MYELODYSPLASTIC SYNDROMES

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome who also have anemia. The goal is to see if the drug is safe and can help patients need fewer or no blood transfusions. The study is in early to mid stages an…

This study tests a drug called olutasidenib in 15 people with specific blood conditions (CCUS, lower-risk MDS, or CMML) that have an IDH1 mutation. The goal is to see if the drug can help control the disease and to check for side effects. Participants must have a confirmed diagno…

This study tests a drug combination (venetoclax plus a hypomethylating agent) followed by an infusion of immune cells from a partially matched donor for people with intermediate- to high-risk myelodysplastic syndromes who cannot have a standard stem cell transplant. The goal is t…

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 placenta tissue. T…

This early-stage study tests a new daily pill, BH-30236, in adults whose acute myeloid leukemia or high-risk myelodysplastic syndrome has returned or stopped responding to other treatments. The main goals are to check safety, find the right dose, and see if the drug can shrink ca…

This early-phase study tests whether adding an experimental drug (defactinib) to an approved oral treatment (decitabine/cedazuridine) is safe and tolerable for people with high-risk myelodysplastic syndromes, certain acute myeloid leukemias, and chronic myelomonocytic leukemia. A…

This study looks at how the body handles the cancer drug ASTX727 in people with severe kidney impairment compared to those with normal kidneys. It includes adults with certain blood cancers or solid tumors. The goal is to understand safety and drug levels, not to cure the disease…

This study tests a new drug called AND017 for anemia caused by a bone marrow disorder called lower-risk myelodysplastic syndromes (MDS). About 63 adults with MDS who need few or no blood transfusions will receive different doses of AND017 to see if it can raise hemoglobin levels …

This study tests whether giving two drugs (decitabine and filgrastim) after a stem cell transplant can safely prevent cancer from coming back in children and young adults with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or related blood cancers. About 37 partic…

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 whose MDS has not responded to prior t…

This early-stage study tests a new drug called danvatirsen, first alone and then combined with venetoclax, in adults with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to standard treatments. The goal is to see if the treatme…

This study tests a special stem cell transplant that removes certain immune cells to lower the need for strong anti-rejection drugs. It is for people with inherited bone marrow failure disorders like Fanconi anemia. The goal is to help the immune system recover faster and reduce …

This early-phase 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 of iadademstat for future studies. About 12 adults with …

This study is testing a new drug called BGB-11417 for people with certain blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The goal is to find the safest dose and see if the drug works on its own or when combined with another cancer medic…

This early-phase study tests a new treatment for adults with acute myeloid leukemia or myelodysplastic syndrome who still have small amounts of cancer cells after standard therapy. The treatment uses a patient's own blood cells that are specially armed with a bispecific antibody …

This study tests a new automated method to select specific stem cells (CD34+) for transplant in people with blood cancers like leukemia or lymphoma. The goal is to lower the risk of graft-versus-host disease, a serious complication where donor cells attack the patient's body. Abo…

This early-phase study tests whether specially engineered donor immune cells (TSC-100 or TSC-101) can safely prevent blood cancers like AML, MDS, or ALL from coming back after a stem cell transplant. About 75 adults will receive either standard transplant care or these enhanced c…

This early-phase study tests whether specially trained immune cells (CIML NK cells) given after a stem cell transplant can safely prevent leukemia or related blood cancers from coming back. About 15 adults with high-risk acute myeloid leukemia, myelodysplastic syndrome, or relate…

This study is for people with myelodysplastic syndromes (MDS) who have had a stem cell transplant but still have tiny amounts of cancer cells left (called measurable residual disease). The goal is to see if giving a drug called DEC-C early can lower the chance of the cancer retur…

This study tests whether a new way of giving three drugs (ATG, tacrolimus, and methotrexate) can prevent a serious side effect called graft-versus-host disease (GVHD) after a stem cell transplant for blood cancers. The trial will enroll 29 adults aged 18-60 who have a matched don…

This study tests a drug called pacritinib for 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 control the disease. Participants take capsules twice daily for up to 8 years, with …

This study tests a new drug, venetoclax, added to standard chemotherapy for adults newly diagnosed with acute myeloid leukemia (AML) or advanced myelodysplastic syndrome (MDS). The goal is to find the safest and most effective dose. About 99 participants will receive the combinat…

This early-stage study tests a new oral drug, GLB-001, in about 48 adults whose acute myeloid leukemia or high-risk myelodysplastic syndromes have returned or not responded to other treatments. The main goals are to check safety, find the best dose, and see early signs of effecti…

