Inborn error of immunity

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 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 early study tests baricitinib, a drug already approved for other immune conditions, in 20 people with Job syndrome who also have lupus-like disease or eczema. Participants take the pill daily for 6 months. The main goal is to see if it is safe and tolerable, while also check…

This study tests a stem cell transplant to control peripheral T-cell lymphoma in people whose cancer has not responded to standard treatments. Participants receive a donor's stem cells after chemotherapy or antibody therapy to prepare their body. The goal is to see if this approa…

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 phase 2 trial tests whether the drug nivolumab can slow or stop the growth of Epstein-Barr virus (EBV)-positive lymphoproliferative disorders and non-Hodgkin lymphomas. About 40 people aged 12 and older whose cancers have not responded to standard therapy will receive nivolu…

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 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…

The DETERMINE trial is testing whether drugs already approved for some cancers can help people with rare cancers or common cancers that have rare genetic mutations. It will enroll 825 adults, teenagers, and children whose cancers have specific DNA changes. The goal is to find new…

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 whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

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 study tests an experimental drug called Tulmimetostat (DZR123) in people with advanced solid tumors or lymphomas that have not improved with standard treatments. The drug is taken as a daily pill, either alone or combined with another drug (enzalutamide) for certain prostate…

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 drug called BCX17725 in healthy volunteers and people with Netherton syndrome, a rare genetic skin condition. The study aims to check safety, how the drug moves through the body, and whether it can reduce skin redness and scaling. About 78 parti…

This study tests a drug called soquelitinib in 15 people aged 16 and older with autoimmune lymphoproliferative syndrome (ALPS). The goal is to see if the drug can shrink an enlarged spleen or lymph nodes and improve low blood cell counts. Participants take the pill twice daily fo…

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 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 phase 2 trial tests the drug daratumumab in 28 adults with three rare and aggressive blood cancers: primary effusion lymphoma, plasmablastic lymphoma, and multicentric Castleman disease. Participants must have failed standard therapy or be unable to receive it. Daratumumab i…

This study tests a drug called pacritinib in people with HIV who have two rare conditions caused by the Kaposi sarcoma herpesvirus (KSHV): KSHV-associated multicentric Castleman disease (MCD) and KSHV inflammatory cytokine syndrome (KICS). These conditions cause severe, life-thre…

This phase 2 study tests whether giving emapalumab or a combination of fludarabine and dexamethasone before a stem cell transplant can reduce inflammation and help the donated cells take hold better. The trial involves 39 people with primary immune regulatory disorders or autoinf…

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 whether a blood test (ctDNA) can help doctors choose the right treatment for people with a type of lymphoma that happens after an organ transplant (PTLD). About 30 participants will be split into low-risk and high-risk groups. Low-risk patients will get only the …

This phase 3 trial tests tabelecleucel, a cell therapy made from donor immune cells, in 115 organ or stem cell transplant patients who developed a rare cancer (EBV+ PTLD) after standard treatment failed. The goal is to see if it can shrink tumors and improve outcomes. Participant…

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 lab-made human protein called CYT107 in 55 people with HIV and Kaposi sarcoma. The protein aims to boost T cells, which may help shrink cancer tumors. Participants receive weekly injections for up to 4 weeks, with follow-up for 3 years.

This study tests whether a stem cell transplant from a donor can help people with VEXAS syndrome, a rare autoinflammatory disease. Participants receive donor stem cells after drugs to prepare their bone marrow. The goal is to see if the transplant can reverse symptoms and allow p…

This study looks at whether a simple breathing test (spirometry) can help doctors decide the right dose of immunoglobulin therapy for people with common variable immune deficiency (CVID). Researchers will enroll 22 adults with CVID who already take a stable dose of Hizentra. Half…

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 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 early-stage trial tests a new gene therapy called KLN-1010 for people with multiple myeloma that has come back or stopped responding to treatment. Instead of removing cells and engineering them in a lab, this therapy works inside the body to create special immune cells that …

This study tests a drug called ruxolitinib as the first treatment for children with a rare, life-threatening immune condition called primary HLH. The goal is to calm the overactive immune system and help more children survive long enough to receive a stem cell transplant. The tri…

This study tests the drug ruxolitinib in 14 adults with idiopathic multicentric Castleman disease (iMCD) whose condition did not improve with standard therapies like siltuximab or tocilizumab. The goal is to see if ruxolitinib can improve symptoms such as fatigue, fever, and nigh…

This study tests a lotion called QRX003 in people with Netherton Syndrome, a rare genetic skin condition. About 16 participants aged 4 and older will apply the lotion twice daily for 12 weeks. Researchers will check if it improves skin appearance and reduces itching.

