MYELODYSPLASTIC SYNDROMES

This study tests a new way to prepare donor stem cells (called Orca-Q) for people with blood cancers like leukemia or MDS who need a transplant. The goal is to see if this engineered graft is safe and helps the body accept the new cells with fewer complications. About 300 partici…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

This phase II trial tests whether adding targeted total marrow irradiation to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia, chronic myeloid leukemia, or myelodysplastic syndromes. The study will enroll 3…

This early-phase trial tests a cell therapy called Orca-T combined with standard drugs to prevent graft-versus-host disease (GVHD) in 24 adults with advanced blood cancers (like leukemia or MDS) receiving stem cell transplants from partially matched donors. The main goal is to se…

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 study tests a new experimental drug called EP0042 in adults with advanced acute myeloid leukemia (AML) or related blood cancers. The goal is to find a safe dose that might work against the cancer, either alone or with other treatments. About 70 participants will take part to…

This early-stage trial tests a special type of immune cell (CIML NK cells) combined with a low dose of IL-2 in people whose blood cancer (like AML or MDS) came back after a stem cell transplant. The goal is to find a safe dose and see if it can put the cancer back into remission.…

This early-phase trial tests whether specially trained natural killer (NK) cells, called CIML NK cells, along with interleukin-2 (IL-2), can safely prevent leukemia from coming back after a stem cell transplant. The study enrolls 15 adults with high-risk acute myeloid leukemia, m…

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like acute myeloid leukemia, that have a specific IDH1 mutation. The study aims to find the safest dose and see if the drug can control the disease. About 291 participants will take the…

This study tests a new treatment for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to standard therapy. The treatment uses specially engineered natural killer (NK) cells designed to recognize and attack cancer cells…

This study tests whether alternating two different drug combinations can help control higher-risk myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML). About 60 adults who have not responded to or relapsed after standard treatment will receive cladribine, low…

This early-stage trial is testing an experimental drug called JNJ-95804306 in people with blood cancers like leukemia that have come back or not responded to standard treatments. The study has two parts: first, finding a safe dose, and then checking if the drug shrinks tumors whe…

This study tests a stem cell transplant method that removes certain immune cells (T-cells and B-cells) from the donor cells. The goal is to reduce graft-versus-host disease, a serious complication where donor cells attack the patient's body. The trial includes 20 children and you…

This study tests a drug called momelotinib in 80 adults with low-risk myelodysplastic syndromes who have anemia. The goal is to see if it can help them need fewer red blood cell transfusions. Participants will receive different doses to find the safest and most effective one.

This early-phase study tests whether adding hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) to standard stem cell transplant preparation is safe and helps recovery. It involves 24 adults with various blood cancers, such as leukemia and myelofibrosis. Th…

This study looks at how the cancer drug ASTX727 (decitabine and cedazuridine) works in people with moderate or severe liver problems. About 27 adults with certain blood cancers or solid tumors will take the drug for up to 8 weeks. The goal is to understand how the liver affects t…

This early-stage study looks at how the body handles the cancer drug ASTX727 (decitabine and cedazuridine) in people with severe kidney problems compared to those with normal kidneys. It includes 18 adults with acute myeloid leukemia, myelodysplastic syndromes, or solid tumors. T…

This early-stage study tests a new drug called MGD024 in people with certain blood cancers (like leukemia and lymphoma) that came back or didn't get better with standard treatments. The main goal is to find the safest dose and watch for side effects. About 130 adults will receive…

This study tests a combination of three drugs—olutasidenib, decitabine, and venetoclax—in people with acute myeloid leukemia (AML) that has a specific IDH1 gene mutation. The goal is to find a safe and effective dose for those whose cancer has returned or not responded to treatme…

This early study tests a new way of giving two FDA-approved drugs (5-azacitidine and decitabine) for myelodysplastic syndromes (MDS), a type of bone marrow failure. Instead of standard doses, the drugs are given at low doses on alternating weeks to try to overcome resistance. Abo…

This phase 3 trial tests elritercept, a drug given as a shot every 4 weeks, to see if it can help adults with a bone marrow disease called MDS go without red blood cell transfusions for at least 8 weeks. About 225 participants with very low- to intermediate-risk MDS who need regu…

