Myelodysplastic syndrome

Researchers are testing whether adding abatacept to standard immune-suppressing drugs can prevent severe graft-versus-host disease after a double umbilical cord blood transplant. The trial enrolls people with blood cancers such as leukemia, lymphoma, and myelodysplastic syndromes…

This early-stage trial is testing an experimental cell therapy for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) that has returned or not responded to standard treatments. The therapy uses a patient's own immune cells, modified to recognize …

This trial tests a new, lower-intensity combination of chemotherapy and radiation before a donor stem cell transplant for people with high-risk blood cancers or aplastic anemia. The goal is to see if this approach is safe enough to allow the donor cells to take hold and cure the …

This phase II trial tests a vaccine designed to protect stem cell transplant recipients from cytomegalovirus (CMV), a common virus that can cause serious illness in people with weakened immune systems. The vaccine uses a modified, harmless virus to deliver three CMV proteins, tra…

This study tests a vaccine called Triplex to prevent cytomegalovirus (CMV) infection in adults who have received a stem cell transplant. CMV is usually harmless but can be life-threatening for people with weakened immune systems. The vaccine uses harmless virus pieces to train th…

This early-stage trial tests a new antibody drug called Hu8F4 in 72 people with advanced blood cancers like leukemia and myelodysplastic syndrome. The goal is to find the safest dose and see if it can slow or stop cancer growth. Researchers will also track how long patients live …

This study tests a new drug, seclidemstat, combined with a standard drug, azacitidine, for people with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The goal is to find the best dose and see if the combination can shrink or control the cancer. About 2…

Researchers are observing how well eltrombopag works in adults with hypocellular myelodysplastic neoplasms, a bone marrow condition that lowers blood cell counts. The study follows patients in Italy who receive eltrombopag alone or with immunosuppressive therapy as part of routin…

This study tests an experimental drug, HT-6184, in adults with a bone marrow disorder called myelodysplastic syndrome (MDS) who have low red blood cell counts (anemia). The goal is to see if the drug can improve blood counts and reduce the need for transfusions. Participants take…

This study offers continued RVU120 treatment to up to 10 patients with advanced solid tumors, acute myeloid leukemia, or high-risk myelodysplastic syndrome who are already benefiting from the drug in an earlier trial. The main goal is to monitor safety and see how long patients c…

This early-phase study is testing a drug called TAK-243 in people whose acute myeloid leukemia or myelodysplastic syndrome has come back or is not responding to treatment. The goal is to find the best dose and check for side effects. The trial is currently on hold.

This study tests a new way to treat children and young adults with high-risk blood cancers like leukemia and lymphoma. Doctors take blood cells from a family donor, remove certain T cells that can cause a harmful immune reaction, and then transplant the modified cells. The goal i…

This phase 2 trial tests a combination of two immunotherapy drugs (nivolumab and ipilimumab) plus a chemotherapy drug (azacitidine) in 99 people with myelodysplastic syndromes (MDS), a type of blood cancer. The goal is to see if the combination improves response rates, especially…

This early-phase trial tests a personalized cell therapy called CART123 for people with certain blood cancers (like AML or MDS) that have not responded to standard treatments. Each patient's own immune cells are modified in a lab to recognize and attack cancer cells. The main goa…

This study was designed to test whether adding olutasidenib to standard chemotherapy drugs could help control certain blood cancers in people with an IDH1 gene mutation. The trial was withdrawn before enrolling any participants, so no results are available.

This early-stage trial tests a new drug called DFV890 in about 62 adults with low-risk myelodysplastic syndromes, low-risk chronic myelomonocytic leukemia, or high-risk clonal cytopenia of undetermined significance. The main goals are to check the drug's safety, find the best dos…

This phase 2 trial tests the drug olaparib in 14 adults with acute myeloid leukemia or myelodysplastic syndrome that has returned or not responded to treatment, and whose cancer has a specific change in the IDH gene. Olaparib is a pill that may block enzymes cancer cells need to …

This early study is testing whether bone marrow from deceased donors can be safely used for transplants in people with blood cancers like leukemia and lymphoma. Twelve patients will receive the transplant along with chemotherapy or radiation. The main goal is to see if the new ma…