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 study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests an experimental oral drug called emavusertib (CA-4948), taken alone or with ibrutinib, in adults with relapsed or refractory primary central nervous system lymphoma (PCNSL). The trial has multiple parts to find the safest dose and see if the drug shrinks tumors. …

This study tests a drug called AVM0703, a very strong steroid, given as a one-time IV infusion. It aims to see if it is safe and can shrink tumors in people with certain blood cancers like lymphoma and leukemia. The trial will enroll 144 participants aged 12 and older.

This study tests whether specially grown immune cells (BK-specific cytotoxic T-cells) from a related donor can safely lower BK virus levels in children, teens, and young adults. Participants have BK infections that did not get better with standard treatment, often after a stem ce…

This study tests whether a medicine called XEMBIFY, given every two weeks alongside standard care, can reduce serious bacterial infections in adults with certain blood cancers (chronic lymphocytic leukemia, multiple myeloma, or non-Hodgkin lymphoma) who also have low antibody lev…

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 early-stage study is testing whether quercetin, a natural antioxidant found in foods, is safe and can reduce inflammation in male patients with XIAP deficiency, a rare genetic immune disorder. Twelve participants will take oral quercetin daily. The main goals are to check fo…

This study tests a one-time gene therapy for a single person with X-linked Hyper-IgM syndrome, a rare immune disorder caused by a faulty CD40L gene. The participant's own stem cells and T cells are collected, edited in a lab to fix the gene mutation, and then infused back after c…

This study tests a two-step transplant for children with type 1 diabetes who also have an immune deficiency. First, they receive umbilical cord blood stem cells to rebuild their immune system. Then, if that works well, they get a transplant of insulin-producing islet cells. The g…

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 2 trial is testing whether epcoritamab, a type of antibody, can help people with Waldenstrom Macroglobulinemia (a rare blood cancer) who have already received treatment. The study will enroll 20 participants and measure how many respond to the drug. Epcoritamab is give…

This study tests whether IgPro20 (HIZENTRA), a concentrated antibody solution given under the skin, can prevent infections in people with multiple myeloma who have low antibody levels due to bispecific antibody therapy. About 100 participants will either receive IgPro20 plus stan…

This study tests a new way to make immune cells (called GRALE T cells) that are trained to attack cells infected with the Epstein-Barr virus (EBV), which is linked to some lymphomas. The trial includes people with Hodgkin or non-Hodgkin lymphoma, or related diseases, whose cancer…

This study looks at whether giving a longer course (10 days instead of 5) or adding another antiviral (remdesivir) to the standard Paxlovid treatment helps immunocompromised people clear COVID-19 faster. About 256 adults with mild or no symptoms will be randomly assigned to diffe…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

This study is testing whether giving a longer course of the antiviral Paxlovid (10 days instead of 5) or combining it with another antiviral (remdesivir) helps immunocompromised people clear COVID-19 faster. About 256 participants with mild to moderate COVID-19 and weakened immun…

This study is testing the long-term safety of a drug called leniolisib in people with primary immunodeficiency disorders linked to PI3K signaling. Participants are 12 people who already took leniolisib in a previous study and may benefit from continuing it. The goal is to see if …

This phase 2 trial is testing a combination of two drugs, acalabrutinib and obinutuzumab, in people with untreated follicular lymphoma or other slow-growing non-Hodgkin lymphomas. The goal is to see if this drug pair can shrink tumors and control the disease. About 49 participant…

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 drug called pacritinib in 100 people with T-cell lymphoma that has returned or not responded to prior therapy. The main goal is to see how many patients have their tumors shrink or disappear. Participants take the drug and are monitored with scans and skin asse…

This early-stage trial tests whether specially grown immune cells (norovirus-specific T-cells) can safely treat chronic norovirus infections in people with weakened immune systems, such as those who had a stem cell or organ transplant. The study will enroll 48 participants and gi…

This early-stage trial tests a new medicine called IMSB301 in people with rare diseases where the immune system attacks the body. The study includes up to 6 participants aged 12 and older. The main goal is to check if the drug is safe and how it behaves in the body.

