Myelodysplastic syndrome

Researchers test a reduced-intensity conditioning regimen before allogeneic stem cell transplant in adults with high-risk AML, MDS, or ALL who cannot tolerate standard intensive treatment. The regimen combines clofarabine, busulfan, and thymoglobulin. The trial measures relapse r…

Researchers are testing a vaccine called 3D189 in adults with blood cancers such as acute leukemia, multiple myeloma, non-Hodgkin lymphoma, and higher-risk myelodysplastic syndrome. Participants must be in remission after standard treatment and unable to receive a stem cell trans…

Researchers are testing a drug called APR-246 combined with azacitidine in people with myeloid blood cancers that carry a TP53 mutation, including myelodysplastic syndrome, acute myeloid leukemia, myeloproliferative neoplasms, and chronic myelomonocytic leukemia. The trial aims t…

Researchers are testing whether a liposomal chemotherapy drug called CPX-351 works better than standard chemotherapy before an allogeneic stem cell transplant in adults with higher-risk myelodysplastic syndromes (MDS) or oligoblastic acute myeloid leukemia (AML). The trial enroll…

This early-phase trial tested the drug enasidenib in 17 people with low-risk myelodysplastic syndrome or a related leukemia who had anemia and did not have a specific gene mutation. The goal was to see if the drug could safely improve red blood cell counts and reduce the need for…

This early-phase trial tests whether specially trained immune cells from a donor can safely target and kill cancer cells in people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after a stem cell transplant. The cells are grown in a lab to recognize four prot…

This study tests a new approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Patients receive a combination of umbilical cord blood and stem cells from a family member, with the cord blood chosen based on specific genetic markers rather than …

This phase III trial is testing whether taking oral azacitidine as a maintenance therapy after an allogeneic stem cell transplant can help prevent relapse in people with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Participants are randomly assigned to receive…

This phase 1 trial is testing an investigational drug called AEB1102 in adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has returned or stopped responding to standard treatments. AEB1102 is a modified human enzyme that breaks down arginine, an amin…

This trial is testing whether a chemotherapy drug called treosulfan, given in different doses along with fludarabine, can safely prepare patients with advanced blood cancers for a stem cell transplant. The study focuses on patients who are not eligible for the standard, more inte…

This early-stage trial tests a combination of two drugs, venetoclax and azacitidine, in people with a serious type of myelodysplastic syndromes (MDS) who have not had treatment before. The goal is to find the safest dose and see how well the drugs work together to control the dis…

This trial is testing an experimental drug called BST-236 in adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) that have come back or not improved after initial treatment. Participants receive BST-236 as a one-hour intravenous infusion for six …

This study tests a combination of two chemotherapy drugs (CPX-351 and gemtuzumab ozogamicin) in about 50 adults with acute myeloid leukemia (AML) that has returned or not responded to treatment, or with high-risk myelodysplastic syndrome (MDS). The goal is to see if the combo is …

This Phase 3 trial tested a stem cell transplant using blood stem cells from a half-matched (haploidentical) donor in 21 people with various blood cancers. The goal was to see if this approach could help patients who don't have a fully matched donor. Participants received chemoth…

This study tests a new drug, AZD9829, in people with blood cancers that have come back or not responded to other treatments. The drug targets a protein called CD123 found on cancer cells. The main goals are to find a safe dose and check for side effects. About 56 adults will take…

This study tests a drug called FT-2102 (olutasidenib) in people with acute myeloid leukemia or myelodysplastic syndrome whose cancers have a specific IDH1 gene mutation. The drug is given alone or combined with standard chemotherapy drugs (azacitidine or cytarabine). The goal is …

This study tested a daily pill called E7820 in 12 adults with relapsed or treatment-resistant acute myeloid leukemia, myelodysplastic syndromes, or chronic myelomonocytic leukemia. All participants had specific mutations in splicing factor genes. The goal was to see if the drug c…

This study tested the drug RAD001 (everolimus) in 29 patients with relapsed or hard-to-treat blood cancers like leukemia, lymphoma, and myelofibrosis. The goal was to find the safest highest dose and see if it helps control the disease. It was a small early-phase trial at a singl…

This study tested a drug called fedratinib in 25 adults with rare blood cancers (MDS/MPN and CNL). The goal was to see if the drug could shrink the spleen and improve blood counts. While not a cure, the treatment aims to control the disease and reduce symptoms.

