Myelodysplastic syndrome

Researchers test whether adding venetoclax to a standard conditioning regimen called FLAMSA-treosulfan is safe for people with high-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or secondary acute myeloid leukemia who are receiving a donor blood stem cell trans…

This study tests a new approach to prevent leukemia from coming back after a stem cell transplant. It uses donor T cells that have been engineered to recognize and attack a specific target (HA-2) on leukemia cells. The trial enrolls adults with acute myeloid leukemia (AML) or mye…

This study tests whether adding enasidenib to standard treatment (ASTX727) improves outcomes for people with a high-risk form of myelodysplastic syndrome (MDS) that has a specific gene mutation (IDH2). About 54 adults who have not had prior cancer therapy will be randomly assigne…

This study tests whether adding the drug venetoclax to standard chemotherapy can better treat acute myeloid leukemia (AML) that is newly diagnosed or has come back. About 116 adults with AML or high-risk MDS will receive the combination. The goal is to see if the combo improves r…

This study tests whether giving donor white blood cells (lymphocytes) soon after a stem cell transplant can lower the chance of cancer returning in people with high-risk blood cancers like leukemia or lymphoma. Participants receive chemotherapy, a stem cell transplant, and then a…

This phase 2 trial is testing a combination of five drugs (fludarabine, cytarabine, filgrastim-sndz, gemtuzumab ozogamicin, and idarubicin) in 270 people with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome that have specific genetic changes. The goal…

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 3 trial compares elritercept to epoetin alfa in 300 adults with low- to intermediate-risk myelodysplastic syndromes (MDS) who need red blood cell transfusions. The main goal is to see if elritercept can help patients go without transfusions for at least 12 weeks while …

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This early-stage trial is testing whether two drugs that lower metal levels in the body can help people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) respond better to chemotherapy. The study will enroll 58 adults and aims to find the safest dose and check f…

This study tests whether giving a chemotherapy drug (decitabine) plus a targeted therapy (ruxolitinib, fedratinib, or pacritinib) before a stem cell transplant helps people with advanced myeloproliferative neoplasms (MPN) get to transplant and have better outcomes. About 25 adult…

This early-phase trial tests a two-step approach for people with acute myeloid leukemia or related blood cancers that have returned or not responded to treatment. First, patients receive intensive chemotherapy (CLAG-M or FLAG-Ida) followed immediately by a reduced-intensity stem …

This phase 2 trial tests whether adding the targeted drug venetoclax to standard chemotherapy is safe and effective for children and young adults (up to age 22) with newly diagnosed acute myeloid leukemia (AML). The study aims to see if this combination can reduce the amount of c…

This study tests whether a stem cell transplant from a healthy donor can treat GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of leukemia. Participants aged 6 to 70 with GATA2 mutations will receive chemotherapy or radiation before t…

This study looks at two different chemotherapy combinations given before a half-matched stem cell transplant in adults with myelodysplastic syndrome (MDS). One regimen is designed for people aged 55 and older to reduce side effects, while the other is a standard stronger regimen …

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

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-stage trial is testing an experimental drug called AMX-883, taken by mouth, for people with acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS) that has come back or not responded to standard treatments. The study will first test AMX-883 alone, th…

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 early-stage study tests an oral drug called AUTX-703 in 34 adults with relapsed or hard-to-treat acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The main goal is to check safety and find the right dose. It's an open-label trial, meaning everyone knows they'r…

This Phase II trial tests whether combining luspatercept and darbepoetin alfa can help people with a certain type of lower-risk myelodysplastic syndrome (MDS) need fewer red blood cell transfusions. The study enrolls 60 adults who currently rely on transfusions. Participants rece…

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-phase trial tests whether giving patients a special infusion of donor immune cells (with certain T and B cells removed) after a stem cell transplant is safe and can help prevent their blood cancer from coming back. The study enrolls high-risk patients with hard-to-trea…

This phase II study is testing a combination of two drugs, enasidenib and azacitidine, in people with acute myeloid leukemia (AML) that has come back or not responded to treatment. The trial enrolls 50 adults with a specific IDH2 gene mutation. The goal is to see if the combinati…

This study tests a combination of three drugs—ivosidenib, venetoclax, and azacitidine—in people with blood cancers like acute myeloid leukemia (AML) that have a specific IDH1 mutation. The goal is to see if the combo is safe and works better than current treatments. About 96 adul…

This early-phase trial is testing a new drug called Debio 1562M in about 134 people with acute myeloid leukemia (AML) that has come back or not responded to other treatments. The study first checks safety and finds the right dose, then looks at whether the drug can shrink the can…

This study is testing whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (a common complication where donor cells attack the patient's body) while still being effective. The trial involves 260 adults with b…

This study tests a standardized, optimized approach to cord blood transplantation for adults with high-risk blood cancers like leukemia and lymphoma. The method includes specific chemotherapy, radiation, and stem cell dosing, followed by careful monitoring. Researchers hope to im…

This phase 2 trial tests a drug called olutasidenib combined with other targeted therapies for people with relapsed or hard-to-treat blood cancers that have a specific IDH1 mutation plus other signaling mutations. The study aims to see how safe and tolerable these drug combinatio…

