Lymphoproliferative syndrome

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…

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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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…