This early-phase trial tests a drug called lintuzumab-Ac225 in 18 adults with high-risk myelodysplastic syndrome (MDS) that did not improve after standard treatment. The drug is an antibody that seeks out cancer cells and delivers a radioactive payload to kill them. The main goal…

This early-stage trial tests whether adding the immunotherapy drug ipilimumab to the chemotherapy drug decitabine is safe and tolerable for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has come back or stopped responding to treatment. About 54 p…

This study compares the standard drug azacitidine alone versus azacitidine combined with either lenalidomide or vorinostat in 282 patients with higher-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The goal is to see if any combination improves re…

This early-phase study tests a new drug called SEA-CD70, alone or with standard drugs azacitidine and venetoclax, in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The main goal is to check safety and find the right dose. About 178 participants will b…

This study tests a combination of two drugs, venetoclax and ASTX727, in people with high-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) who haven't had treatment before. The goal is to find the safest dose and see if the combo can shrink or control …

This phase 3 trial compares two treatments for people with a bone marrow disorder called myelodysplastic syndrome (MDS) who have low red blood cell counts (anemia). One group gets lenalidomide alone, the other gets lenalidomide plus epoetin alfa, a drug that stimulates red blood …

This study tests a new way to deliver radiation before a stem cell transplant for people with leukemia or myelodysplastic syndrome. Instead of radiating the whole body, the new method targets only the bone marrow and lymph nodes, which may reduce damage to healthy organs. The goa…

This study monitors long-term safety in 120 cancer patients who are already benefiting from treatments like JNJ-75348780, safimaltib, or ibrutinib in earlier trials. Participants have blood cancers, solid tumors, or prostate cancer. The goal is to track serious side effects over …

This early-stage trial tests a new drug called JNJ-89853413 in 64 people whose acute myeloid leukemia (AML) or myelodysplastic neoplasms (MDS) have come back or stopped responding to treatment. The main goal is to check safety and find the right dose, with a second part looking a…

This trial tests whether a drug called cyclophosphamide (PTCY) can prevent graft-versus-host disease (GVHD) better than the current standard, ATG, in patients receiving stem cell transplants from unrelated donors. GVHD is a serious complication where donor immune cells attack the…

This phase 2 trial tests giving two chemotherapy drugs, clofarabine and melphalan, before a donor stem cell transplant in 72 patients with leukemia or myelodysplastic syndrome in remission. The goal is to prevent the cancer from coming back after the transplant while causing fewe…

This study tests an experimental drug called AG-946 (tebapivat) in 87 adults with lower-risk myelodysplastic syndromes (MDS) who have anemia. The goal is to see if the drug can raise hemoglobin levels or help patients go without blood transfusions for at least 8 weeks. Participan…

This early-phase trial is exploring whether combining two drugs, selinexor and azacitidine, can help prevent relapse in people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who carry a TP53 mutation and have already undergone a stem cell transplant…

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 3 trial is testing whether adjusting chemotherapy based on a child's early response can improve outcomes for acute myeloid leukemia (AML) or myelodysplastic syndrome in children with Down syndrome. After the first round of treatment, patients are placed into risk group…

This study tests whether adding venetoclax to ASTX727 (a two-in-one pill) works better than ASTX727 alone for people with chronic myelomonocytic leukemia or related bone marrow cancers with too many immature cells. About 132 adults will be randomly assigned to one of the two trea…

This study tests a pill called ruxolitinib for people whose immune system attacks their bone marrow, causing conditions like severe anemia. The drug aims to stop this attack and help the bone marrow recover. Participants take the pill twice daily for up to 6 months and are follow…

This study tests whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant from a partially matched donor, can reduce serious infections in the first 100 days while still preventing graft-versus-host disease (a common complication where donor cells att…

This study tests a new two-drug combination (pacritinib plus azacitidine) for people with moderate to very high risk myelodysplastic syndrome (MDS), a bone marrow disorder. The first part finds the safest dose of pacritinib, and the second part checks how well the combination wor…

This study tests a new treatment called Orca-T for people with advanced blood cancers like leukemia. It uses a special mix of stem cells and immune cells from a donor to help the body accept the transplant while reducing serious side effects like graft-versus-host disease. The go…

