Myelodysplastic syndrome with excess blasts

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 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 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 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 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 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 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 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 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 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 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 study tests a radioactive antibody (211At-BC8-B10) that seeks out and attacks cancer cells, followed by a donor stem cell transplant for people with high-risk leukemia or myelodysplastic syndrome (MDS) that has returned or not responded to treatment. The goal is to find the …

This study is testing a new approach for people with high-risk acute leukemias or myelodysplastic syndrome. Before a donor stem cell transplant, patients receive a radioactive antibody (211At-BC8-B10) designed to target and kill cancer cells while sparing healthy tissue. The goal…

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

This early-stage study tests an experimental drug called APL-4098, alone or with standard treatments, in adults with acute myeloid leukemia (AML) or related blood disorders. The goal is to find a safe dose and see if it shrinks cancer. About 100 people will take part.

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 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 is testing an experimental oral drug called ASTX030 (a combination of azacitidine and cedazuridine) for people with certain blood cancers like myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The drug is given a…

This study tests whether 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 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 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 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 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 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 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 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 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 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 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 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 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 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…