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome (MDS) who also have anemia. The goal is to see if the drug can reduce the need for blood transfusions and increase red blood cell levels. The study is in earl…

This study tests whether a cord blood transplant can help children and young adults (up to age 26) with blood cancers like leukemia and lymphoma. Participants receive the transplant as their first or second treatment. The main goal is to see how many are cancer-free one year afte…

This early-phase trial tests a new cell therapy for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who still have small amounts of cancer cells after standard treatment. The therapy uses the patient's own blood cells, armed with a special antibody to t…

This phase II trial tests different drug combinations to prevent graft-versus-host disease (GVHD) in 400 blood cancer patients receiving stem cell transplants from unrelated donors. Participants will receive either thymoglobulin or a combination of cyclophosphamide with other imm…

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 a low-dose, weekly chemotherapy combination (decitabine and venetoclax) given after a bone marrow transplant can help prevent high-risk myeloid cancers like acute myeloid leukemia from coming back. About 20 participants will receive the treatment for up t…

This phase 2 trial tests an optimized cord blood transplant for 35 patients with high-risk blood cancers (like leukemia) who relapsed after their first stem cell transplant. Participants receive chemotherapy followed by a cord blood infusion, plus drugs to prevent rejection. The …

This program provides galinpepimut-S, a cancer vaccine that aims to train the immune system to fight cancer cells, to individual patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who have no other treatment options. It is for people who cannot join a cl…

This study is testing a new drug called BGB-11417 (sonrotoclax) in people with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or related blood cancers. The drug works by blocking a protein that helps cancer cells survive. The trial will give the drug alone or toge…

This early-stage trial tests whether adding leflunomide to the standard drug decitabine is safe and effective for people with myelodysplastic syndromes (MDS) that have returned or not responded to prior treatment. About 26 adults will receive the combination in cycles. The main g…

This phase 2 trial tests a stem cell transplant using a milder chemo and radiation regimen for people with blood cancers like AML and MDS. The goal is to see if this approach improves survival at 100 days after transplant. Participants receive donor stem cells along with drugs to…

This early-stage trial tests an experimental oral drug called GLB-001 in adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) have come back or not responded to treatment. The study aims to find a safe dose and see if the drug shows any signs of …

This early-phase study tests a new automated method (CliniMACS Prodigy) to select CD34 stem cells for patients with blood cancers who need a stem cell boost or second transplant. The goal is to reduce the risk of severe graft-versus-host disease (GVHD) while improving blood cell …

This study tests an experimental drug called CTX-712 in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to treatment. The drug is taken by mouth once or twice weekly. The trial aims to find the safest dose and see i…

This early-phase study tests whether specially engineered donor immune cells (TSC-100 or TSC-101) can safely reduce the risk of cancer returning after a stem cell transplant. About 75 adults with acute myeloid leukemia, myelodysplastic syndromes, or acute lymphoblastic leukemia w…

This study tests whether statins, common cholesterol-lowering drugs, can reduce inflammation and slow the progression of two blood disorders: CCUS and lower-risk MDS. These conditions can lead to more serious blood cancers or heart problems, and there are no approved treatments t…

This early-phase trial tests a new drug called iadademstat combined with a standard chemotherapy (azacitidine) in people with myelodysplastic syndrome (MDS), a bone marrow disorder. The main goal is to find the safest dose for future studies. About 12 adults with higher-risk MDS …

This early-phase trial tests whether adding the drug momelotinib to standard azacitidine treatment can better control rare blood cancers like chronic myelomonocytic leukemia and chronic neutrophilic leukemia. About 18 adults will take momelotinib pills daily plus azacitidine inje…

This study tests a new oral drug called enzomenib (DSP-5336) in people with acute leukemia and certain other blood cancers that have not responded to standard treatments. The trial has two phases: first, finding the safest dose, and then checking how well it works alone or combin…