This study is testing a combination of two oral medications, Onureg and Venclyxto, in people with a high-risk form of myelodysplastic syndromes (a blood cancer) who cannot have a stem cell transplant. The goal is to find the safest and most effective dose of Onureg when used with…

This trial is testing an oral drug called AG-120 (ivosidenib) in adults with myelodysplastic syndrome (MDS) or a related early form of leukemia that carries a specific IDH1 gene mutation. The drug is designed to block the mutated enzyme that drives abnormal blood cell growth. Res…

This trial is testing an oral drug called enasidenib (AG-221) in people with myelodysplastic syndrome (MDS) who have a specific genetic change called an IDH2 mutation. The drug is designed to block the mutated enzyme and help restore normal blood cell production. Participants rec…

This early-phase trial tests a drug called DR-18 in 40 adults with acute myeloid leukemia or myelodysplastic syndrome whose cancer returned or persisted after a stem cell transplant. DR-18 is a modified immune protein designed to boost the body's natural defenses against cancer c…

This study tests two drugs, vorinostat and azacitidine, together in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to find the best dose and see if the combination is safe and effective. About 135 participants will take part in this early-…

This study tests a specific timing of chemotherapy drugs (busulfan and fludarabine) before a donor stem cell transplant, followed by cyclophosphamide after the transplant, to treat high-risk blood cancers like leukemia and lymphoma. The goal is to reduce the chance of the transpl…

This early-phase study tests a new way to deliver total body radiation before a stem cell transplant for people with acute leukemia or myelodysplastic syndrome. The goal is to reduce radiation to the lungs to prevent serious lung damage. Twenty participants will receive intensity…

This phase 3 trial compares two types of stem cell transplants for children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). One uses a half-matched family donor (haploidentical), the other a fully matched unrelated donor. The goal is to see w…

This early-phase trial tests a new targeted drug called pivekimab sunirine (PVEK) alongside a standard chemotherapy regimen (FLAG-Ida) in 30 adults with newly diagnosed, high-risk acute myeloid leukemia or related blood cancers. PVEK works like a smart bomb, attaching to a protei…

This study offers continued treatment with the drug ASTX727 to adults with acute myeloid leukemia, chronic myelomonocytic leukemia, or myelodysplastic syndromes who were already benefiting from it in earlier studies. The main goal is to monitor long-term safety. About 332 partici…

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 looks at the long-term safety of the drug sabatolimab in people with myelodysplastic syndromes or chronic myelomonocytic leukemia who already benefited from it in a previous trial. About 33 participants will continue treatment and be monitored for side effects. The goa…

This study tests an experimental oral drug called LY3410738 in people with advanced blood cancers such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) that have specific IDH1 or IDH2 gene mutations. The drug is designed to block the mutated IDH proteins that he…

This study tests whether giving patients hyperbaric oxygen (breathing pure oxygen in a pressurized chamber) after an umbilical cord blood stem cell transplant helps the donated stem cells grow faster. The trial enrolls people with blood cancers like leukemia or lymphoma who lack …

This study tests whether combining two drugs, luspatercept and lenalidomide, can improve anemia in people with lower-risk myelodysplastic syndrome (MDS) who lack a specific chromosome change. Participants take lenalidomide daily by mouth and receive luspatercept as a shot under t…

This study tests a drug called imetelstat in adults with a bone marrow disorder (MDS) who currently need regular red blood cell transfusions because standard treatments have stopped working. The main goal is to see if the drug can help patients go 8 weeks or more without a transf…

This phase 1 trial tests whether the drug tagraxofusp can help prevent relapse in people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, myelofibrosis, or chronic myelomonocytic leukemia) who have recently received a stem cell transplant. Participant…

This early-phase trial tests a combination of three drugs—azacitidine, venetoclax, and pevonedistat—in adults newly diagnosed with a challenging form of acute myeloid leukemia (AML). The goal is to find the best dose and see if the combo can improve remission rates. About 40 part…

This trial tests whether adding two drugs—vorinostat and low-dose azacitidine—after a stem cell transplant can help prevent relapse in children with certain myeloid cancers. The study includes children aged 1 to 21 with acute myeloid leukemia or related conditions. Researchers ar…

This early-phase study tests whether giving healthy donor NK cells along with two medicines (vactosertib and IL-2) is safe and helps the cells stay active in people with advanced colorectal, stomach, esophageal, or blood cancers. The NK cells are meant to attack cancer, but the b…