This early-phase study tests a new drug called rezatapopt combined with the standard drug azacitidine in 24 adults with a specific type of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has a TP53Y220C mutation. The main goal is to check safety and find the b…

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…

Researchers are testing an experimental drug called LP-108, a BCL-2 inhibitor, combined with azacitidine in adults with acute myeloid leukemia, myelodysplastic syndromes, or chronic myelomonocytic leukemia. The trial aims to find the highest dose people can tolerate and the best …

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 whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

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-stage trial is testing whether an oral form of arsenic trioxide can safely help people with low-risk myelodysplastic syndromes (MDS) who have not improved with standard treatments. The study will first find the safest dose, then check how well it works after several cy…

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 early-stage trial is testing a new antibody drug called GEN3018 in people whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) has come back or stopped responding to treatment. The main goal is to check safety and find the right dose. Up to 78 parti…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study tests a combination of three drugs—ASTX727, venetoclax, and gilteritinib—for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome that has a FLT3 mutation. The drugs work in different ways to stop cancer cells from growing. The trial aims to f…

This study tests a combination of two drugs, azacitidine and enasidenib, in people with a high-risk blood cancer called myelodysplastic syndrome (MDS) that has a specific genetic change (IDH2 mutation). The goal is to see if the drugs can stop cancer growth and improve outcomes. …

This study tests a new type of immune cell therapy for people with myeloid cancers (like AML or MDS) that have come back or not responded to treatment. The cells are taken from donated cord blood, engineered to better recognize and attack cancer cells, and given after chemotherap…

This study is for people with a rare form of myelodysplastic syndromes (MDS) that has a specific genetic change called IDH1 mutation. Participants have not had prior treatment with certain drugs. They will be randomly assigned to receive either ivosidenib (a daily pill) or azacit…

This study screens people with acute myeloid leukemia (AML) for specific genetic markers to assign them to a treatment sub-study with a targeted drug or combination. It aims to improve response rates by personalizing therapy based on each person's cancer profile. The trial enroll…

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 tests two chemotherapy drugs, treosulfan and melphalan, given before a stem cell transplant in 220 adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The goal is to see which drug works better at helping patients survive longer with fewer side …

This study tests a drug called TAK-226 (Elritercept) in 27 Japanese adults with lower-risk myelodysplastic syndromes (MDS) who need regular blood transfusions. The goal is to see if the drug can help them go without transfusions for at least 8 weeks. Participants will receive inj…

This early-phase trial tests a new treatment called CD64 CAR T cells for adults with acute myeloid leukemia (AML) that has come back or not responded to standard therapy. The treatment uses a patient's own immune cells, modified in a lab to target and attack leukemia cells. The m…

This study tests whether adding the drug ruxolitinib to standard medications (cyclophosphamide and tacrolimus) can prevent severe graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers like leukemia. About 124 adults will receive the drug com…

This early-stage study tests a new treatment called NT-175 for people with advanced solid tumors (like lung, colon, or breast cancer) that have a specific mutation (TP53 R175H) and a certain immune marker (HLA-A*02:01). NT-175 is made from a patient's own immune cells, which are …

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 early-stage trial is testing an experimental drug called JNJ-90189892 in about 155 people with acute myeloid leukemia (AML) or myelodysplastic neoplasms (MDS) that have come back or not responded to treatment. The study has several parts: first, finding a safe dose of JNJ-90…

This study tests a new antibody called axatilimab, given with or without the chemotherapy drug azacitidine, for people with advanced myeloproliferative neoplasms (MPN), MPN/MDS overlap, or high-risk chronic myelomonocytic leukemia (CMML). The goal is to find the safest dose and s…

This phase II trial is testing whether the drug ivosidenib, taken as a pill, can help prevent relapse in people with acute myeloid leukemia (AML) or myelodysplastic neoplasia (MDS) who have an IDH1 mutation and have recently undergone a stem cell transplant. Participants will sta…

This early-phase study tests a new treatment called CER-1236 for people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, or myelofibrosis) that have not responded to standard therapy. The treatment uses a patient's own immune cells, which are modified…

This study tests a combination of two drugs, CPX-351 and ivosidenib, in about 30 adults with a specific genetic form of acute myeloid leukemia or high-risk myelodysplastic syndrome. The goal is to see if the combo can shrink or control the cancer. Participants must have the IDH1 …

This early-phase study is testing two drugs together — alpelisib and pembrolizumab — in people with metastatic breast cancer or melanoma that has spread to the brain or other organs. The main goal is to find a safe dose and see what side effects occur. Up to 50 adults whose cance…

This early-stage trial tests a new drug called imetelstat combined with two standard chemotherapies for children and teens with acute myeloid leukemia, myelodysplastic syndrome, or juvenile myelomonocytic leukemia that has returned or not responded to treatment. The main goal is …

This early-phase study tests a new drug called eganelisib, given alone or with standard chemotherapy (cytarabine), in adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) that have returned or not responded to prior treatments. The main goals are …