This early-phase trial tests whether giving patients extra gamma delta T-cells—a type of immune cell that can kill cancer—after a half-matched bone marrow transplant can improve outcomes. The study includes up to 38 adults with leukemia or myelodysplastic syndromes. The goal is t…

This study tests whether giving a two-drug combination (decitabine and cedazuridine, or DEC-C) early can prevent myelodysplastic syndromes (MDS) from coming back after a stem cell transplant. About 209 adults with MDS who have tiny amounts of cancer cells left after transplant wi…

This early-phase trial is testing whether combining two drugs—decitabine/cedazuridine (already approved for MDS) and the experimental drug defactinib—is safe and tolerable for people with high-risk myelodysplastic syndromes, certain acute myeloid leukemias, and chronic myelomonoc…

This study tests whether giving two drugs, decitabine and filgrastim, after a stem cell transplant can help prevent blood cancers like AML and MDS from coming back in children and young adults. About 37 participants will receive the drug combination for up to 6 cycles. The main g…

This phase 2 trial tests a reduced-intensity chemotherapy regimen (fludarabine, busulfan, melphalan) before stem cell transplant in 61 patients aged 55 and older with myeloid cancers like AML, MDS, or CMML. The goal is to see if this gentler approach can improve disease-free surv…

This study tests whether adding high-dose vitamin C to a standard chemotherapy drug (azacitidine) can improve outcomes for adults with myelodysplastic syndrome (MDS), a bone marrow disorder. About 38 people with higher-risk MDS who haven't had much prior treatment will receive th…

This study tests a drug called olutasidenib in people with certain early blood conditions (CCUS, lower-risk MDS, or CMML) that have a specific IDH1 gene change. The goal is to see if the drug can control the disease and is safe. About 15 participants will take the drug and be mon…

This early-stage trial is testing whether adding two drugs, decitabine and venetoclax, to a standard reduced-intensity stem cell transplant can safely help control high-risk myeloid cancers like acute myeloid leukemia. About 20 adults aged 18-75 with a matched donor will receive …

This study tests a combination of two drugs—luspatercept and erythropoietin (EPO)—for people with a low-risk form of myelodysplastic syndrome (MDS) that does not involve ring sideroblasts. Participants must have already tried EPO alone without success or be unable to take it. The…

This study tests a daily pill called olutasidenib given after a stem cell transplant to people with certain blood cancers (like AML or MDS) that have an IDH1 mutation. The goal is to see if the drug is safe and tolerable as maintenance therapy. About 31 participants will take the…

This early-phase trial is testing a new drug called AZD2962, which blocks a protein called IRAK4 involved in cancer growth. The study includes about 72 people with certain blood cancers (myelodysplastic syndromes or chronic myelomonocytic leukemia) that have not responded to prio…

This study tests whether using stem cells from both a half-matched family donor and a cord blood donor can improve survival without cancer returning. It is for people with certain high-risk blood cancers like leukemia or myelodysplastic syndromes. The approach aims to control the…

This Phase 2 trial tests a once-daily pill called AND017 for anemia caused by lower-risk myelodysplastic syndromes (MDS). About 63 participants who do not need frequent transfusions will take the drug to see if it can raise hemoglobin levels or reduce the need for blood transfusi…

This study tests whether giving children with leukemia a special type of donor immune cell (CD45RA-depleted) after a stem cell transplant can help their immune system recover faster and lower the risk of serious viral or fungal infections. The trial enrolls 50 children under 25 w…

This study tests a drug called elritercept (KER-050) in 160 adults with a bone marrow condition called myelodysplastic syndromes (MDS) who have anemia. The main goals are to check the drug's safety and how well people tolerate different doses. Researchers will also see if it help…

This phase 2 trial tests a personalized approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Instead of a one-size-fits-all chemotherapy and radiation regimen, doctors adjust the doses based on each patient's risk. The goal is to improve the…

This study is observing how well a double umbilical cord blood transplant works for 40 adults with blood cancers like leukemia or lymphoma. The transplant uses two units of cord blood from unrelated donors to help rebuild the patient's blood system. Researchers are tracking survi…