This study tests a combination of two drugs—tagraxofusp and decitabine—in people with chronic myelomonocytic leukemia or high-risk myelodysplastic syndromes. Tagraxofusp is a targeted therapy that delivers a toxin to cancer cells, while decitabine is a chemotherapy drug. The tria…

This early-stage trial tests a combination of three drugs—pembrolizumab, decitabine, and venetoclax—in people with acute myeloid leukemia or myelodysplastic syndrome that has returned or not responded to treatment. The goal is to find the safest dose and see how well the drugs wo…

This phase 2 trial tests whether adding lab-grown umbilical cord blood cells (dilanubicel) to a standard cord blood transplant helps people with blood cancers like leukemia recover their blood and immune cells faster. Participants receive chemotherapy and radiation to prepare the…

This trial tests whether combining roxadustat with retinoic acid can help people with low-risk myelodysplastic syndromes (MDS) whose disease hasn't responded to initial treatment. The goal is to improve blood cell counts and reduce the need for transfusions. Participants will tak…

This early-phase study tests two drugs—guadecitabine and atezolizumab—together in adults with myelodysplastic syndrome or chronic myelomonocytic leukemia that has come back or not responded to prior therapy. The goal is to find the safest dose and see if the combination can shrin…

This trial tests whether adding a radioactive antibody (90Y-DOTA-anti-CD25 basiliximab) to standard chemotherapy and targeted radiation can better prepare the bone marrow for a stem cell transplant in people with high-risk acute leukemia or myelodysplastic syndrome. The antibody …

This study tests a new approach for people receiving a stem cell transplant from a partially matched family donor. The donor's immune cells (T cells) are modified in the lab to include a 'suicide gene' that can be activated by a drug if the cells attack the patient's body, causin…

This trial tests whether specially modified T cells from a partially matched donor can help patients recover their immune system faster after a stem cell transplant. The T cells are engineered with a 'suicide gene' that allows doctors to destroy them if they cause graft-versus-ho…

This phase 1 trial is testing a combination of total marrow and lymphoid irradiation (TMLI) plus two chemotherapy drugs, fludarabine and melphalan, given before a donor stem cell transplant. The goal is to find the safest dose of radiation that can kill cancer cells while prepari…

This phase 2 trial tests whether adding the drug pomalidomide to standard chemotherapy (daunorubicin and cytarabine liposome) helps people with a newly diagnosed, aggressive form of acute myeloid leukemia (AML) linked to prior bone marrow disorders. About 50 participants will rec…

This study tests whether luspatercept works better than epoetin alfa for treating anemia in adults with a certain type of low-risk MDS who have not needed blood transfusions or prior anemia drugs. About 360 participants will receive one of the two drugs, and researchers will trac…

This trial investigates the best way to treat children with acute myeloid leukaemia (AML) by comparing different chemotherapy drugs and doses. It tests whether adding up to three doses of the targeted drug gemtuzumab ozogamicin to standard chemo is safe and effective. The study a…

This pilot study is testing whether the drug luspatercept can help people with lower-risk myelodysplastic syndromes (MDS) need fewer blood transfusions. Ten adults with MDS will receive the drug by injection. The main goal is to see if it is safe and reduces transfusion needs.

This study tests whether starting luspatercept (Reblozyl) at the maximum approved dose can help people with low-risk myelodysplastic syndromes (MDS) go without red blood cell transfusions for at least 8 weeks. About 106 participants who currently need regular transfusions will re…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

This study looks at whether lower doses of the chemotherapy drug CPX-351 can help older patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have come back or not responded to other treatments. The goal is to control the disease while being gentler o…

This early-stage trial tests a new drug called MP0533 in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to treatment. The drug is designed to help the body's immune cells find and attack cancer cells. The study will …

This phase 2 trial tests whether eltrombopag, a drug already approved for other blood disorders, can improve blood cell counts in people with low-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) who have a mutation in the TET2 gene. Participants take…

This early-phase study tests a new drug called uproleselan combined with two standard chemotherapy drugs for children and teens with acute myeloid leukemia or related blood cancers that have returned or not responded to treatment. The drug works by blocking a protein that helps c…

