Autoimmune enteropathy and endocrinopathy - susceptibility to chronic infections syndrome

This early-stage trial tests a single infusion of allogeneic CAR-T cells, called RN1201, in 18 adults with autoimmune diseases that have not responded to standard treatments. The therapy aims to reset the immune system by targeting and eliminating faulty immune cells. Before the …

This study tests whether a stem cell transplant can cure people with serious immune system disorders. Participants, aged 4 to 69, receive healthy donor stem cells to rebuild their immune system. The goal is to see if the transplant can permanently fix the immune defect and elimin…

This study tests whether a stem cell transplant from a healthy donor can help people with severe primary immunodeficiencies. Participants aged 4 to 75 receive chemotherapy before the transplant to prepare their body. The goal is to see if the procedure is safe and can improve imm…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

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 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 expanded access program provides TAK-881, a subcutaneous immunoglobulin treatment, to people with primary immunodeficiency diseases (PIDD) who completed a prior study and continue to benefit from the therapy. Participants receive regular infusions under the skin using a spec…

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This study is testing a new blood filter called the Plasma Filter PX2. It is used in a procedure called therapeutic plasma exchange, which removes harmful substances from the blood of people with autoimmune diseases. About 46 adults will take part, and the study will check if the…

This early-stage study tests a new mRNA-based treatment (ABO2203) for people with autoimmune diseases that haven't improved with standard care. The therapy works by directing the body's immune cells to attack and control overactive immune cells causing the disease. The study will…

This phase III trial tests V-IMMUNE, an intravenous immunoglobulin (IVIG) product, in about 50 people with primary immunodeficiency (PID). Participants, aged 2 and older, will switch from their current IVIG to V-IMMUNE every three weeks for a year. The study aims to see if V-IMMU…

This study tests a new antibody replacement therapy (BP-SCIG 20%) given as a weekly injection under the skin. It aims to see if it is safe and effective at preventing serious infections in people with primary immunodeficiency, a condition where the immune system cannot produce en…

This trial is developing an AI-powered device that listens to children's heart, breath, and bowel sounds to identify signs of common diseases like congenital heart disease, bronchopneumonia, and abdominal issues. It will involve 30,000 children aged 0-18, some with these conditio…

This study is testing whether the shingles vaccine (RZV) works and is safe for adults with autoimmune rheumatic diseases like rheumatoid arthritis or lupus who are taking immune-suppressing drugs. Two hundred participants will receive two doses of the vaccine. Researchers will me…

This study is looking for the genes that cause certain immune system problems. Researchers want to understand why some people get sick and others don't, even in the same family. Up to 5,000 people with immune disorders and their relatives will give blood or cheek cell samples for…

This study looks at how genetic immune system problems might lead to gum disease and other mouth issues. Researchers will collect samples like saliva, plaque, and blood from 700 people, including those with immune defects, healthy adults with gum disease, and healthy adults witho…

This study will analyze DNA from up to 20,000 people, including those with immune disorders and their relatives, to find genetic variants linked to conditions like atopy, autoimmunity, and primary immunodeficiency. Researchers will also study how best to return genetic results to…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

Researchers are running a worldwide survey to learn how COVID-19 affects children and adults who have primary immune deficiencies, a group of rare inherited conditions that weaken the immune system. Doctors enter health information about their patients who had confirmed or probab…

This study aims to collect blood, saliva, and other samples from up to 3,000 people with primary immunodeficiency (PID) or their relatives. Researchers will analyze the samples to find genetic causes of PID. The goal is to better understand these inherited immune disorders, which…

This registry follows pregnant women who have taken mycophenolate, a drug used to prevent organ rejection and treat autoimmune diseases. Researchers will collect data on pregnancy complications and birth defects up to one year after birth. The goal is to better understand the dru…

This study aims to learn more about primary immunodeficiency disorders (PIDs), which weaken the immune system and make people more prone to infections. Researchers will compare genetic and health data from people with PIDs, their healthy relatives, and healthy volunteers to find …

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 looks back at medical records of about 100 people in Japan with primary immunodeficiency who received Cuvitru. The goal is to track serious side effects like allergic reactions, blood clots, and meningitis. No new treatment is given—researchers are simply analyzing exi…

This study is measuring a protein called STIM1 on the surface of immune cells in 670 people with various autoimmune diseases. The goal is to find which diseases have high levels of this protein, which could help researchers develop new antibody-based treatments. Participants prov…

This study surveys 200 adult women with primary immune deficiency diseases about their use of intrauterine devices (IUDs), tampons, and menstrual cups. Researchers want to see if these products lead to infections like pelvic inflammatory disease or toxic shock syndrome. By collec…

This study aims to find new genetic causes of primary immunodeficiency, a condition where the immune system doesn't work properly, leading to frequent or severe infections. Researchers will compare the genes of 500 people with suspected immunodeficiency, their relatives, and heal…

This study aims to better understand primary immune deficiencies (PID), a group of diseases where the immune system doesn't work properly. Researchers will collect blood, stool, urine, and tissue samples from 300 people with PID and healthy volunteers to analyze immune responses.…

This study collects and freezes testicular tissue from boys and young men who are at risk of becoming infertile due to cancer, autoimmune disorders, or their treatments. The tissue is stored in a research bank, with the hope that future medical advances will allow it to be used t…

This study looks at 320 adults who are about to start strong immune-suppressing therapy and have already had at least two COVID-19 shots. Researchers want to find out if giving a COVID-19 booster right before starting therapy or waiting six months provides better protection. Part…

This study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

This study aims to find the genetic causes behind certain allergic and immune system disorders. Researchers will collect blood, saliva, and tissue samples from up to 10,000 people of all ages, along with some family members. No treatments are given; the goal is simply to learn mo…

This study looks at how JAK-inhibitor drugs affect the immune system and tests a gene therapy approach in cells from patients with primary immunodeficiency diseases. Researchers will collect blood samples from 20 adults to measure immune cell changes and gene editing efficiency. …