Inborn error of immunity

This study tests the drug upadacitinib (Rinvoq) in 30 people with rare genetic disorders that overactivate the JAK-STAT immune pathway. The trial has four phases over 12 months, including an open-label phase to find the best dose and a randomized withdrawal phase to compare the d…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…

This study tests whether a medicine called Panzyga can prevent major infections in people with autoimmune or rheumatic conditions who have low antibody levels after receiving B-cell depletion therapy. About 360 adults will receive either Panzyga or a placebo, and researchers will…

This study tests a drug called IgPro20 in 63 adults who have a weakened immune system after receiving CAR T-cell therapy for blood cancers like lymphoma or multiple myeloma. The goal is to see if the drug can lower the number of serious bacterial infections, such as pneumonia or …

This study tests a new medicine called Boya IVIG in 50 people with primary immunodeficiency (CVID or XLA), a condition where the body can't fight infections well. Participants get the drug every 21 or 28 days for a year to see if it keeps serious bacterial infections below one pe…

This trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplan…

This Phase II trial tests a drug called NM8074 in 12 adults with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots, low platelets, and kidney injury. The drug is given by IV infusion over several weeks. The goal is to see if NM8074 can bring bloo…

This trial tests whether two approved drugs, dabrafenib and trametinib, can treat rare cancers that have a specific genetic change called BRAF V600. These drugs are already used for melanoma and lung cancer, but researchers want to see if they help people with other cancer types.…

This study will follow about 80 Polish patients with atypical hemolytic uremic syndrome (aHUS) who are receiving ravulizumab (Ultomiris) as part of routine care. Researchers will collect data from medical records and follow patients over time to see how well the drug controls the…

This study will test whether analyzing stool and urine samples using a technique called metagenomics can find infections in people with weakened immune systems (due to HIV, chemotherapy, or immunosuppressive drugs). Currently, doctors often need to take invasive samples like bloo…

This study compares two types of nerve blocks given before lung surgery to see which one controls pain better. Sixty adults having video-assisted lung surgery will receive either a serratus posterior superior intercostal plane block or a deep serratus anterior plane block. The ma…

This study looks at how patients with primary or secondary immunodeficiency feel about using mechanical versus electronic infusion pumps for their immunoglobulin therapy. About 52 adults who have used electronic pumps will try a mechanical pump for three months and then report th…

This study looks back at medical records of about 60 people with primary immune deficiencies who also developed lymphoproliferative disorders (abnormal growth of immune cells). The goal is to track their health over time, including risks of death, cancer, or organ damage. By iden…

This study will follow 15 people with primary antibody immunodeficiency who are receiving immunoglobulin therapy. Researchers will track changes in specific immune cells (T and B cells) and infection rates over time. The goal is to see if these cell changes can help predict how w…

This study will measure two immune system markers, CD305 and CD85d, on cancer cells from 180 people newly diagnosed with B-cell blood cancers. The goal is to see how these markers relate to different cancer types and patient outcomes. No treatment is given; the study only observe…

This study will follow 5,000 adults with immune system problems to see if certain blood cell mutations (called clonal hematopoiesis) are linked to their condition. Researchers will track new cases of immune-related diseases over time. No treatments or drugs are tested—this is pur…

This study will test a new way to offer vaccines to vulnerable patients—those with chronic diseases or weakened immune systems—during hospital visits. Researchers will see if offering, prescribing, and giving vaccines in the hospital increases how many people get vaccinated. The …

This study looks at why some people with Common Variable Immunodeficiency (CVID) develop complications not caused by infections. Researchers will test whether a type of immune cell called peripheral helper T-cells (Tph) helps faulty B-cells produce antibodies that lead to these c…

This study looks at how children aged 6 to 18 with inborn errors of immunity (a condition where the immune system doesn't work properly) feel about their quality of life. Researchers will compare their answers to those of healthy children using a special questionnaire. The goal i…

This study aims to better understand why some people's immune cells attack their own body, causing autoimmune diseases like lupus and scleroderma. Researchers will analyze blood samples from 200 adults with these conditions to identify and measure specific harmful immune cells. T…

This study will look at 100 adults with low platelet counts to see how many have an enlarged spleen and whether that affects their symptoms, complications, or response to treatment. The goal is to better understand the role of the spleen in these blood disorders. No new treatment…