This study tests whether adding the drug itacitinib to the usual two-drug regimen (cyclophosphamide and tacrolimus) can better prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. GVHD is a serious complication where donor cells at…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

This study tests a combination of two chemotherapy drugs (CPX-351) plus a targeted drug (quizartinib) in people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The goal is to find the best dose and see if the combo helps control the cancer. About 52…

This early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy before and after a bone marrow transplant can help prevent cancer from coming back in people with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or related blood…

This trial aimed to test the drug ASTX727 (Inqovi) alone or with donor immune cells in people with blood cancers who had a stem cell transplant. The goal was to see if it could control cancer that remained or came back. However, the study was withdrawn before enrolling any patien…

This early-phase study tests a stem cell transplant method for people with advanced blood cancers like leukemia. Instead of using strong drugs to prevent complications, doctors give specially selected immune cells (regulatory T-cells) along with regular T-cells to help the body a…

This early-phase trial is testing a drug called AZD6738 in 52 people with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) whose disease got worse after standard therapy. The drug works by blocking a DNA repair pathway that cancer cells rely on, causing th…

This study tests an experimental drug called imetelstat in 46 adults with high-risk myelodysplastic syndromes or acute myeloid leukemia that did not improve after standard therapy. The goal is to see if the drug can improve blood cell counts and control the disease. Participants …

This study tests whether combining two drugs, azacitidine and quizartinib, can help people with myelodysplastic syndromes or related blood cancers that have specific genetic changes (FLT3 or CBL mutations). The trial involves 30 adults and aims to find the best dose and see how w…

This study tests whether adding the drug venetoclax to standard azacitidine treatment helps people with a severe form of myelodysplastic syndrome (MDS) live longer. MDS is a bone marrow disorder that can lead to leukemia. About 500 adults with newly diagnosed high-risk MDS will r…

This study tests a drug called luspatercept in 6 adults with lower-risk myelodysplastic syndromes (MDS) or related blood cancers who have anemia. The goal is to see if the drug can reduce or eliminate the need for regular blood transfusions. Participants receive luspatercept plus…

This clinical trial is testing different treatment strategies to improve outcomes for older adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who are getting a stem cell transplant. The study compares a new pre-transplant drug (Vyxeos) with stan…

This study tests whether giving a personalized dose of a drug called r-ATG before a stem cell transplant helps the immune system recover faster and reduces serious side effects. It includes children and adults with acute leukemia or myelodysplastic syndrome (MDS) receiving a dono…

This study tests a new way to do stem cell transplants for children and young adults (up to age 22) with blood cancers like leukemia. Doctors remove certain immune cells from donated stem cells to lower the risk of graft-versus-host disease, a serious complication. The goal is to…

This study tests whether stem cells from donated umbilical cords are safe and can help people with myelodysplastic syndrome (MDS), a bone marrow disorder. About 40 participants with moderate to severe symptoms will receive the stem cells and be monitored for side effects and impr…

This study tests a new drug called bexmarilimab, given together with standard treatments, for people with certain blood cancers (MDS, CMML, or AML). The goal is to first find a safe dose and then see if the combination helps shrink or control the cancer. About 181 adults are taki…

This phase II trial tests whether giving a short course of azacitidine or decitabine before standard chemotherapy can improve outcomes for children with newly diagnosed acute myeloid leukemia (AML). The study enrolls 206 participants and focuses on tolerability, changes in DNA me…

This early-phase study tests CPX-351 (Vyxeos), a two-drug chemotherapy, in 20 adults aged 18-70 with higher-risk myelodysplastic syndrome (MDS) who are eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, with secondary goals of measurin…

This study tests a stem cell transplant method for people with blood cancers like leukemia or lymphoma. The approach uses chemotherapy, total body radiation, and a drug called cyclophosphamide after the transplant to prevent graft-versus-host disease, where donor cells attack the…

This phase 2 trial tests whether adding venetoclax to a specific chemotherapy regimen before a donor stem cell transplant can improve outcomes for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study enrolls 116 adults aged 18-70 who have a matche…

This study compares stem cell transplants from two types of donors for people with leukemia or myelodysplastic syndromes (MDS): younger non-family members (age 40 or under) and older first-degree relatives (over 50). The goal is to see which donor leads to better overall survival…