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 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 phase 2 trial tests the drug venetoclax (a pill) in 110 people with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The study includes two groups: those with a specific genetic change (17p deletion) and those who have failed oth…

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 study is for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), rare conditions that cause severe pain and skin reactions to sunlight. The purpose is to check the long-term safety of an oral medication called dersimelagon. About 301 participant…

This study tests whether the drug pacritinib is safe and effective for adults with Waldenström macroglobulinemia, a rare blood cancer, when previous treatments have stopped working. About 30 participants will receive pacritinib and be monitored for tumor shrinkage and side effect…

People without a working spleen are at high risk of severe infections from pneumococcus bacteria. This study looks at how well the pneumococcal vaccine works in adults aged 18-65 who have a spleen injury and are treated with monitoring, embolization, or spleen removal. Researcher…

This phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…

This trial tests the drug entrectinib (Rozlytrek) in people with rare cancers that have a specific genetic change called ROS1 fusion. The drug is already approved for some lung cancers, but researchers want to see if it works for other tumor types. About 30 adults, teens, and chi…

This trial tests atezolizumab, a drug already approved for several cancers, in people with rare cancers that have high tumor mutational burden (TMB) or microsatellite instability (MSI). The goal is to see if the drug can shrink tumors or stop them from growing for at least 24 wee…

This study tests a drug called alectinib, already approved for some lung cancers, in people with rare cancers that have a specific genetic change called ALK. About 30 adults, teens, and children with ALK-positive cancers will receive alectinib to see if it shrinks tumors or stops…

This phase 3 trial is testing three different ways to give the meningococcal B vaccine to adults who have had their spleen removed. People without a spleen are at higher risk for serious infections. The study will compare how well each vaccine schedule triggers the immune system …

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This phase 2 trial tests whether abatacept (Orencia) can improve lung disease in people with common variable immunodeficiency (CVID). About 38 children and adults will receive either the drug or a placebo for 6 months. The main goal is to see if lung damage on CT scans shrinks by…

This study tests an experimental therapy that uses special immune cells (T-cells) from a donor to fight cytomegalovirus (CMV) infection in people who have had a stem cell transplant. The therapy aims to boost the patient's immune system to clear the infection. About 20 participan…

This study is testing whether umbilical cord blood stem cells from unrelated donors can safely and effectively restore blood cell production in people with serious conditions like leukemia, immune deficiencies, and inherited blood disorders. The main goal is to see how quickly th…

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 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 tests an 8-week virtual rehab program (CaRE@Home) for adults who finished cancer treatment for breast, colorectal, head and neck cancers, or lymphoma within the last two years. The goal is to see if the program reduces disability and improves physical function, anxiety…

This study aims to learn more about Hyper IgE syndrome, a rare immune disorder that causes skin and lung infections. Researchers will follow 600 patients and their relatives over time, examining their health and genetics. The goal is to better understand the disease and improve c…

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 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 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 looks at people who have had unusually severe or long-lasting viral infections, like serious cases of herpes, HPV, or the flu. Researchers want to find out if these people have hidden immune system or genetic problems that make them more vulnerable. Up to 600 participa…

This study follows 900 people with bronchiectasis—a condition where airways are damaged and prone to infection—to learn why they get sick repeatedly. Researchers will collect medical history, lung function tests, and blood, urine, and sputum samples from patients and their family…

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 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 find the genetic causes of inherited immune problems that affect white blood cells called lymphocytes. Researchers will review medical records and collect blood samples from up to 5,000 people with suspected immune disorders, as well as their relatives. The goa…

This study collects blood, bone marrow, and tissue samples from people with HIV, KSHV, or certain cancers to help researchers learn more about these diseases. Participants must be 18 or older and may have HIV, KSHV, or related conditions. No treatment is given; the goal is to gat…

This study follows people with autoimmune lymphoproliferative syndrome (ALPS) and their relatives over many years. Researchers will collect blood and tissue samples, track symptoms, and study genetics to better understand why the immune system attacks the body. The goal is to lea…

This study looks at people with severe or hard-to-treat Valley Fever, a lung infection caused by a fungus found in the southwestern U.S. Researchers want to understand why some people get seriously ill by studying their immune systems and genetics. Up to 400 participants will giv…

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 looks at the bacteria, fungi, and viruses living on the skin, in the gut, and in the mouth of people with Netherton syndrome, a rare condition that damages the skin barrier and often causes allergies. Researchers will compare these microbes to those of healthy voluntee…

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 research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This natural history study will follow 700 people with Down syndrome and their healthy relatives for up to 15 years. Researchers want to learn how removing part of the thymus during heart surgery affects the immune system over time. Participants will have yearly check-ups, blood …

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 at how well the immune system works in people with end-stage kidney disease. Researchers will take blood samples from 100 patients and healthy donors to study T-cells, a key part of the immune system. They also plan to test a lab technique to restore T-cell funct…