This early-phase study tests a new drug called RJK-RT2831 in 71 adults with blood cancers. The main goals are to check safety, find the right dose, and see if the drug shows any early signs of working. Participants must be 18 to 74 years old and in good enough health to follow th…

This phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…

This early-phase trial is testing whether adding the targeted drug venetoclax to standard chemotherapy can safely treat adults newly diagnosed with acute myeloid leukemia or advanced myelodysplastic syndrome. About 99 participants will receive different doses of venetoclax alongs…

This phase 2 trial tests a low-dose weekly schedule of two drugs, decitabine and venetoclax, in people with myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), or chronic myelomonocytic leukemia (CMML). The goal is to see if this gentler approach reduces treatment dela…

This early-phase study tests a combination of two drugs, selumetinib and azacitidine, in adults with higher-risk blood cancers like MDS and myelofibrosis. The main goal is to find the safest dose of selumetinib when given with standard azacitidine and to monitor side effects. Par…

This early-stage trial is testing a new oral drug called SX-682, alone or with the chemotherapy drug decitabine, in people with myelodysplastic syndrome (MDS) — a bone marrow disorder. The main goal is to find a safe dose and check for side effects. About 151 adults whose MDS has…

This study tests whether giving extra natural killer (NK) cells from a donor can safely help prevent leukemia or MDS from coming back after a stem cell transplant. About 10 adults with acute myeloid leukemia or myelodysplastic syndromes who don't have a fully matched donor will r…

This study tests whether a tailored exercise program combined with a dietary supplement called nicotinamide riboside (NR) can improve muscle mass, strength, and fitness in adolescents and young adults who had a bone marrow transplant for leukemia or other blood disorders. About 8…

This phase 2 trial tests a combination of venetoclax (a targeted drug), standard chemotherapy (azacitidine or decitabine), and an infusion of immune cells from a partially matched donor for people with intermediate- or high-risk myelodysplastic syndromes (MDS) who cannot or choos…

This early-stage study tests a drug called danvatirsen, first by itself and then combined with venetoclax, in 38 adults with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The goal is to see if the treatment is safe and can shrink or contr…

This early-phase study tests a new stem cell transplant method for people with aggressive or hard-to-treat acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The approach uses specially engineered donor cells to reduce the risk of graft-versus-host disease, potentia…

This phase 2 trial tests whether combining interferon-gamma (a protein that boosts the immune system) with donor immune cells can help patients whose acute myeloid leukemia or myelodysplastic syndromes have returned after a stem cell transplant. The study will enroll 45 adults an…

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 2 trial tests whether a new way of giving anti-thymocyte globulin (ATG) along with standard drugs tacrolimus and methotrexate can prevent acute graft-versus-host disease (GVHD) after a stem cell transplant. The study enrolls 29 adults aged 18-60 with blood cancers who …

This early-stage trial tests a new cell therapy called MNV-201 for people with low-risk myelodysplastic syndrome (MDS), a bone marrow disorder that causes low blood counts. The treatment uses the patient's own blood stem cells, which are mixed with mitochondria from donated place…

This early-phase trial tests an experimental oral drug called BH-30236 in adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study first tests BH-30236 alone, then in combination with another drug, venetoclax. About 17…

This study tests whether adding the targeted drug venetoclax to standard chemotherapy before a stem cell transplant can help prevent cancer from returning in people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). About 138 participants aged 12 to 6…

This study compares two strategies for people with higher-risk myelodysplastic syndrome (MDS): getting a donor stem cell transplant right away versus receiving one to two cycles of chemotherapy first (called bridging therapy) before the transplant. The goal is to see if immediate…

This study tests whether a fecal microbiota transplant (FMT) can prevent graft-versus-host disease (GvHD) in 150 adults with blood cancers who receive a stem cell transplant. Patients get a single FMT enema within four weeks of transplant. The main goal is to see if FMT improves …

This study tests whether tranexamic acid (TXA), a drug that prevents blood clots from breaking down, can safely reduce bleeding in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). About 75 outpatients receiving less-intensive chemotherapy will be rando…

This study tests whether giving blood transfusions at home is practical and acceptable for cancer patients who need regular transfusions. About 30 adults with blood cancers or bone marrow failure will receive 1-5 blood product units at home. Researchers will track how often trans…