This study tests a new way to do stem cell transplants for 20 children with blood cancers or myelodysplasia who don't have a perfect donor match. Doctors remove certain immune cells from a parent's partially matched stem cells before transplant to help the new cells grow faster a…

This early-stage trial tests an experimental drug called INCB057643, given alone or with the standard drug ruxolitinib, in about 140 adults with myelofibrosis or other advanced blood cancers. The main goal is to check safety and side effects, while also measuring if the drug can …

This phase 1 trial tests whether the oral drug Inqovi (decitabine/cedazuridine) can help prevent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML) from returning after a stem cell transplant. About 22 adults who have had a reduced-intensity transplant will …

This early-stage trial tests a precise type of radiation (IMTMI) given with chemotherapy before a second donor stem cell transplant for people with advanced blood cancers that have returned. The goal is to find the safest dose and see if it can reduce side effects and prevent the…

This phase 2 study tests whether the drug ruxolitinib can help prevent acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) from coming back after a stem cell transplant. About 64 older adults in remission will take ruxolitinib as maintenance therapy. The goal is to see…

This early-stage trial tests the safety and best dose of combining two drugs—everolimus and midostaurin—for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or is hard to treat. The drugs work by blocking faulty signals that help cance…

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This study tests an oral drug called emavusertib (CA-4948) in adults with acute myeloid leukemia (AML) or a high-risk bone marrow disorder (MDS) that have not responded to or returned after prior treatment. The goal is to find the safest and most effective dose and to see if the …

This study tests a drug called eltrombopag in adults with a low-risk form of myelodysplastic syndromes (MDS) who regularly need platelet transfusions. The goal is to see if the drug can help them go without transfusions for at least 8 weeks. About 36 people in Japan will take par…

This study tests a stem cell transplant using donors who are not a perfect match for patients with blood cancers like leukemia and lymphoma. Doctors give special drugs after the transplant to help prevent the donor cells from attacking the patient's body. The goal is to see if th…

This study tests a combination of two drugs (venetoclax and azacitidine) plus donor immune cells in 55 adults with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that returned after a stem cell transplant. The goal is to find the best dose and see if the treatment…

This phase 3 trial tests a new treatment called Orca-T for people with advanced blood cancers like leukemia. Orca-T is a special mix of stem cells and immune cells from a matched donor, designed to reduce a serious side effect called graft-versus-host disease. The study compares …

This phase II trial tests a special stem cell transplant that removes immune T-cells from the donor's blood to lower the risk of graft-versus-host disease (GVHD), a serious complication where the donor cells attack the patient's body. About 50 adults with myelodysplastic syndrome…

This early-phase study tests whether the drug talazoparib can help people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who have a specific genetic mutation (cohesin) and cannot receive standard intensive chemotherapy. The main goal is to see if the drug red…

This Phase 1 trial aimed to test whether bone marrow from deceased, mismatched unrelated donors could be safely used for transplants in patients with blood cancers like leukemia. The approach used stored (cryopreserved) marrow along with drugs to prevent rejection. However, the s…

This study tests an oral medication called azacitidine (ONUREG) for people with a low- or intermediate-risk form of myelodysplastic syndrome (MDS), a bone marrow disorder. About 230 participants will receive either the drug or a placebo, along with standard supportive care. The g…

This study tests whether a geriatric assessment—looking at thinking, mobility, mood, nutrition, and medications—can help doctors choose the right transplant intensity for patients aged 60 and older with myeloid blood cancers. The goal is to reduce deaths from transplant complicat…

This study tests a new conditioning regimen using a precise type of radiation (helical tomotherapy) along with two chemotherapy drugs (fludarabine and melphalan) before a donor stem cell transplant. The goal is to reduce side effects and improve outcomes for people with leukemia …

This early-stage trial tests a new therapy called SENTI-202, which uses specially designed immune cells (CAR NK cells) to find and attack cancer cells in people with certain blood cancers like acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The cells are 'off-the…

This early-stage trial aimed to test a combination of two drugs—ASTX727 (already approved for some blood cancers) and ASTX029 (an experimental ERK inhibitor)—in adults with myelodysplastic syndromes (MDS) or related blood cancers that have a mutation in the RAS pathway. The goal …

