Hematologic disorder

This study tests a new oral drug, QTX-2101, combined with standard ATRA therapy for adults newly diagnosed with a low-risk form of acute promyelocytic leukemia (APL). The goal is to see if the oral capsule works as well as the current intravenous treatment. About 150 participants…

This Phase 3 trial tests whether mezagitamab, given as a shot under the skin, can safely keep platelet counts stable in adults with chronic ITP. ITP is a condition where the immune system destroys platelets, leading to easy bruising and bleeding. The study will compare mezagitama…

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 study tests an experimental drug called CGT9486 (bezuclastinib) in people with advanced systemic mastocytosis, a rare and serious blood disorder. The drug aims to control the disease by blocking a key protein that drives abnormal mast cell growth. About 140 adults with aggre…

This study tests a drug called crizanlizumab in people aged 12 and older with sickle cell disease who have frequent pain crises. The goal is to see if the drug can reduce the number of these crises compared to a placebo. Participants may also continue taking their usual treatment…

This study tests a medicine called KIg 10 (intravenous immunoglobulin) in adults with chronic ITP, a condition where the immune system destroys platelets, causing bleeding and bruising. The goal is to see if KIg 10 can safely raise platelet counts and stop bleeding. About 40 peop…

This study tests whether low-dose sirolimus can safely improve blood cell function in adults with RUNX1 familial platelet disorder, a genetic condition that raises bleeding and leukemia risks. Six participants receive the drug and are monitored for side effects and changes in blo…

This study tests the long-term safety of a drug called mezagitamab for adults with chronic immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing easy bruising and bleeding. About 150 participants who completed earlier mezagitamab studies w…

This study tests an experimental drug called ATV-1601 in people with moderate to severe HHT, a condition that causes frequent nosebleeds and anemia. The trial has two parts: first, participants receive either the drug or a placebo to check safety and effectiveness; then, all elig…

This trial tests whether having clinical dietitians take the lead on nutrition care—rather than waiting for referrals—can help adult medical inpatients at risk of malnutrition. Researchers will compare a dietitian-driven model with usual care on three hospital wards, measuring le…

This study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …

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 SIL-8301 (senicapoc) in 105 adults with sickle cell disease who have low hemoglobin but few pain crises. Participants take the drug or a placebo daily for 24 weeks, along with their usual hydroxyurea. The main goal is to see if the drug raises hemog…

This study tests whether a newer drug called pirtobrutinib works better than the current standard drug ibrutinib for people with chronic lymphocytic leukemia or small lymphocytic lymphoma. About 737 adults who need treatment for their cancer will be randomly assigned to receive o…

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 phase 2 trial is testing a new treatment called Orca-T for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Orca-T is a personalized cell therapy made from a donor's blood that includes stem cells and immune cells. The goal is to see if it can impr…

This trial tests whether a telemonitoring app called Cureety helps blood cancer patients take their oral therapies correctly and manage side effects. Participants will either use the app to report symptoms and track adherence or receive standard follow-up care. The goal is to see…

This trial tests a second stem cell transplant using a gentler conditioning regimen of busulfan, fludarabine, and low-dose total body irradiation. It is for people with blood disorders, hemoglobin diseases, or immune deficiencies whose first transplant did not achieve enough dono…

This early-phase trial tests whether stem cell transplants from donors whose tissue type does not fully match the patient can be safe and effective for people with blood cancers who have no other donor options. About 29 adults aged 18-60 will receive chemotherapy followed by the …

This early-phase study tests an oral drug called PMD-026 in 18 adults with myelofibrosis, a type of bone marrow cancer. Participants must have already tried a JAK inhibitor without success. The main goals are to check safety, find the best dose, and see if the drug can shrink the…

This study is testing an experimental oral drug called ASTX030 (a combination of azacitidine and cedazuridine) for people with certain blood cancers like myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The drug is given a…

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 study tests whether giving the immune-suppressing drug tacrolimus earlier than usual can lower the risk of cytokine release syndrome (CRS), a severe inflammatory reaction, in patients receiving a half-matched stem cell transplant for blood cancer. About 20 adults will receiv…

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 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 a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

This early-phase trial tests whether infusing specially designed immune cells can prevent blood cancer from coming back after a stem cell transplant. The cells target a specific protein (HLA-DPB1*04:01) found on many cancer cells and include a 'suicide switch' to turn them off if…

This early-phase study tests whether a special cell therapy called T-allo10 can safely help rebuild the immune system in children and young adults (up to age 45) with blood cancers who have received a stem cell transplant. The treatment is given after the transplant to speed up r…