This study looks at whether non-chemo approaches like physical activity, intermittent fasting, or adding a beta-blocker (propranolol) can improve immune function and quality of life in people with multiple myeloma or related blood disorders. Researchers will measure changes in im…

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 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 gathers blood and bone marrow samples from 150 adults with myelodysplastic syndrome (MDS), a blood disorder that can turn into acute leukemia. Researchers want to find biological markers that help predict which patients are likely to progress to leukemia. The goal is t…

This study collects blood, bone marrow, skin, saliva, and stool samples from up to 1,000 healthy volunteers aged 18 and older. The samples will be stored in a database for researchers to compare with samples from people who have myelodysplastic syndromes (MDS), a group of blood d…

This study follows people with myelodysplastic syndromes (MDS) for up to 20 years to understand how the disease changes over time. Researchers will collect blood, bone marrow, and other samples from 1,100 participants, including healthy donors. The goal is to learn more about MDS…

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 uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

This study looks at genetic changes in people with early-stage blood cancers or conditions that may lead to cancer. Researchers will analyze blood, bone marrow, or cheek cell samples from up to 10,000 participants to find DNA errors linked to cancer risk and progression. The goal…

This study is collecting information from 260 children and teens with rare blood cancers called myelodysplastic syndromes (MDS) and juvenile myelomonocytic leukemia (JMML). Researchers are using advanced genetic tests to improve how these diseases are diagnosed and classified. Th…

This study follows people with a gene change (DDX41) that raises the risk of blood cancers like MDS and AML, along with their family members. Researchers will collect health information and samples over 10 years to learn how these inherited risks lead to cancer. No treatment is g…

This study looks at 60 adults with acute myeloid leukemia or related blood cancers who are receiving a gentler type of chemotherapy. Researchers will measure certain proteins in the blood to see if they can predict how well the treatment works. The goal is to better understand wh…

This study gathers basic health information from 2000 people receiving stem cell transplants for blood cancers or immune disorders. The goal is to track survival and complications to help doctors and researchers improve future treatments. Participants must be getting a standard t…

This study tests a telehealth program that helps older adults (60+) with blood cancers like leukemia or MDS talk to their doctors about their care wishes. The program also helps them fill out advance directive forms. Researchers want to see if it improves communication and reduce…

This study looks at whether detecting MDS stem cells using a special lab test (flow cytometry) can predict if the disease will come back after a stem cell transplant. Researchers will follow 163 MDS patients for one year after transplant to see if this method is better than curre…

This study looks at how services from Blood Cancer United (formerly The Leukemia & Lymphoma Society) affect people with blood cancers like leukemia, lymphoma, and myeloma. Researchers will track 400 patients over time to see if the organization helps with access to care, quality …

This study is collecting genetic and drug sensitivity data from 300 children with leukemia to better understand why some cases relapse and how to target them more precisely. Researchers will analyze tumor DNA, gene activity, and test drugs on patient cells in the lab. The goal is…

This study is looking at whether certain carbohydrates in food, called FODMAPs, are related to graft-versus-host disease (GVHD) in people who have had a bone marrow transplant. GVHD is a common complication where donor cells attack the patient's body. Researchers will track 100 t…

This study looks at whether a new whole genome test called ChromoSeq can find genetic changes in people with myelodysplastic syndromes (MDS) as well as current standard tests. About 60 adults with MDS will provide samples, and doctors will compare results. The goal is to see if C…

This study creates a registry of 5,000 adults aged 50 and older with blood cancers like multiple myeloma and lymphoma. Researchers will track frailty, muscle loss, and other age-related health issues to better understand this group's needs. The goal is to gather information that …

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 testing whether an artificial intelligence (AI) tool can help match cancer patients with the right clinical trials. Researchers will enroll up to 50,000 people with various cancers to see if the AI improves trial enrollment rates and patient outcomes. The goal is to…

This study is testing a new whole genome sequencing approach called ChromoSeq for patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Researchers want to see if ChromoSeq can find more genetic changes than current standard tests, and if it can be used in…