This study compares an oral drug (ASTX727) to an intravenous (IV) drug (decitabine) in 72 Chinese adults with myelodysplastic syndromes (MDS), a bone marrow disorder. Participants will receive both treatments in a crossover design to see if the pill works as well as the infusion.…

This early-phase study tests the safety of combining three drugs—navitoclax, venetoclax, and decitabine—for people with advanced myeloid blood cancers like leukemia and myelofibrosis. The goal is to find a safe dose that can control the disease. Sixteen adults aged 18 and older a…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children, adolescents, and young adults receiving a bone marrow or blood stem cell transplant for blood cancers. Researchers are first finding the best dose of vor…

This first-in-human trial tests BYON4413, an antibody-drug conjugate designed to target and kill cancer cells in people with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic neoplasms (MDS). The study involves 16 participants and aims to check safety, how th…

This phase II trial tests whether adding targeted total marrow irradiation to standard chemotherapy before a stem cell transplant can help prevent relapse in people with high-risk acute myeloid leukemia or myelodysplastic syndromes. About 38 adults aged 18–65 will receive the com…

This study tests a new stem cell treatment (MGTA-456) made from umbilical cord blood for people with high-risk blood cancers like leukemia and lymphoma. About 22 patients will receive high-dose chemotherapy followed by the stem cell infusion. The goal is to see if this treatment …

This phase 2 trial tests whether adding the drug tocilizumab to a standard transplant regimen can prevent graft failure and graft-versus-host disease in people with blood cancers receiving a special type of bone marrow transplant. The study involves 21 participants and aims to re…

This study looks at whether adding venetoclax to standard chemotherapy (HMAs) works better than chemotherapy alone for people newly diagnosed with higher-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). About 224 adults are being observed to see how…

This study tests whether giving palifermin and leuprolide acetate can help the immune system recover more quickly after a stem cell transplant for blood cancers like leukemia and lymphoma. About 82 participants will receive these drugs alongside their transplant. The goal is to s…

This early-phase trial tests a combination of two treatments for people whose myeloid blood cancer (like AML or MDS) has returned after a matched stem cell transplant. The treatments are a specially processed donor immune cell infusion and an immunotherapy drug called ipilimumab.…

This study tests two drugs—omacetaxine and azacitidine—given together to people with untreated high-grade myelodysplastic syndromes (MDS), a serious bone marrow condition. The first part finds the safest dose of omacetaxine, and the second part checks how many patients achieve re…

This early-phase trial tests whether adding regorafenib to the standard venetoclax and azacitidine regimen is safe and effective for people with acute myeloid leukemia (AML) that has returned or not responded to prior treatment. About 20 adults will receive the three-drug combina…

This study tests a stem cell transplant method that removes certain immune cells (T and B cells) from donated stem cells before giving them to patients with blood cancers like leukemia or lymphoma. The goal is to help the new cells grow (engraft) while reducing the risk of graft-…

This early-stage trial tests two drugs together—canakinumab and darbepoetin alfa—in people with a lower-risk form of myelodysplastic syndromes (MDS) whose previous treatment stopped working. The goal is to see if the combination is safe and can help the body make more red blood c…

This study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…

This phase II trial tests a new way to use cord blood transplants for people with high-risk acute leukemia or myelodysplasia. The cord blood is treated with a molecule called UM171 to increase the number of stem cells, which may help the transplant work better and reduce serious …

This study tests a drug called luspatercept in 70 adults with a low-risk form of MDS that causes anemia. The goal is to see if the drug can reduce the need for red blood cell transfusions. Participants receive the drug and are monitored for safety and effectiveness over time.

This early-phase trial tests whether the drug itacitinib can prevent cytokine release syndrome (a severe inflammatory reaction) after a mini stem cell transplant in people aged 60 and older with certain blood cancers. The study also aims to see if itacitinib allows doctors to use…

This trial is testing a new imaging tracer called FLT, which is used with a PET scan to measure how fast tumors are growing. The study includes people with various cancers or those awaiting a biopsy for a suspicious mass. The goal is to see if FLT PET imaging can help find and di…

This early-phase study tests whether vaccinating stem cell donors against cytomegalovirus (CMV) can prevent the virus from causing problems in blood cancer patients after a transplant. About 34 donor-recipient pairs are taking part. The vaccine is given to the donor before stem c…

