Defective phagocytic cell engulfment

This study tests whether adding certain drugs to a stem cell transplant can cure chronic granulomatous disease (CGD), a condition that weakens the immune system. About 50 people aged 4 to 65 with CGD will receive donor stem cells after a special drug and radiation regimen. The go…

This study tests if the drug vorinostat is safe and can help people with moderate-to-severe Crohn's disease by reducing gut inflammation. Participants who respond well may then receive a maintenance treatment with ustekinumab for up to two years. The goal is to control symptoms a…

This early-stage trial tests a new approach for chronic granulomatous disease (CGD), a genetic condition that leaves people vulnerable to severe infections. Researchers take a participant's own white blood cells, use mRNA to correct the genetic defect, and infuse the cells back. …

This trial tests a gene therapy for chronic granulomatous disease (CGD), a rare immune disorder that leaves patients vulnerable to severe infections. Ten patients will receive their own stem cells modified with a lentivirus to correct the faulty gene. The goal is to restore immun…

This early-stage trial tests a new gene-editing approach for X-linked chronic granulomatous disease (X-CGD), a rare immune disorder. Researchers will collect stem cells from 10 adult male participants, use base editing to correct the genetic mutation, and return the cells after m…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests a stem cell transplant for people with common variable immunodeficiency (CVID) and other immune disorders. The goal is to replace the faulty immune system with a healthy donor's stem cells. Participants are aged 5 to 40 and will receive a reduced-intensity transp…

This study tests a one-time gene therapy called EN-374 for people with X-linked chronic granulomatous disease (X-CGD), a rare immune disorder that causes severe infections. The treatment uses a modified virus to deliver a working gene to blood stem cells, aiming to restore the ab…

This study tests whether transplanting healthy donor stool into the gut can reduce colon inflammation in people with chronic granulomatous disease (CGD) who have colitis not helped by standard treatments. Twenty participants aged 10-60 will receive the transplant during a colonos…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This phase II trial tests whether a combination of ibuprofen, G-CSF, and plerixafor can safely and effectively mobilize stem cells from the bone marrow into the blood in people with X-linked chronic granulomatous disease (X-CGD). Only 3 adults aged 18-45 are being enrolled. The g…

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 is observing up to 3,500 people with immune system problems, including rare genetic conditions like chronic granulomatous disease and severe combined immunodeficiency. Researchers will track how these diseases change over time through blood tests, imaging, and checkups…

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study looks at people with unusual or repeated infections and their family members to find the genetic and biochemical causes of immune system problems. Researchers will collect blood, saliva, urine, and other samples over time to better understand these conditions. The goal…

This study aims to understand why some people are more prone to fungal infections, especially from Candida yeast. Researchers will collect blood, saliva, urine, and tissue samples from people with immune disorders, their family members, and healthy volunteers. No treatment is giv…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study is observing 400 people aged 12 and older who have noncirrhotic portal hypertension (NCPH) or are at risk for it. NCPH is a liver condition that increases blood pressure in the liver's blood vessels without causing cirrhosis, and often has no early symptoms. Researcher…

This study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

This study looks at how common certain antibodies and inflammatory markers are in people with chronic granulomatous disease (CGD). Researchers will measure levels of adenovirus-fighting antibodies and inflammatory cytokines in blood samples from 60 adults and children with CGD. T…

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 looks at how immune cells behave in people with lung cancer, sarcoidosis, and COPD. Researchers will collect blood and tissue samples from 425 participants to analyze immune function. No experimental treatments are given. The goal is to understand why some patients don…