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 early-stage trial tests a new treatment called QUAIL-100, made from a live weakened bacteria, in children and young adults with high-risk leukemia or myelodysplastic syndrome who have already had a stem cell transplant. The main goal is to check safety and side effects. Only…

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 study tests a targeted radiation technique (TMLI) combined with two chemotherapy drugs before a stem cell transplant in people aged 50 and older with acute myeloid leukemia or myelodysplastic syndrome that has not responded to treatment or has returned. The goal is to see if…

This study is testing a combination of three drugs—quizartinib, decitabine, and venetoclax—in people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome. The goal is to find the safest dose and see if the combination improves remission rates. The trial is for …

This early-phase trial is testing whether combining two oral drugs, eltanexor and venetoclax, is safe and effective for people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that has returned or not responded to prior treatment. The study will enroll 60 adult…

This study tests a new way to do stem cell transplants for children with blood cancers like leukemia. It uses a half-matched family donor and removes certain immune cells from the donated cells to lower risks. The goal is to see if this method is safe and helps the child's body a…

This early-phase study tests a new drug (ICP-248) combined with a standard medicine (azacitidine) in people with acute myeloid leukemia or myelodysplastic syndromes. The goal is to find a safe dose and see if the combo helps control the disease. About 266 adults who have not resp…

This early-stage trial is testing a new combination of chemotherapy drugs (thiotepa, busulfan, and fludarabine) followed by a stem cell transplant for people with certain blood cancers like leukemia and myelodysplastic syndrome. The goal is to see if this approach can reduce the …

This study tests whether patients can safely stop the drug tacrolimus earlier than usual after a stem cell transplant. Tacrolimus is given to prevent graft-versus-host disease (GVHD), but newer methods have lowered GVHD risk. The trial will enroll 50 people with blood cancers and…

This study tests a drug called BPC2001 to prevent acute graft-versus-host disease (aGVHD), a serious complication where donor immune cells attack the patient's body after a stem cell transplant. Fifty adults with blood cancers like leukemia will receive six weekly doses of BPC200…

This trial tests whether giving patients special immune cells, called TAA-T, after a stem cell transplant can help prevent high-risk blood cancers like acute myeloid leukemia from coming back. The cells are designed to recognize three common tumor markers and attack any remaining…

This trial is testing whether an experimental drug called PBSS1113, taken as a pill alongside the standard medicine azacitidine, can help people with acute myeloid leukemia (AML) or a related condition called myelodysplastic syndrome (MDS). The study includes adults who have eith…

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 clinical trial is testing whether adding the drug venetoclax to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The study will enroll 324 participants and aims …

This phase 2 trial tests the drug momelotinib in 57 people with VEXAS syndrome, a rare inflammatory condition that may also involve a bone marrow disorder called MDS. Participants receive momelotinib daily, and doctors check if symptoms improve and if steroid doses can be lowered…

This early-stage trial is testing an oral form of the cancer drug azacitidine (called ASTX030) for people with myelodysplastic syndrome (MDS). The goal is to see if the pill version reaches similar levels in the blood as the standard injection. About 40 adults with MDS will take …

This study tests an experimental drug called R289 in about 86 adults with a type of blood cancer called lower-risk myelodysplastic syndromes (MDS). Participants must have tried other standard treatments without success. The main goal is to check the drug's safety and side effects…

This study tests a new treatment for people with blood cancers like leukemia, lymphoma, and multiple myeloma that have returned or not responded to treatment. Participants receive chemotherapy followed by specially engineered natural killer (NK) cells from donated cord blood. The…

This early-phase trial tests the safety of a new drug called karonudib in up to 9 adults with blood cancers like leukemia who have no standard treatment options left. The study aims to find a safe dose and look for any signs that the drug works. It is too early to know if karonud…

This trial tests whether a drug called ATLG, used before a stem cell transplant, can help the immune system recover better and reduce viral infections compared to the standard drug ATG. The study involves adults with blood cancers like leukemia, lymphoma, or MDS who are receiving…

This study tests an experimental inhaled drug called PUL-042 in 100 people with blood cancers or who have had a stem cell transplant and have a viral lung infection. Participants receive either the drug or a placebo three times over six days. The goal is to see if PUL-042 can red…

This early-phase trial tests whether adding a radioactive antibody (225Ac-DOTA-daratumumab) to standard chemotherapy and targeted radiation can safely improve the conditioning regimen before a donor stem cell transplant. The study includes 15 adults with high-risk acute myeloid l…

This phase 2 trial tests whether giving patients their own personalized natural killer (NK) cells after chemotherapy and a cord blood transplant can help kill any remaining cancer cells. The study includes 100 people with various blood cancers like leukemia, lymphoma, and multipl…

This program gives patients with certain blood cancers access to Orca-T, a donor stem cell therapy, when the commercial version cannot be used. It is for people who have no other treatment options. The program monitors safety but does not test if the therapy works.