Researchers are testing whether starting epoetin alfa early, as soon as anemia appears, works better than waiting until a person needs blood transfusions. The trial enrolls adults with lower risk myelodysplastic syndromes (MDS) who have mild anemia and are not yet dependent on re…

Researchers in Greece are testing a nine-session group program called TRANSFORM for people who have finished treatment for blood cancers such as leukemia, lymphoma, and myeloma. The program combines teaching, reflection, coping exercises, and mindfulness to see whether it can spa…

This study looks at whether starting palliative care early can help adults with aggressive leukemias or myelodysplastic syndrome (MDS) have a better end-of-life experience. Researchers will compare where patients die (home, hospice, or hospital) and how often they need hospital c…

This study looks at whether adding a palliative care team to standard leukemia treatment helps people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) feel better and have better end-of-life planning. About 320 patients receiving non-intensive therapy will take…

This study is testing a new blood test called AlloHeme that measures how much of a patient's own cells remain after a bone marrow transplant. The goal is to see if this test can predict whether the cancer (acute leukemia or myelodysplastic syndrome) will come back. About 307 adul…

This study looks at how well the heart, lungs, and muscles work together in people who have had a stem cell transplant. Researchers want to find out why transplant survivors have a higher risk of heart disease. About 350 adults with blood cancers will be followed for two years af…

This study is investigating how common muscle loss, known as sarcopenia, is in people with blood cancers like leukemia, lymphoma, and myeloma. Researchers will measure body composition using DXA scans and track hospital stays, disease progression, and survival. The goal is to see…

This study is collecting blood, bone marrow, and tissue samples from 5,000 people with blood cancers like leukemia and lymphoma. Researchers will analyze the samples to find genetic abnormalities that cause cancer cells to grow. The goal is to understand why these cancers develop…

This study looks at whether a special type of testing, called leukemia profiling, can help find personalized treatment options for children and young adults up to age 30 with recurrent, refractory, or high-risk leukemias. Researchers analyze the genetic changes in each patient's …

This study collects blood and bone marrow samples from up to 550 people with blood cancers like leukemia, lymphoma, and myelodysplastic syndrome. Researchers will study the cells, genes, and proteins in these samples to better understand how these cancers develop and to find new …

This study is testing whether a specially designed video game can help young people (ages 15-39) who have had a stem cell transplant for leukemia or lymphoma improve their health habits. Researchers will gather feedback from 40 participants to see if the game is easy and enjoyabl…

This study looks at how patients with advanced blood cancers (like acute myeloid leukemia) who are not eligible for a cure, along with their nurses and doctors, feel about the benefits of blood transfusions. Researchers will give questionnaires before each transfusion to compare …

This early-stage study tests whether the cholesterol drug atorvastatin can reduce levels of a faulty protein (p53) in people with various cancers, including solid tumors and leukemia. About 50 participants will take atorvastatin for 1 to 4 weeks before their planned surgery or tr…

This study follows 60 people with lower-risk myelodysplastic neoplasms (MDS) who need regular red blood cell transfusions. Over 6 months, participants use a smartphone app to answer weekly quality-of-life questions and take photos of their fingernails or eyelids. Researchers aim …

This study was designed to learn how the protein calprotectin might change blood stem cells in people with severe COVID-19 or certain blood cancers. Researchers planned to compare cells from patients and healthy donors in the lab. However, the study was withdrawn before any parti…

This study uses a large Spanish database to understand how many people have transfusion-dependent lower-risk myelodysplastic syndromes (MDS) and what treatments they receive first. Researchers will look at medical records of about 1,300 adults to see patterns in care and health o…

This study looks at whether testing cancer cells in a lab can predict how well chemotherapy will work for people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Researchers will take blood and bone marrow samples before and during treatment to see if lab res…

This study follows 300 people with persistent low blood cell counts (cytopenia) who do not yet have myelodysplastic syndromes (MDS), a type of blood cancer. Researchers aim to track how often these pre-MDS conditions (called ICUS and CCUS) progress to MDS. Participants receive on…

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…

This study follows 144 blood cancer patients undergoing stem cell transplants to see how muscle function changes and whether it predicts complications like infections, relapse, or survival. Researchers will measure muscle mass and track medical events over time. The goal is to un…