This study looks at how well umbilical cord blood stem cells from unrelated donors help patients with serious blood cancers, immune problems, or genetic disorders. About 30 people will receive a transplant to see how quickly their blood cells recover and how often the transplant …

This study tests a combination of interferon-γ and donor immune cells for adults whose acute myeloid leukemia or myelodysplastic syndromes have returned after a stem cell transplant. The goal is to see if this treatment can control the disease and improve survival. About 45 parti…

This early-phase trial tests a special type of immune cell (CIML NK cells) combined with a low dose of IL-2 in people whose blood cancer (AML, MDS, MPN, or JMML) has returned after a stem cell transplant. The main goal is to find the safest dose and see if it can put the cancer b…

This study tests an experimental 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 …

This study tests whether adding high-dose vitamin C to a standard drug (azacitidine) can improve outcomes for adults with myelodysplastic syndrome (MDS), a bone marrow disorder. About 38 participants with higher-risk MDS will receive the combination intravenously. The goal is to …

This early-phase study tests a new way to do stem cell transplants for people with aggressive or returning acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The approach uses specially processed donor cells to reduce the risk of graft-versus-host disease, aiming to…

This study tests a new treatment called Orca-T for people with acute myeloid leukemia or myelodysplastic syndrome. Orca-T is made from a donor's blood cells and is given after a gentle chemotherapy or radiation preparation. The goal is to see if it helps the body accept the trans…

This early-phase study is testing a new way to prepare donor stem cells, called Orca-Q, for people with certain blood cancers (like leukemia) who need a stem cell transplant. The goal is to see if this engineered graft is safe and helps the body accept the new cells without sever…

This early-stage trial 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 a safe dose and understand side effects. About 130 adults will receive th…

This study tests a low-dose weekly combination of two FDA-approved drugs, decitabine and venetoclax, for people with bone marrow cancers like MDS, AML, and CMML. The goal is to see if this gentler schedule can keep patients on treatment longer with fewer interruptions. About 91 a…

This study looks at how the cancer drug ASTX727 (decitabine and cedazuridine) is processed by the body 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 find safe dosin…

This study is for children under 25 who have had a stem cell transplant for blood cancers like leukemia. After transplant, their immune system is weak, putting them at high risk for dangerous infections. Researchers are adding back a specific type of donor cell (CD45RA-depleted) …

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

This study tests whether adding luspatercept to standard EPO therapy helps low-risk MDS patients without ring sideroblasts who did not respond to or cannot take EPO alone. About 150 adults will join this phase 1/2 trial to find the best dose and see if the combination works bette…

This study tests whether adding venetoclax to the standard chemotherapy given before a stem cell transplant can help prevent the cancer from coming back in people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). About 138 participants aged 12 to 60 …

This study tests whether using stem cells from both a half-matched family donor and a cord blood donor can improve survival for people with high-risk blood cancers like leukemia or myelodysplastic syndromes. About 36 participants will receive the transplant and be followed for up…

This study tests whether a tailored exercise program combined with a vitamin-like supplement (NAD+ precursor) can improve muscle mass, strength, and fitness in teens and young adults who had a bone marrow transplant for leukemia or other blood disorders. Participants will be rand…

This early-stage trial tests a cell therapy called Orca-T combined with standard drugs to prevent graft-versus-host disease (GVHD) in up to 24 adults with advanced blood cancers like leukemia or MDS. Participants receive a stem cell transplant from a donor who is only a 7 out of …

This early-phase study tests whether adding the drug momelotinib to standard treatment (azacitidine) can better control rare blood cancers like chronic myelomonocytic leukemia and chronic neutrophilic leukemia. About 18 adults will take the combination for up to 24 months. The go…

This early-stage study tests whether adding two drugs, decitabine and venetoclax, to a standard stem cell transplant can make the procedure safer for people with aggressive blood cancers like leukemia. About 20 adults aged 18-75 with a matched donor will receive the experimental …

This study tests an experimental drug, enzomenib, in people with acute leukemia and other blood cancers that have not responded to standard treatments. The goal is to find a safe dose and see if the drug can shrink or control the cancer. About 600 participants will take part in t…

This study tests an oral drug (ASTX030) that combines two medicines to treat certain blood cancers like MDS and AML. The goal is to see if taking a pill works as well as getting a shot, and if it can be safely combined with another drug (venetoclax) for AML. About 316 adults will…

This study is for people with certain blood cancers (like leukemia) who are getting a half-matched bone marrow transplant. Researchers are adding a dose of specially grown immune cells (gamma delta T-cells) after the transplant to see if it can kill any leftover cancer cells whil…