This early-stage study tests a new treatment called IL-5 CAR-T cell therapy for people with a rare and hard-to-treat blood cancer called eosinophilic leukemia. The therapy uses a patient's own immune cells, modified to target and attack cancer cells. The main goals are to check s…

This study tests a new type of CAR T cell therapy that targets two proteins (CD19 and CD22) on leukemia cells. It is for children whose B-cell acute lymphoblastic leukemia has come back or not responded to standard treatments. The main goal is to find a safe dose and see if the t…

This study in Taiwan will follow about 100 people with moderate to severe hemophilia A who are already taking efanesoctocog alfa (a clotting factor) as part of their routine care. Researchers will track joint health, bleeding rates, and safety over up to 5 years using medical rec…

This Phase 2 trial tests a new method to improve stem cell transplants for people with blood disorders who lack a fully matched donor. Researchers will give patients specially processed donor immune cells (CD62L-depleted lymphocytes) alongside a T-cell depleted stem cell transpla…

This study compares a new radiation technique called total lymphoid irradiation (TLI) to the standard total body irradiation (TBI) for children aged 0-18 who need a stem cell transplant from a half-matched donor. The goal is to see if TLI reduces complications like graft-versus-h…

This study tests whether a simple card game, the Go Wish Game, can help people with advanced blood cancers or solid tumors discuss their end-of-life priorities with doctors. About 75 patients will use the cards to sort what matters most to them, then complete an advance care plan…

This study tests a group-based telehealth program called HORIZONS for adults living with chronic graft-versus-host disease (cGVHD) after a stem cell transplant. The program includes 8 sessions led by a therapist to help with self-management and quality of life. Researchers will c…

This study tests whether using a goal-setting tool (Goal Attainment Scale) helps children and teens with cancer maintain their physical abilities during a stem cell transplant hospital stay. About 176 participants aged 6 months to 18 years will be randomly assigned to either stan…

This study tests a 6-session counseling program called BMT-CARE for caregivers of people receiving a bone marrow transplant. The goal is to see if it improves the caregiver's quality of life compared to standard support. The study will enroll 800 caregivers and also look at what …

This study tests a new program that gives blood cancer patients access to blood transfusions at home while they receive hospice care. The goal is to see if this improves their quality of life, mood, and reduces hospital visits near the end of life. The trial will include 700 adul…

This study builds a registry of people who are receiving or have received radiation therapy for blood-related conditions. Researchers will gather medical records, imaging scans, lab results, and treatment data to see how radiation affects bone marrow reserve and patient outcomes.…

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 follows 100 adults with blood disorders who receive an unrelated cord blood transplant. Researchers will measure levels of γδ T cells—a type of immune cell—in the blood at several time points over the first year. The goal is to see if the pattern of γδ T cell recovery …

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 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 looks at whether a tailored exercise program can help older blood cancer survivors slow down the aging process. Researchers will enroll 180 people aged 50 and older who have had blood cancer. The goal is to see if participants can stick with the exercise plan and if it…

This observational study will look at 500 people with diffuse large B-cell lymphoma (DLBCL) in the Middle East and Africa. Researchers want to understand patient characteristics, current treatment patterns, and how easily patients can access newer therapies like CAR-T cell therap…

This study follows 100 people aged 55 and older who have clonal hematopoiesis (CH) or unexplained low blood counts. Researchers will monitor their health over time, assess risks for heart disease and blood cancers, and provide personalized support. The goal is to create a bluepri…

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 follows 2,000 people who have had ovarian or other solid cancers to learn why some later develop blood cancers like leukemia. Researchers will look at genetic changes in blood cells and treatment history to identify risk factors. No new treatments are being tested—the …

This study is building a large database of people with Diamond Blackfan anemia, a rare inherited bone marrow condition that causes anemia. Researchers will collect medical information from up to 900 participants to better understand how the disease develops and progresses. The go…

This research study collects blood, bone marrow, and tissue samples from people with cancer, blood disorders, and healthy volunteers. The goal is to study how the immune system changes during different treatments, including immunotherapy and chemotherapy. By comparing samples fro…

This study tests whether giving critically ill ICU patients red blood cells from a donor of the same sex (sex-matched) versus the opposite sex (sex-mismatched) affects their chance of survival. Over 11,000 adults in ICUs who need a transfusion will be randomly assigned to receive…

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 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 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 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…

This study follows 120 men with hemophilia A who have developed inhibitors (antibodies that block standard treatment). Researchers will observe how doctors manage their condition using different therapies, including immune tolerance induction and newer drugs like emicizumab. The …