Complement deficiency

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 study tests an experimental drug called ADX-097 in people aged 16 and older with three kidney conditions: IgA nephropathy, lupus nephritis, and C3 glomerulopathy. The drug is given as an injection under the skin and is designed to reduce inflammation and protein leakage in t…

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 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 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 looks at the long-term safety and effectiveness of the drug iptacopan in people with a rare blood disorder called atypical hemolytic uremic syndrome (aHUS). Participants must have completed a previous iptacopan study and still benefit from the drug. The goal is to moni…

This study is for adults with a rare blood disorder called aHUS who are currently receiving antibody infusions. Researchers want to see if switching to a daily pill called iptacopan is safe and works just as well to prevent dangerous blood clots. About 50 participants will take i…

This study is testing the drug ravulizumab in 20 people with atypical hemolytic uremic syndrome (aHUS), a rare condition that causes blood clots and kidney damage. The goal is to see if the drug improves platelet counts and kidney function over 26 weeks. All participants receive …

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 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 study is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

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…

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 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 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 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 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 reviews medical records of 200 people diagnosed with a rare blood and kidney disease called aHUS in Egyptian hospitals. Researchers want to learn about patient symptoms, treatments, and long-term health to help doctors diagnose and manage the disease better. No new tre…

This study follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This study aims to improve diagnosis for people with rare genetic diseases whose standard genetic tests came back negative. Researchers will take a small skin sample and analyze RNA to find hidden genetic changes. The study involves 105 participants, including healthy volunteers …

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. …

This study looks at whether low levels of a protein called mannose-binding lectin (MBL) in the blood are linked to repeated pregnancy loss or failed IVF attempts. Researchers will measure MBL levels in 500 women with these conditions and compare them to the general population. Th…

This study looks at people with atypical hemolytic uremic syndrome (aHUS), a rare disease that damages small blood vessels and kidneys. Researchers want to understand how certain rare gene mutations cause the disease by growing cells from blood samples in the lab. The goal is to …

This observational study follows 3,000 people with atypical hemolytic-uremic syndrome (aHUS), a rare and serious kidney condition. Researchers will collect safety and effectiveness data on patients treated with eculizumab or ravulizumab, as well as those receiving other care. The…

This study is collecting blood, urine, and stool samples from 500 children with autoimmune diseases and healthy controls. Researchers will analyze these samples to find molecular patterns that could help diagnose these conditions and guide treatment decisions. The goal is to crea…