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

This study tests a stem cell transplant using a milder chemotherapy combination (treosulfan and fludarabine) followed by donor stem cells and drugs to prevent graft-versus-host disease. It includes 132 people aged 2-75 with blood cancers like AML or MDS. The goal is to see if thi…

This study is for people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who have received a stem cell transplant from a half-matched donor. After the transplant, participants will receive extra immune cells called natural killer (NK) cells from the same dono…

This study tests an investigational drug called elritercept in adults with a bone marrow disorder (MDS) who need regular red blood cell transfusions. The main goal is to see if the drug can help patients go at least 8 weeks without a transfusion. About 225 participants will recei…

This study tests a rotating combination of four drugs (cladribine, cytarabine, venetoclax, and azacitidine) to see if it can help control higher-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). It is for adults aged 18 and older whose cancer has not …

This study tests a drug called momelotinib in 80 adults with low-risk myelodysplastic syndromes (MDS) who have anemia. The goal is to see if the drug can help them go without red blood cell transfusions for at least 12 weeks. The trial has two parts: first to find the best dose, …

This study looks at whether getting a stem cell transplant right away is as good as getting some treatment first and then the transplant for people with higher-risk myelodysplastic syndrome (MDS). About 236 adults who are eligible for a transplant will be randomly assigned to one…

This study tests whether tranexamic acid, a medicine that helps blood clots stay in place, can reduce bleeding in people with myelodysplastic syndromes or acute myeloid leukemia who are on chemotherapy. About 75 adults with low platelet counts will be randomly assigned to receive…

This study looks at whether giving blood transfusions at home is practical and acceptable for people with blood cancers or bone marrow failure who need regular transfusions. About 30 adults will receive 1-5 blood product units at home, and researchers will track how often transfu…

This study tests if the drug luspatercept can help people with lower-risk myelodysplastic syndromes (MDS) need fewer blood transfusions. About 40 adults will receive the drug, and researchers will track side effects and transfusion needs. The goal is to improve quality of life by…

This study tests a program that gives blood cancer patients access to blood transfusions at home while in hospice care. The goal is to see if this improves quality of life, mood, and reduces hospital visits near the end of life. About 700 adults with advanced blood cancers like l…

This study tests if a special blood test that looks for MDS stem cells can better predict whether the disease will come back after a stem cell transplant. Researchers will follow 163 people with MDS who receive a transplant. The goal is to improve how doctors monitor for remainin…

This study looks at genetic changes in people with early-stage blood cancers or conditions that may lead to cancer. Researchers want to understand why some cases progress and others don't. Participants provide extra blood, bone marrow, or cheek swab samples during routine care. N…

This study looks at whether a new whole genome sequencing test called ChromoSeq can be used alongside standard genetic tests for people with myelodysplastic syndrome (MDS). Researchers will compare how well ChromoSeq finds genetic changes compared to current methods. About 60 adu…

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 follows people with inherited gene changes (DDX41) that raise the risk of blood cancers like MDS and AML. Researchers will track health over 10 years using exams, blood tests, and optional bone marrow biopsies. No treatment is given; the goal is to understand how these…

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. The study does not offer a new treatment but will…

This study is gathering basic health and survival information from 2000 people who are getting a stem cell transplant for blood cancers or immune diseases. The goal is to build a database that doctors can use to improve care and answer future questions. No experimental treatments…

This study is testing whether an artificial intelligence tool can help match cancer patients to clinical trials they might be eligible for. Researchers will enroll up to 50,000 people with various types of cancer and use the AI to suggest trials. The goal is to see if this approa…

This study collects blood, bone marrow, and other samples from 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 diagnosis an…

This study looks at whether a new whole genome sequencing test called ChromoSeq can find more genetic changes in people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) than current standard tests. Researchers will compare ChromoSeq results to conventional meth…

This study looks at whether measuring certain proteins in the blood can help predict outcomes for people with acute myeloid leukemia, high-risk myelodysplastic syndrome, or chronic myelomonocytic leukemia who receive a gentler type of chemotherapy. Researchers will collect blood …

This study tests a telehealth program to help older patients (60+) with blood cancers like leukemia and MDS communicate better with their doctors and complete advance care plans. About 207 patients and their caregivers will be randomly assigned to the program or usual care. The g…

This study collects blood, bone marrow, skin, saliva, and stool samples 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. Participan…

This study collects blood, bone marrow, and tissue samples from up to 15,000 people with blood cancers or related disorders. The samples are stored in a central bank for researchers to use in future studies. Participants do not receive any treatment as part of this study. The goa…