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 tests a special cord blood transplant for people with high-risk blood cancers like leukemia and lymphoma. Participants get chemotherapy and radiation before the transplant to kill cancer cells and prepare the body. After the transplant, drugs are given to prevent the n…

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 early-phase study is testing an experimental oral drug called AZD3632 in about 84 people with advanced blood cancers (acute leukemias or myelodysplastic syndromes) that have certain genetic changes. The main goals are to check the drug's safety and find the right dose, while…

This pilot study tests a new treatment plan for adults aged 60 and older with high-risk acute myeloid leukemia (AML) or similar blood cancers. The plan combines a strong chemotherapy regimen (FLAG-Ida) with a lower-intensity stem cell transplant just a few days later. The goal is…

This early-phase trial tests whether combining tagraxofusp (a drug that targets CD123 on cancer cells) with azacitidine (a drug that helps bone marrow make normal cells) can safely prevent relapse in people with CD123-positive acute myeloid leukemia or myelodysplastic syndrome af…

This Phase 2 trial tests whether combining cladribine, low-dose cytarabine, and decitabine can help control acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). About 160 older adults (60+) who haven't had prior intensive treatment will receive the drugs in c…

This phase 1 trial tests a new drug called GTB-3650 for adults with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) that has come back or not responded to treatment. The drug is designed to connect natural killer cells—a type of immune cell—to cancer cel…

This phase 2 trial is testing whether adding the targeted drug venetoclax to a standard chemotherapy regimen (FLAG IDA) works better than the usual treatment for adults newly diagnosed with acute myeloid leukemia (AML). About 102 participants will receive either the new combinati…

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 expanded access protocol provides stem cell transplants for people with serious blood diseases like leukemia, bone marrow failure, or immune deficiencies who do not have a fully matched sibling donor. The donated stem cells are processed to remove certain immune cells (alpha…

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 stem cell transplant using a reduced-intensity conditioning regimen (lower doses of chemotherapy and radiation) followed by a donor stem cell infusion. The goal is to treat various blood cancers and blood disorders while lowering the risk of graft-versus-host d…

This early-phase trial is testing whether adding the drug emapalumab to standard medications can safely prevent graft-versus-host disease (GVHD) in 15 adults with leukemia or myelodysplastic syndrome receiving a reduced-intensity stem cell transplant. Emapalumab blocks a protein …

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

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 phase 2 trial tests whether adding vedolizumab to standard drugs (cyclophosphamide and tacrolimus) can prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers like leukemia and myelodysplastic syndrome. GVHD occurs when donor immu…

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 Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of…

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 study tests a new drug called CCS1477 (inobrodib) in people with advanced blood cancers like leukemia, lymphoma, and myeloma that have not responded to standard treatments. The drug is given as an oral capsule, either alone or combined with other cancer medicines. The goal i…

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 Phase 2 trial tests a combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can im…

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-phase study tests a new drug called SL-401 combined with two other drugs (azacitidine and venetoclax) in people with certain blood cancers—acute myeloid leukemia, myelodysplastic syndrome, and blastic plasmacytoid dendritic cell neoplasm. The main goal is to find the s…

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 is testing a new way to do stem cell transplants for people with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). It combines stem cells from a half-matched donor (haploidentical) with cord blood, given at different times. The goal is to help …

This early-stage trial is testing a new drug called cirtuvivint, alone or with another drug combo (ASTX727), in people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). The goal is to find the safest dose and check for side effects. About 54 participants whose…

This study combines three chemotherapy drugs (cladribine, idarubicin, cytarabine) with a targeted drug (quizartinib) to treat acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). It includes up to 80 adults who are newly diagnosed or whose cancer has returne…

This early-phase trial tests a genetically modified virus (VSV-IFNβ-NIS) alone or with chemotherapy and immunotherapy drugs in people with blood cancers like multiple myeloma, acute myeloid leukemia, and lymphomas that have returned or not responded to treatment. The virus is des…

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 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 new daily pill called JBI-802 in people with certain blood cancers (myeloproliferative neoplasms and MDS/MPN) who have too many platelets. The trial has two phases: first, finding a safe dose in about 30 adults, then checking if it works. Participants take the …

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 phase 2 trial is testing whether two dietary supplements—nicotinamide riboside (a vitamin B3 derivative) and pterostilbene (a plant compound)—can safely improve low blood counts in people with lower-risk myelodysplastic syndrome (MDS) or high-risk clonal cytopenia of undeter…

This study tests a stem cell transplant from half-matched (haploidentical) donors for people with various blood cancers. Before the transplant, patients receive chemotherapy and low-dose radiation to prepare their body. The goal is to replace diseased bone marrow with healthy don…

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 study tests a daily pill called ivosidenib in about 20 adults with a specific genetic form of acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who cannot receive a stem cell transplant. The goal is to see if the drug can keep the cancer from coming ba…

This study tests a drug called canakinumab in people with low- or intermediate-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML). The drug is a lab-made antibody that may help slow cancer growth and improve blood cell counts. About 76 adults will rece…

This early-stage trial is testing a chemotherapy drug called CPX-351 in people with high-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) that came back or didn't respond to standard treatment. The goal is to find the best dose that is safe and to se…

This study compares two drug combinations to prevent graft-versus-host disease (GVHD) in people aged 65 and older with certain blood cancers who receive a stem cell transplant. Participants will receive either cyclophosphamide, sirolimus, and MMF or cyclophosphamide, sirolimus, a…

