MYELODYSPLASTIC SYNDROMES

This early-stage trial tests an oral drug called AG-120 in people with advanced blood cancers, like 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 drug daily un…

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 early-phase trial tests whether specially trained natural killer (NK) cells, called CIML NK cells, given with interleukin-2 can safely prevent relapse in people with high-risk acute myeloid leukemia, myelodysplastic syndrome, or related blood cancers after a stem cell transp…

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 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 early-phase study tests whether adding hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) to standard stem cell transplant conditioning is safe and helpful. It involves up to 24 adults with certain blood cancers or bone marrow disorders. The goal is t…

This phase 2 trial is testing a treatment called Orca-T for people with acute myeloid leukemia or myelodysplastic syndrome. Orca-T is made from a donor's blood and contains stem cells plus special immune cells designed to help the transplant work better and reduce complications. …

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 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 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 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 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 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 study tests an oral cancer drug (decitabine and cedazuridine) in 18 adults with acute myeloid leukemia, myelodysplastic syndromes, or solid tumors. Half have severe kidney impairment, half have normal kidneys. Researchers measure how the drug moves through the bo…

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 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 study tests whether elritercept can help adults with a bone marrow disease called MDS who need regular red blood cell transfusions. About 225 participants will receive either the drug or a placebo to see if it reduces or eliminates transfusion needs for at least 8 weeks. The…

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 early-stage study is testing an experimental drug called JNJ-95804306 in people with blood cancers (like leukemia) that have returned or not improved after standard treatments. The main goals are to check the drug's safety, find the best dose, and see if it can shrink tumors…

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 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 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 early-stage trial tests whether adding two drugs, decitabine and venetoclax, to a standard reduced-intensity stem cell transplant is safe for people with high-risk blood cancers like leukemia. The study involves 20 adults aged 18-75 who have a matched donor. The main goal is…

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 is testing a new drug called venetoclax combined with standard chemotherapy for adults with newly diagnosed acute myeloid leukemia (AML) or advanced myelodysplastic syndrome (MDS). The goal is to find the safest dose and see how well the combination works. About 99 par…

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 program offers galinpepimut-S, an experimental immune therapy, to patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who have run out of standard treatment options. It is designed for single patients who cannot join a clinical trial and whose doctor…

This study tests a treatment for people with intermediate- to high-risk myelodysplastic syndromes (a bone marrow disorder) who cannot have a standard stem cell transplant. The treatment combines a drug called venetoclax with another drug and an infusion of immune cells from a par…

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

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 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 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 an experimental drug called elritercept (KER-050) in 160 adults with a bone marrow condition called myelodysplastic syndromes (MDS) who have low red blood cell counts (anemia). The main goal is to check the drug's safety and find the best dose. Researchers will a…

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 whether a new way of giving three drugs (ATG, tacrolimus, and methotrexate) can prevent acute graft-versus-host disease (GVHD) in people receiving a stem cell transplant for blood cancers like leukemia. About 29 adults aged 18-60 will take part. The goal is to se…

This study tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The approach removes certain immune cells from the donor cells to lower the chance of graft-versus-host disease and reduce the need for long-term immune-su…

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 study tests a new way to prepare stem cell transplants for people with certain blood cancers like leukemia and lymphoma. The method removes specific immune cells from the donated stem cells to lower the risk of a serious complication called graft-versus-host disease. About 2…

This study tests a treatment for people whose acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) has come back after a stem cell transplant. The treatment combines a protein called interferon-γ with donor immune cells. The goal is to see if this combination can contr…

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 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 early-phase study tests a new treatment for adults with acute myeloid leukemia or myelodysplastic syndrome who still have detectable cancer cells after standard therapy. The treatment uses the patient's own blood cells, which are armed with a special antibody to better targe…

This early-stage study tests a new drug, SX-682, alone or with another 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 who have not responded to prior treatm…

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 early-stage study tests whether giving extra immune cells (gamma delta T-cells) after a half-matched bone marrow transplant can help kill cancer cells while reducing the risk of graft-versus-host disease. About 38 adults with certain blood cancers or pre-cancers will take pa…

This study tests a drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers. 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 8 years, with r…

This early-phase study tests a new oral drug, GLB-001, in about 48 adults whose acute myeloid leukemia or high-risk myelodysplastic syndromes have returned or stopped responding to standard therapy. The main goals are to check safety, find the best dose, and see if the drug can h…

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 study tests a new drug, enzomenib, in adults with acute leukemia and other blood cancers that have come back or not improved with standard treatments. The goal is to find a safe dose and see if the drug can shrink or control the cancer. About 606 people will take part in thi…

This study tests two different drug combinations to prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body after a stem cell transplant. About 400 adults with blood cancers like leukemia or lymphoma will receive either thymogl…

This study tests an experimental drug called CTX-712 in adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) that have come back or not responded to prior treatments. The goal is to find a safe dose and see if the drug can shrink or control the ca…