This study collects blood stem cells from adults aged 18-40 with DOCK8 deficiency, LAD-1, or GATA2 deficiency. Participants receive daily injections of G-CSF for 5 days to move stem cells into the blood, then undergo a single leukapheresis procedure to collect them. The cells are…

This study aims to learn more about non-tuberculous mycobacterial (NTM) infections, which cause repeated lung and other organ infections. Researchers will study up to 1,000 participants to understand the genetics, symptoms, and best treatments for these infections. The study also…

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 looks at the tiny organisms living on our skin—like bacteria and fungi—and how they might differ in people with eczema (atopic dermatitis). Researchers will collect skin samples from healthy volunteers and patients aged 2 to 40 with moderate to severe eczema, as well a…

This study collects tissue and blood samples from adults with HIV, other immune disorders, or cancers thought to be caused by viruses. Researchers will compare genes in cancer tissue to normal tissue to find genetic changes that drive these cancers. The goal is to better understa…

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 aims to find new genes that cause immune system problems by examining up to 500 people with immune disorders and their relatives. Participants provide blood, saliva, or tissue samples and may have medical tests, but no experimental treatments are given. The goal is to …

This observational study is recruiting 300 active-duty service members and military beneficiaries diagnosed with B-cell non-Hodgkin lymphoma. Researchers want to understand how exposure to toxins, genetic differences, and immune system problems are linked to this cancer. Particip…

This study aims to learn more about how HIV infects cells and affects the immune system by collecting blood samples from up to 2,419 adults, both with and without HIV. Researchers will analyze these samples to compare genetic and protein differences. The goal is to better underst…

This study aims to understand why some people get severe or unusual fungal infections due to immune system problems. Researchers will collect blood, tissue, and other samples from up to 1,200 participants over 25 years. No new treatments are being tested; the goal is to learn mor…

This study follows 950 people with idiopathic CD4+ lymphocytopenia (ICL), a rare condition where low CD4+ white blood cells raise infection risk. Researchers will track participants for up to 10 years, collecting medical history, blood samples, and infection data. The goal is to …

This study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers u…

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 collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

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 tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study follows 195 adults with KSHV-associated multicentric Castleman's disease (MCD) to better understand how the disease progresses and responds to treatments. Participants receive standard care and regular check-ups over several years. The goal is to improve future treatme…

This study aims to learn more about Chediak-Higashi syndrome, a rare genetic disorder that causes light skin and hair, easy bruising, and frequent infections. Researchers will observe up to 60 patients over time, collecting clinical and genetic data to better understand the disea…

This study collects blood and tissue samples from 200 adults with HIV and certain cancers (like Kaposi sarcoma, lymphoma, or anal cancer) to help researchers understand how HIV and cancer are connected. Participants donate samples, which are stored for future research. There is n…

This study aims to find genetic mutations that cause chronic active Epstein-Barr virus (CAEBV), a rare and serious disease mainly affecting children and young adults. Researchers will analyze blood and tissue samples from up to 50 patients and 150 relatives, plus anonymous donor …

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 aims to understand how the immune system and skin healing are connected in people with primary immunodeficiency, psoriasis, or eczema, as well as in healthy volunteers. Researchers will collect skin biopsies, blood samples, and optional skin swabs to analyze microbes a…

This study aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity. Researchers will observe up to 500 patients over time using blood tests, imaging, and other exams. The goal is to find better ways to diagnos…

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 looks at how well vaccines against respiratory viruses (like flu or COVID-19) work in people with weakened immune systems, including those with blood cancers. Researchers will measure both antibody and T cell responses after vaccination. The goal is to understand why v…

This study looks at how the immune system of healthy adults in Cambodia reacts to viruses that can spread from animals to people. Researchers will collect blood samples from 400 people who have close contact with animals, such as those working in wet markets or living near bat ca…

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 is a large registry that collects health information and leftover tumor or blood samples from up to 75,000 children and young adults with cancer. The goal is to track outcomes and gather data to help doctors find better ways to treat and care for these patients over ti…

This study looks at whether low levels of immunoglobulin G (IgG), a type of antibody, are linked to fatigue in people with multiple sclerosis (MS). Researchers will compare fatigue scores and infection rates between MS patients with low IgG and those with normal IgG. The goal is …

This study collects tumor samples and medical information from people with lymphoma or related blood cancers who have a family history of these diseases. Researchers want to find genetic changes that may increase the risk of developing lymphoma. Participants provide blood or tiss…

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 looks at infants having heart surgery, where the thymus gland is often fully removed to access the heart. The thymus helps build the immune system early in life. Researchers want to see if removing only part of the thymus keeps the immune system stronger and reduces in…