This phase 3 trial compares two drug combinations given after a stem cell transplant to prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body. The study will enroll 572 adults with blood cancers like leukemia or myelodysplasi…

This phase 2 trial is testing a combination of four chemotherapy drugs—cladribine, idarubicin, cytarabine, and venetoclax—in people with acute myeloid leukemia, high-risk myelodysplastic syndrome, or blast-phase chronic myeloid leukemia. The goal is to see if this mix can kill ca…

This early-phase trial tests a new treatment called CD123-CAR T cells for children and young adults (up to age 21) with certain types of leukemia or related blood cancers that have come back or not responded to standard therapy. The treatment involves collecting the patient's own…

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 early-phase trial tests a new immunotherapy for children and adults with acute leukemia that has come back or not responded after a donor stem cell transplant. The treatment uses specially engineered donor T cells designed to recognize and attack a protein called HA-1 found …

This pilot study tests a stem cell transplant method that removes specific immune cells (alpha/beta T cells and B cells) from the donor cells before transplant. The goal is to reduce the risk of graft-versus-host disease, a serious complication, while still allowing the transplan…

This study tests a new approach for adults with aggressive blood cancers (like AML) that are likely to return after standard treatment. Participants receive a combination of chemotherapy drugs (decitabine plus FLAG-Ida) and low-dose radiation, followed immediately by a stem cell …

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 early-stage study tests a new oral drug, LP-118, in up to 100 adults with blood cancers like lymphoma, leukemia, or myeloma that returned or didn't respond to prior therapy. The main goals are to find the safest dose and see if the drug can shrink tumors or slow the disease.…

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 a new drug called lisaftoclax, alone or combined with other medicines, in people with acute myeloid leukemia that has come back or not responded to treatment. The main goals are to check safety and find the best dose. About 682 adults will take part across multip…

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 early-phase trial tests a stem cell therapy called Orca-T combined with targeted radiation and chemotherapy for people aged 60-75 with acute leukemias or myelodysplastic syndrome. The goal is to see if this approach is safe and can reduce side effects while effectively treat…

This phase 3 trial tests two drugs, Thymoglobulin and Grafalon, to see which one better prevents graft-versus-host disease (GVHD) in patients aged 50-70 with acute myeloid leukemia or myelodysplastic syndrome. All participants receive a stem cell transplant from a matched unrelat…

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 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 early-phase trial tests whether two drugs, ruxolitinib and abatacept, can prevent graft-versus-host disease (GVHD) and cytokine release syndrome in people receiving stem cell transplants from half-matched donors. About 41 adults with blood cancers will take these drugs aroun…

This small study tests a device called MEX-CD1 that removes excess iron from the blood during a special type of dialysis. It is for people with myelodysplastic syndrome or myelofibrosis who have iron overload from blood transfusions but cannot take standard chelation therapy. Eac…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests a new combination of drugs (cladribine, lisaftoclax, busulfan, and others) given before a stem cell transplant in 30 older adults (age 50+) with relapsed or refractory acute myeloid leukemia or myelodysplastic syndromes. The goal is to see if this approach can re…

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 study tests whether a drug called sargramostim can help restore white blood cells as quickly as the standard drug (G-CSF) after a half-matched stem cell transplant for blood cancers, while possibly lowering the risk of serious infections. About 38 adults with blood cancers l…

This study tests whether a once-weekly injection (romiplostim) can help platelets recover faster and more safely than a daily injection (rhTPO) after a donor stem cell transplant. It includes 66 adults with MDS or severe aplastic anemia. The goal is to reduce bleeding risk and tr…

This study tests a precise type of radiation called proton total marrow irradiation (TMI) given before a stem cell transplant for people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The goal is to see if adding TMI to standard chemotherapy is saf…

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-stage study tests a new drug, BMS-986497, in people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has returned or not improved after prior treatments. The drug is given alone or combined with standard medicines (azacitidine and venetoclax). T…

This study tests whether adding a precise, targeted form of radiation to the standard chemotherapy and stem cell transplant can kill more cancer cells in the bone marrow and reduce the chance of relapse. About 27 adults with acute leukemia or myelodysplastic syndrome (MDS) will r…

This study tests a special stem cell transplant for children and adults with blood cancers like leukemia. The transplant uses stem cells that have had certain immune cells removed to lower the risk of graft-versus-host disease, a serious complication. About 70 participants will r…

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 early-stage study is testing a new drug called RPT1G in 24 adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) have not responded to standard treatments or have returned. The main goals are to see if the drug is safe and to find the best do…

This phase 2 trial is testing a new combination of chemotherapy, radiation, and immune-suppressing drugs for people aged 18-65 with blood cancers who need a stem cell transplant. The goal is to see if this regimen improves survival one year after transplant. About 209 participant…

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 new drug combinations can better prevent graft-versus-host disease (GVHD) in people with blood cancers who receive a stem cell transplant from a mismatched unrelated donor. About 358 participants will get either the standard GVHD prevention or one of two …