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 study tests an experimental drug called BGB-11417 for people with acute myeloid leukemia (AML) and related bone marrow disorders. The drug works by blocking a protein that helps cancer cells survive. The trial will find the safest dose and see if the drug, alone or with anot…

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 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 study is for people with myelodysplastic syndromes (MDS) whose disease has returned or not responded to standard treatment. Researchers are testing a combination of two drugs, leflunomide and decitabine, to find the safest and most effective doses. The goal is to control the…

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 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 study tests an oral drug (ASTX030) that combines two medicines to treat certain blood cancers like myelodysplastic syndromes and acute myeloid leukemia. The goal is to see if taking a pill works as well as getting a shot, with fewer trips to the clinic. About 316 adults will…

This study tests whether adding venetoclax to the standard chemotherapy before a stem cell transplant can help people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) stay cancer-free longer. About 138 patients aged 12 to 60 will be randomly assigned…

This early-stage study tests a new drug called iadademstat combined with a standard chemotherapy (azacitidine) for 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 study looks at whether getting a stem cell transplant right away is as good as getting other treatments first and then the transplant for people with high-risk myelodysplastic syndrome (MDS). About 236 adults will be randomly assigned to one of two groups. The goal is to see…

This study tests a new way of giving two approved drugs (5-azacitidine and decitabine) to people with myelodysplastic syndromes (MDS) or related bone marrow disorders. Instead of standard high doses, the drugs are given in a low-dose, alternating schedule to reduce side effects a…

This study tests whether adding a specific type of donor cell (CD45RA-depleted) can help children recover their immune system faster after a stem cell transplant for blood cancers. The goal is to lower the risk of serious viral or fungal infections without increasing graft-versus…

This study tests whether adding high-dose vitamin C to a standard chemotherapy drug (azacitidine) can help adults with a serious bone marrow disorder called myelodysplastic syndrome (MDS). About 38 people with higher-risk MDS will receive the combination intravenously. The goal i…

This study tests a drug called luspatercept in 40 adults with lower-risk myelodysplastic syndromes (MDS), a bone marrow condition that often requires frequent blood transfusions. The goal is to see if luspatercept can reduce the number of transfusions needed. Participants will re…

This study tests a reduced-intensity chemotherapy combination (fludarabine, busulfan, melphalan) given before a stem cell transplant in people aged 55 and older with myeloid cancers like AML, MDS, or CMML. The goal is to see if this gentler approach can help patients stay free 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 is testing whether giving patients a fecal transplant (a stool sample from a healthy donor, delivered as an enema) after a stem cell transplant can prevent graft-versus-host disease (GvHD), a common and serious complication. The trial will enroll 150 adults with blood …

This study tests a new program that brings blood transfusions to the homes of people with advanced blood cancers who are in hospice care. The goal is to see if this improves their quality of life, mood, and reduces hospital visits near the end of life. About 700 patients will tak…

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 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 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 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 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 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 how the body breaks down certain carbohydrates (FODMAPs) in the gut and whether that affects graft-versus-host disease (GVHD) after a bone marrow transplant. Researchers will follow 100 transplant patients to track when and how severe GVHD occurs. The goal is …

This study looks at whether a new whole genome sequencing test called ChromoSeq can provide better genetic information for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) compared to standard tests. Researchers will collect samples and surveys from 325 …

This study collects samples like blood, bone marrow, skin, saliva, and stool from 1,000 healthy adults aged 18 and older. The samples are used as a comparison to better understand myelodysplastic syndromes (MDS), a group of blood stem cell disorders that can lead to cancer. Parti…

This study aims to improve treatment for childhood leukemia, the most common pediatric cancer. Researchers will analyze genetic changes and test drug sensitivity in samples from 300 children with various types of leukemia. The goal is to find safer, targeted therapies for high-ri…

This study is observing how safe and effective a double umbilical cord blood transplant is for Korean adults with blood cancers like leukemia and lymphoma. About 40 participants will receive either a mild or strong pre-transplant treatment, then the transplant. Researchers will t…

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 is for children and teens under 18 with myelodysplastic syndromes (MDS) or juvenile myelomonocytic leukemia (JMML). Researchers want to improve how these rare blood cancers are diagnosed by using a standard set of lab tests and genetic analysis. The study does not prov…

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 is for people with myelodysplastic syndromes (MDS) who are getting a stem cell transplant. Researchers want to see if measuring certain stem cells in the bone marrow can better predict if the disease will come back after transplant. About 163 participants will be follo…

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 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 aims to learn more about myelodysplastic syndromes (MDS) by following people with the condition for up to 20 years. Researchers will collect blood, bone marrow, and other samples to better understand how MDS progresses. Up to 1,100 participants, including healthy donor…

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 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 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 collects blood, bone marrow, and other samples from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze genes to find causes of these diseases and why some people get sicker than others. The goal is to improve future …

This study looks at how anemia affects brain function, especially thinking and memory. Researchers will use a non-invasive light-based brain scan (fNIRS) to measure brain activity in 323 adults with different types of anemia. The goal is to find which brain areas are affected and…