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 is a large registry that will follow 9,000 adults with lymphoproliferative disorders (a type of blood cancer) for up to 10 years. Researchers aim to learn how long people live, how different treatments work, and what factors affect the disease. No new treatments are gi…

This study is developing a questionnaire in Italian to find out how much people who have had their spleen removed know about preventing infections. A team of 20 healthcare experts will use a structured method to agree on the best questions. The final tool will help researchers an…

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 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 aims to find new biomarkers to better screen and monitor Activated PI3K Delta Syndrome (APDS), a rare immune disorder. Researchers will collect blood, urine, and stool samples from 14 APDS patients over two years to analyze changes in immune cells and other markers. Th…

This study collects and analyzes genetic and molecular data from 250 people with various blood cancers, including leukemia and lymphoma. Researchers will test cancer cells in the lab with many drugs to see which ones work best. The goal is to match genetic features with drug resp…

This study is a global registry for people with Castleman disease, a rare lymph node disorder. Researchers will collect medical records, lab results, and patient surveys from up to 1,000 participants to better understand symptoms, treatments, and outcomes. No experimental drug or…

This study is collecting bone marrow, blood, and tissue samples from over 1,000 people with various blood cancers. Researchers will use advanced genetic and protein analysis to identify unique markers for each cancer type. They will also test cancer cells against a library of 300…

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 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 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 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…

This study looks at whether giving splenectomy patients a structured lesson about infection risks and recommended vaccines helps them understand and remember better. About 100 adults who had their spleen removed at one hospital will take a quiz before and after the lesson. The go…

This study looks at gene changes that might cause low blood cell counts (cytopenia) in people with multiple myeloma or certain lymphomas after CAR-T therapy. About one-third of patients have low blood counts lasting more than 30 days after treatment. Researchers will test blood a…

This study looks at different forms of IgE (a protein involved in allergic reactions) in healthy volunteers and people with conditions like severe allergies, chronic hives, mastocytosis, and immune deficiencies. Researchers aim to understand how these IgE variations relate to hea…

This study looks back at medical records of 30 non-HIV patients with weakened immune systems who had toxoplasmosis, a brain infection. The goal is to understand how many are affected and how to better diagnose and treat them. Researchers will track survival rates to improve care …

This study aims to learn more about rare genetic diseases that affect both the immune system and the nervous system. Researchers will collect medical information and samples from 1,000 participants, including patients, their relatives, and healthy volunteers. The goal is to bette…

This study looks at how a person's body composition (fat and muscle) affects the way immunoglobulin G (IgG) moves through the body after being given through a vein or under the skin. Researchers will enroll 20 adults with a nerve condition called CIDP who already receive IgG ther…

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 120 people with various immune deficiencies to see if a chronic gut virus infection is tied to liver or digestive problems. Researchers will collect blood, stool, urine, and biopsy samples to screen for enteric viruses. The goal is to identify which patients a…

This study aims to learn how the binding of RNA with DNA (called R-loops) is linked to amyotrophic lateral sclerosis type 4 (ALS4) and other inherited neurological disorders. Researchers will observe up to 330 people aged 5 and older, including those with ALS4, related conditions…

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 creates a registry for adults with plasma cell disorders, such as multiple myeloma and amyloidosis. Researchers will collect health information and optional blood samples to better understand these conditions. The goal is to gather data that could support future resear…

This study looks at whether clozapine, a drug used to treat psychosis in Parkinson's disease, weakens the immune system over time. Researchers will measure immune markers in 24 patients before and after 6 and 12 months of treatment. The goal is to understand infection risks in th…

This study aims to understand why some people with alpha-1 antitrypsin deficiency (AATD) get more respiratory infections. Researchers will compare immune responses to a pneumonia vaccine (PCV20) in 30 adults who carry one abnormal copy of the SERPINA1 gene. Half have had frequent…

This study collects blood, tissue, and body fluid samples from up to 15,000 people with blood cancers or related disorders. The samples are stored in a central lab for future research on diagnosis, prognosis, and immune monitoring. Participants do not receive any treatment as par…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

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 study looks at how immune system diseases can cause problems in the brain and nerves, affecting movement, thinking, and feelings. Researchers will follow 350 people aged 2 and older with immune diseases, plus their healthy relatives, for up to 5 years. They will collect bloo…

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…

This study aims to learn more about rare disorders that affect how the body processes chemicals called pyrimidines and purines. These disorders can cause problems in the brain, blood, kidneys, and immune system, ranging from mild to life-threatening. Researchers will compare test…