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 phase 3 trial tests whether adding the targeted drug venetoclax to standard chemotherapy (fludarabine and melphalan) before a donor stem cell transplant can improve outcomes for patients over 50 with acute myeloid leukemia or myelodysplastic syndrome. The study will compare …

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 early-phase trial is testing a new drug called CBX-250 in people with certain blood cancers (like acute myeloid leukemia) that have come back or not responded to standard treatments. The main goals are to check the drug's safety and find the best dose. About 72 participants …

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This early-phase trial is testing whether adding the drug venetoclax to standard chemotherapy is safe and tolerable for children, adolescents, and young adults with high-risk blood cancers like MDS, AML, and ALL. About 30 participants will receive different combinations of chemot…

This phase II trial is testing two different methods to reduce graft-versus-host disease (GVHD) in patients with acute leukemia or myelodysplastic syndrome (MDS) who are receiving a donor stem cell transplant. One method removes certain immune cells from the donor cells before tr…

This early-phase trial tests a new drug called nadunolimab combined with standard treatments (azacitidine, with or without venetoclax) in 40 adults with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The goal is to see if the combination is safe and to …

This early-stage trial is testing whether a drug called olutasidenib can safely prevent blood cancers (AML, MDS, or CMML) from coming back after a donor stem cell transplant. About 15 adults with a specific gene mutation (IDH1) will take the drug daily for up to two years. The ma…

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 early-stage trial is testing a new drug called FHD-286 combined with two standard chemotherapy drugs (decitabine and venetoclax) in 33 people with acute myeloid leukemia that is either newly diagnosed with high-risk features or has returned after one prior treatment. The mai…

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 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 phase 2 trial tests whether low-dose azacitidine can help maintain donor cell levels (chimerism) in patients with MDS, AML, or related blood cancers after an allogeneic stem cell transplant. About 43 adults whose donor cell levels are dropping will receive azacitidine for at…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

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 study tests a drug called romiplostim N01 in 130 blood cancer patients receiving a stem cell transplant from a half-matched donor. The drug is given weekly for up to 4 weeks to help platelets recover faster, reducing the need for transfusions and bleeding risk. Half the pati…

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

This early-phase trial tests a new type of immune therapy called CD70-CAR T cells in children and young adults up to age 21 with blood cancers that have come back or not responded to treatment. The therapy involves taking the patient's own immune cells, engineering them to recogn…

This early-phase trial is testing a new drug called CG009301 in adults with blood cancers like leukemia that have returned or not responded to standard treatments. The main goals are to find a safe dose and see if the drug can help. About 45 participants will receive the drug by …

This early-stage study tests an oral combination of tetrahydrouridine and decitabine in 20 adults with myelodysplastic syndromes (MDS) that returned or didn't respond to prior therapy. The main goals are to check safety and see if the drug improves blood cell counts and reduces h…

This early-stage trial tests a new drug called CLN-049 in about 60 adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to treatment. CLN-049 is designed to help the body's immune cells attack cancer cells. The main goals …

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

This 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 sulforaphane, a compound found in broccoli sprouts, can help blood cells recover faster after a cord blood transplant in adults with leukemia or related blood cancers. The trial will involve 36 patients and focus on safety and how quickly neutrophils (a t…

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 a higher dose of a molecule called UM171 to grow more stem cells from donated cord blood for transplant. It is for adults with high-risk blood cancers like leukemia who have no suitable donor. The goal is to help patients recover blood counts faster, reduce infec…

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 early-stage trial tests a new treatment called MT-401-OTS for people with relapsed acute myeloid leukemia or high-risk myelodysplastic syndromes. The therapy uses donor immune cells specially trained to attack cancer, and is given as a one-time infusion. The main goals are t…

This study tests a new drug called ARD103 in adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) has come back or not responded to standard treatments. The main goals are to check safety and find the best dose, while also seeing if the drug can shrink or c…

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 phase 2 trial tests whether adding two drugs, ruxolitinib and abatacept, to standard transplant care can prevent graft-versus-host disease (GVHD) in 60 patients with high-risk myeloid cancers like AML and MDS. Participants receive a stem cell transplant from a partially matc…

This study tests whether adding lisaftoclax to standard azacitidine helps people with newly diagnosed higher-risk myelodysplastic syndrome (a serious blood cancer) live longer. About 490 participants worldwide will receive either the combo or a placebo plus azacitidine. The main …

This study tests a stem cell transplant approach for people with Fanconi Anemia who don't have a perfectly matched donor. The goal is to see if using lower doses of chemotherapy before the transplant can reduce serious side effects. About 70 participants will receive a transplant…

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 study tests whether adding the drug ruxolitinib to the usual anti-rejection medicines (tacrolimus and methotrexate) can prevent graft-versus-host disease (GVHD) in children and young adults getting a stem cell transplant for leukemia or myelodysplastic syndrome. GVHD happens…

This phase 2 trial tests a two-step, lower-intensity transplant method for people with various blood cancers. The goal is to see if this gentler approach reduces the chance of dying from the transplant itself. About 63 participants will receive a combination of radiation, chemoth…

