Leukopenia

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 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

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 compares two treatments—Shengbai Oral Liquid (a herbal remedy) and Leucogen tablets (a standard drug)—for moderate neutropenia (low white blood cells) caused by anti-tumor drugs in breast cancer patients. Sixty participants will be randomly assigned to receive one trea…

This study tests whether a single injection of Telpegfilgrastim works better than daily Filgrastim shots to prevent fever and low white blood cells in children and young adults (ages 6-24) with solid tumors receiving strong chemotherapy. About 132 participants will receive either…

This phase II trial tests whether taking leucogen alongside standard ribociclib therapy can prevent severe neutropenia (dangerously low white blood cell counts) in women with early-stage hormone-positive breast cancer. About 94 participants will receive the combination and be mon…

This study tests whether testicular tissue frozen before puberty can be transplanted back into adult men to restore sperm production. Five men who had fertility-threatening treatments as children will receive their own preserved tissue. The goal is to see if sperm can be found in…

This early-stage study tests whether belimumab is safe for people with idiopathic CD4 lymphopenia (ICL), a rare condition causing low CD4 white blood cells and increased infection risk. About 20 adults aged 18-70 with ICL and autoantibodies will receive 8 doses of belimumab intra…

This study is for people with blood cancers like leukemia who are getting chemotherapy or a stem cell transplant, which can cause very low white blood cell counts (neutropenia) for over a week. The goal is to see if the antibiotic eravacycline can prevent serious bacterial infect…

This study is checking whether unlicensed cord blood units are safe to use for stem cell transplants in children and adults with blood disorders. Researchers will closely watch for any infusion-related problems. The goal is to see if these units can be a safe option for patients …

This Phase 2 trial tests whether adding trilaciclib to the chemotherapy drug lurbinectedin can reduce severe neutropenia (a dangerous drop in white blood cells) in people with small cell lung cancer that has spread. About 30 participants will receive both drugs intravenously. The…

This study tests the drug luspatercept in 50 adults with clonal cytopenias of uncertain significance (CCUS), a condition where low blood cell counts increase the risk of developing blood cancers like leukemia. Participants receive injections every three weeks for up to 48 weeks. …

This study tests whether the drug lusutrombopag can help restore platelet levels faster in patients who have had a stem cell transplant for blood disorders. About 45 adults will receive the drug after transplant, and doctors will check platelet counts at 21 days. The goal is to i…

This study tests an experimental drug called NT-I7 in people with idiopathic CD4 lymphopenia (ICL), a condition where low CD4 immune cells lead to severe infections and other diseases. The goal is to see if NT-I7 can safely increase CD4 cell counts. About 60 adults aged 18–75 wil…

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 tests a tablet called PRaG-1 (made from cordycepin, a mushroom extract) in people who had solid tumors and now have low CD8+ immune cells after finishing radiation or chemotherapy at least six months ago. About 127 participants will take the tablet for two weeks, and t…

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 tests whether a drug called mecapegfilgrastim can safely prevent moderate-to-severe myelosuppression (low blood cell counts) caused by chemotherapy and radiation for lung or esophageal cancer. About 30 adults aged 18-75 will receive the drug alongside their standard ca…

This study is for adults with blood disorders who are getting a stem cell transplant from a matched donor. Researchers want to see if giving a special potato starch along with a drug that lowers iron (deferasirox) can help prevent serious complications like graft-versus-host dise…

This expanded access program provides nogapendekin-alfa inbakicept (NAI) to adults with solid tumors who have low lymphocyte counts caused by prior cancer treatments like chemotherapy, immunotherapy, or radiation. The goal is to reverse and maintain healthy lymphocyte levels. It …

This study aims to see if a home lactate meter and a standard glucose meter give accurate readings compared to lab tests in people with glycogen storage disease types Ia, Ib, and XI. Ten participants will have an 8-hour hospital stay with hourly blood draws and finger-stick tests…

This real-world study looks at whether a drug called mecapegfilgrastim can prevent neutropenia (a low white blood cell count) in cancer patients after chemotherapy, radiation, or immunotherapy. The study will follow 200 adults with solid tumors who are at high or moderate risk of…

This study tests two oral medications—diosmin-hesperidin (a flavonoid supplement) and loratadine (an allergy drug)—to prevent bone pain caused by G-CSF, a drug that boosts white blood cells in blood cancer patients. Researchers will compare each drug alone and in combination agai…

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 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 collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

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 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 aims to collect stem cells from people with a rare condition called idiopathic CD4 lymphocytopenia (ICL) and from healthy volunteers. Participants receive two medications to move stem cells from bone marrow into the blood, where they are collected. The stem cells are t…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

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 collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

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 looks at how radiation treatment for esophageal cancer affects the body's ability to respond to the pneumonia vaccine. Researchers will give the vaccine to 80 patients who have low white blood cell counts after chemoradiation. They want to see if the vaccine still work…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study is creating a registry for people with Shwachman-Diamond Syndrome (SDS) or similar conditions. Researchers will collect medical records and biological samples from up to 5,000 participants to understand how the disease progresses and what treatments work best. The goal…

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 gut bacteria and the immune system interact in children and adults receiving stem cell transplants or CAR-T therapy. Researchers will collect stool and blood samples over time to find patterns linked to complications like infections or graft-versus-host di…

This study explores whether cancer patients can safely monitor their own blood counts at home during chemotherapy. About 265 participants from Denmark, Poland, and Germany will test a home monitoring device for several cycles. The goal is to reduce unnecessary hospital visits, lo…

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 is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study follows 102 women who had fertility preservation before cancer treatment to see what happens long-term. Researchers want to know if they get pregnant naturally, use stored eggs or tissue, or decide not to have children. The goal is to improve support and care for femal…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study is creating a collection of blood samples from 2,000 people with various blood disorders. The samples will be stored and used for future research approved by an ethics board. No treatments or drugs are being tested, and participants continue their normal care.

This study looks at families who carry a change in the DDX41 gene, which may raise the risk of blood cancers like leukemia. Researchers will collect health questionnaires and saliva samples from up to 910 people to track who develops blood problems and when. The goal is to better…

This study looks at using a needle placed into the bone marrow (intraosseous infusion) to give fluids and medicines quickly to critically ill patients with blood diseases. Researchers will enroll 52 adults aged 18-65 to see how often the first attempt works and how safe it is. Th…

This study looks at how blood cells interact and what goes wrong in inherited bleeding disorders like Glanzmann thrombasthenia. Researchers will analyze blood samples from up to 60 healthy volunteers and patients to find genetic defects. The goal is to better understand these con…

This study looks at how iron supplements, often given for anemia, affect the immune system in people with Hereditary Haemorrhagic Telangiectasia (HHT), a rare genetic disorder that causes frequent nosebleeds and abnormal blood vessels. About 155 adults with HHT will be divided in…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study looks at whether a quick ultrasound of the thigh muscle can detect muscle loss (sarcopenia) in people who have finished cancer treatment. Researchers will compare the ultrasound results with a standard whole-body scan (DEXA) in 55 adults aged 18-74 with certain cancers…