchiectasis. The …

This study looked at whether epidural or spinal anesthesia during childbirth leads to more infections in women with primary immunodeficiencies (weak immune systems). Researchers tracked 30 women from a national registry for 28 days after delivery to see if they developed serious …