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 early-phase trial tests a new type of stem cell transplant for children with blood cancers or solid tumors that have relapsed or not responded to standard treatment. Doctors use a half-matched donor's stem cells, specially filtered to remove certain immune cells, and give th…

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-stage trial is testing a new drug called Q702 in people with various blood cancers and related disorders that have not responded to standard treatments. Q702 is designed to help the immune system attack cancer cells and help the bone marrow make healthy blood cells. 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 phase 2 trial tests a two-step dosing schedule of anti-thymocyte globulin (ATG) combined with standard drugs (tacrolimus and mini methotrexate) to prevent severe graft-versus-host disease (GVHD) in 56 adults with blood cancers like acute leukemia. Participants receive ATG on…

This early-phase trial is testing a new drug called AB8939, given daily through a vein, for people with acute myeloid leukemia that has come back or not responded to standard treatments. About 78 participants will receive AB8939, sometimes with another drug called venetoclax. The…

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 study tests whether Darzalex Faspro can safely lower donor-specific antibodies (DSAs) in 8 adults with blood cancers or bone marrow failure who are at high risk of rejecting a stem cell transplant. The goal is to reduce DSA levels enough to allow the transplant t…

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 trial tests an oral drug called SP-420 in about 90 adults with transfusion-dependent β-thalassemia or low-risk myelodysplastic syndromes (MDS). The goal is to see how well SP-420 removes excess iron from the liver and other organs, and whether it is safe. Participants take t…

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 looks at a new genetic test called Caris Chromoseq for people with certain blood cancers (acute myeloid leukemia, myelodysplastic syndrome, or myeloproliferative neoplasms). The test scans the entire genome to help classify the cancer and guide risk assessment. Researc…

This study is testing a new blood test called PERIBLOOD-MDS that could diagnose a bone marrow disorder called MDS without needing a painful bone marrow biopsy. Researchers will compare results from the blood test with standard bone marrow exams in 1,500 adults with low blood coun…

This study is testing a new platform that uses advanced testing to get a complete picture of a person's tumor. The goal is to see if this detailed information can help doctors choose better treatments for patients with high-risk cancers like leukemia, multiple myeloma, and pancre…

This study looks at whether a low dose of the antifungal drug liposomal amphotericin B can prevent serious fungal infections in adults with blood cancers or who have had stem cell transplants and have a prolonged low white blood cell count (neutropenia). The trial enrolls 30 pati…

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

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 phase 2 trial tests a vaccine called CMV-MVA Triplex, given to stem cell donors before they donate. The goal is to boost the donor's immunity against cytomegalovirus (CMV), which can then be passed to the transplant patient. Researchers hope this will prevent CMV infection i…

This study tests whether a tailored physical activity program can help older adults maintain thinking and memory after a stem cell transplant for blood cancers. Participants aged 60 and older will follow a progressive, light-to-moderate exercise plan with support from therapists …

This study tests an online music therapy program that combines mindfulness and music to help people recovering from stem cell transplants for blood cancers. Researchers want to see if it can improve quality of life, reduce pain, fatigue, anxiety, and depression, and possibly spee…

This trial tests a program called ACT, which trains doctors and nurses to have compassionate, structured conversations with blood cancer patients and their families about end-of-life care. The goal is to help patients understand their prognosis and make informed decisions, potent…

This trial tests whether a specially designed website, called Mosaic, can help blood cancer patients who are about to undergo a stem cell transplant feel less distressed and have a better quality of life. Participants are randomly assigned to use either the Mosaic website or a st…

This study compares giving cancer treatment at home versus in a clinic for 27 adults with various cancers in the Florida Panhandle. The goal is to see if home treatment is preferred and improves satisfaction, while also checking safety. Participants will receive their usual cance…

This study tests a program called Nite2Day+ that uses mindfulness and sleep tips to help people with blood cancers sleep better and manage fatigue, stress, and pain. About 60 adults hospitalized for treatment will either get the program or standard care. The program includes a mo…

This study tests whether a drug called dexrazoxane can protect the heart from damage caused by chemotherapy in people with certain blood cancers, such as acute myeloid leukemia and high-risk myelodysplastic syndrome. About 100 participants will receive dexrazoxane alongside stand…

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 compares giving cancer treatment at home versus in the clinic for people with advanced cancers. The goal is to see if home treatment reduces stress, financial burden, and improves overall experience. About 220 participants will be randomly assigned to receive their reg…

This phase 2 trial tests whether a curcumin supplement (C3 complex with piperine) can reduce inflammation and improve symptoms in people with certain blood disorders, including CCUS, low-risk MDS, and MPNs. Thirty participants will receive either curcumin or a placebo for 12 mont…

This study tests whether telepresence robots can help children aged 5-17 with cancer attend school remotely when they can't be there in person. The robots let kids see, hear, and talk in class from their hospital or home. Researchers will track school attendance, social well-bein…

This study looks at whether using a simple quality-of-life questionnaire to decide when to give blood transfusions can reduce the number of transfusions needed for people with advanced myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who are receiving palliative ca…

