B cell deficiency

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 early-stage trial tests a cream called ATR12-351 for Netherton syndrome, a rare genetic skin condition. The cream contains live bacteria that produce a protein missing in people with the disease. Twelve adults will apply the cream to one side of their body and a placebo to t…

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 Phase 2 trial tested whether Humira (adalimumab), a drug used for arthritis, can help people with Netherton syndrome, a rare genetic skin condition that causes severe inflammation, itching, and pain. Eleven patients received six injections over three months, followed by thre…

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 early study tested a cream called SXR1096 on 9 people with Netherton syndrome, a rare inflammatory skin disease. The cream aims to block certain enzymes that cause skin problems. Participants applied the cream and a placebo to different skin areas for one month to check safe…

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 tested a combination of two immune-targeting drugs, secukinumab and dupilumab, in 15 children with severe Netherton syndrome, a rare genetic skin disorder. The goal was to see if the treatment could improve survival and control the disease. The children received weight…

This study tested whether analyzing proteins from kidney biopsy and urine samples can improve the diagnosis of microvascular inflammation, a key sign of antibody-mediated rejection in kidney transplant recipients. Researchers used mass spectrometry to find protein signatures in 1…

This study tested a document called Voicing My CHOiCES, designed to help young adults (ages 18-39) with serious illnesses like cancer or HIV discuss their care preferences. Participants filled out questionnaires about anxiety, social support, and communication, then reviewed the …

This study looked at how people with autoimmune diseases like multiple sclerosis or blistering skin conditions respond to COVID-19 vaccines while on B-cell depleting therapies. Researchers measured antibody levels and T-cell activity over time to understand vaccine effectiveness …

This small pilot study looked at whether clopidogrel, a drug that stops blood clots, could lower a substance called sCD40L in people with lupus. Lupus is an autoimmune disease where the body attacks itself. The study involved 18 adults with lupus and measured sCD40L levels in the…

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 Netherton syndrome, a rare genetic skin condition, to better understand how it affects the body and immune system. Researchers studied 18 people with the disease, measuring immune markers in the blood and tracking skin changes. The goal was to find new target…

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 …

This study collected blood samples from nearly 1,900 people in families where at least two members have a type of dysglobulinemia, including multiple myeloma, MGUS, or Waldenström's disease. Researchers will analyze the DNA to look for genetic variations that might increase the r…