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 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 tests a new way to screen patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) by analyzing their bone marrow and blood for specific biomarkers. The goal is to quickly match patients with a treatment trial that targets their cancer's unique mark…

This study collects tissue and fluid samples from up to 5,000 cancer patients to help researchers learn more about cancer and develop new tests and treatments. Samples are taken from routine procedures or from extra biopsies done just for research. The goal is to build a resource…

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…

Researchers are building a registry to collect long-term health information from adults with myelodysplastic syndromes and therapy-related acute myeloid leukemia. The study gathers details on blood counts, bone marrow tests, genetic changes, and treatments. By following these pat…

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…

Researchers in Italy are building a large registry of adults newly diagnosed with myelodysplastic syndromes, a group of blood cancers where the bone marrow does not make enough healthy blood cells. The study will collect data on up to 10,000 people to measure how many new cases o…

This study screens children and young adults up to age 22 whose leukemia has returned or is hard to treat. By testing their blood and bone marrow, doctors can identify specific genetic markers to match them with the most promising experimental treatments. The goal is to build a r…

This study looks at whether simple frailty assessments can predict how older patients (60+) with acute leukemia or MDS will fare after intensive treatments like chemotherapy, CAR T-cell therapy, or stem cell transplants. Researchers will track frailty status and compare outcomes …

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study looks at whether screening adults with leukemia for social needs (like housing or food) and offering early social work help is practical and helpful. About 80 newly diagnosed patients will either get the screening and consultation or usual care. The goal is to see if t…

This study tracks over 1,300 people with blood cancers like acute leukemia to see how many receive a stem cell transplant. Researchers want to understand the medical and personal reasons why some patients get this treatment while others do not. The goal is to identify barriers so…

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, 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 research study collects and analyzes blood and bone marrow samples from children and young adults with leukemia, myelodysplastic syndrome, or myeloproliferative syndrome. By reading the genetic instructions in these cancer cells, scientists hope to understand why the disease…

This pilot study is testing whether fecal microbiota transplantation (FMT) — giving processed stool from a healthy donor via enema — is safe and doable in 20 patients with lymphoma or leukemia who are receiving CAR-T therapy or stem cell transplants. The goal is to see if at leas…

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 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 aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

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 is testing whether a specialized blood test (MAIPA) can accurately identify autoimmune thrombocytopenia (ITP) in patients with low platelet counts. Currently, ITP is diagnosed by ruling out other causes, which can be slow and uncertain. Researchers will collect blood s…

This study is creating a collection of blood and bone marrow samples from 1,000 adults with acute myeloid leukemia, acute lymphoblastic leukemia, or high-risk myelodysplastic syndrome. The samples will be used by researchers to better understand these diseases and find new ways t…

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 70 people receiving a stem cell transplant for leukemia or myelodysplastic syndrome. Researchers will test their sense of smell, taste, mouth pain, and saliva production before and after the transplant. The goal is to understand how the treatment changes these …

This study looks for inherited gene mutations that may raise the risk of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Researchers will use advanced genetic testing to compare DNA from affected and unaffected family members. The goal is to find new or known mu…

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 looks at how the body absorbs, breaks down, and gets rid of an experimental drug called DSP-5336. It involves 8 adults with advanced blood cancers (like leukemia or myeloma) that no longer respond to standard treatments. Participants take a single radioactive-labeled p…

This study is for 80 adults with blood cancers like MDS or AML who are starting treatment. Participants wear a smartwatch and answer questionnaires to track their activity, symptoms, and quality of life. The goal is to help doctors better understand when patients need blood trans…

This study is for people with neuroendocrine tumors who are about to receive or have already received peptide receptor radionuclide therapy (PRRT). Researchers want to find out if certain genetic changes in the blood can predict who might later develop myelodysplastic syndrome (M…

This study collects and analyzes genetic and molecular data from 250 people with various blood cancers, including leukemia and lymphoma. Researchers will test cancer cells in the lab with many drugs to see which ones work best. The goal is to match genetic features with drug resp…

This study is collecting bone marrow, blood, and tissue samples from over 1,000 people with various blood cancers. Researchers will use advanced genetic and protein analysis to identify unique markers for each cancer type. They will also test cancer cells against a library of 300…

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 is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

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 creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

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 aims to understand why people with myelodysplastic syndromes (MDS), a type of blood cancer, are more likely to develop heart disease or stroke. Researchers will measure inflammation-related proteins in the blood of 30 adults with MDS over 6 months to see if these marke…

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 how metabolism in cancer and immune cells influences the bone marrow environment and treatment resistance in blood cancers like leukemia and myelofibrosis. Researchers will analyze samples from 350 participants to measure metabolites, lipids, and gene activity…

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…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study aims to develop special lab tests that can find tiny numbers of cancer cells left behind after treatment for acute myeloid leukemia (AML) or high-grade myelodysplastic syndrome (MDS). Researchers will follow 400 patients over time to see if these tests can predict whic…

This study follows 1,000 people who previously received an experimental cell therapy (TSC-100 or TSC-101) for blood cancers like leukemia. Researchers will track side effects and survival for up to 15 years to see if the treatment remains safe and effective over time. No new trea…