This study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it…

This study tested a new way to treat blood cancers like leukemia in 16 adults who did not have a standard bone marrow donor. Doctors used a single unit of cord blood that was grown in a lab to increase the number of stem cells before transplant. The goal was to see if this method…

This phase 2 trial tested a combination of intravenous busulfan, etoposide, and low-dose total-body irradiation before a donor stem cell or bone marrow transplant in 30 patients with advanced leukemia or myelodysplastic syndromes. The goal was to see how well this regimen control…

This study tested whether removing a specific type of immune cell (naïve T cells) from donor stem cell grafts could prevent graft-versus-host disease (GVHD) after a stem cell transplant for blood cancers. The trial enrolled 84 patients who received high- or medium-intensity chemo…

This early-phase trial tested a drug called ONC-201 in 20 people with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who had recently received a stem cell transplant. Participants took the drug by mouth once a week for a year to see if it was safe and co…

This study tested whether adding the drug tocilizumab to standard care could reduce the risk of graft-versus-host disease (GVHD) after a cord blood transplant in adults with blood cancers. GVHD is a serious complication where donor cells attack the patient's body. Tocilizumab blo…

This study tested a three-drug combination (navitoclax, venetoclax, and decitabine) in 6 adults with high-risk myelodysplastic syndrome (MDS) that came back or didn't respond to standard therapy. The goal was to find the safest dose and see if the combo could shrink or control th…

This study looked at 88 adults in China with lower-risk myelodysplastic syndromes (MDS) who had not received prior treatment with erythropoiesis-stimulating agents. They were given luspatercept, and researchers tracked how many became free from red blood cell transfusions and whe…

This study tested a drug called ustekinumab in 8 people with a rare, severe form of Behçet's syndrome affecting the gut, along with a blood disorder called myelodysplastic syndrome. The goal was to see if the drug could control gut symptoms and reduce disease activity over 24 wee…

This phase 1 trial tested two immunotherapy drugs, nivolumab and ipilimumab, in 29 patients whose acute myeloid leukemia or myelodysplastic syndrome came back or did not respond after a donor stem cell transplant. The goal was to find the safest dose and see if the drugs could he…

This study tested whether an oral medication (CC-486) could help elderly patients with a serious blood disorder called myelodysplastic syndrome (MDS) maintain their improvement after initial injectable treatment. Eleven patients who had responded to injectable azacitidine switche…

This study tested a drug called luspatercept in 30 adults from China and Japan who have a bone marrow disorder (MDS) and need regular red blood cell transfusions. The goal was to see if the drug could help them go without transfusions for at least 8 weeks. The drug works by helpi…

This early-stage study tested a new drug called LYT-200 in 101 adults whose acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) had come back or stopped responding to treatment. The main goals were to check the drug's safety and find the right dose. While not…

This early-stage trial tested an oral drug called NTX-301 in 22 adults with certain blood cancers (MDS, AML, or CMML) that had not responded to prior treatments. The main goal was to check safety and find the right dose. Researchers also looked for signs that the drug might help …

This study tested different drug combinations to prevent graft-versus-host disease (GVHD) in 174 people with blood cancers receiving stem cells from an unrelated donor. GVHD is a serious side effect where donor cells attack the patient's body. The goal was to find which drug regi…

This early-phase trial tested a combination of two cancer drugs, bortezomib and clofarabine, in 28 adults with advanced solid tumors, lymphomas, or myelodysplastic syndromes that had stopped responding to standard therapy. The main goal was to find the safest dose and check for s…

This study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…

This study tested a personalized geriatric optimization plan (GO!) in 30 adults aged 60 and older who were getting a donor stem cell transplant for blood cancers or bone marrow failure. The plan included tailored advice on diet, sleep, activity, and medications to improve physica…

This study tested whether adding pacritinib to standard immune-suppressing drugs (sirolimus and tacrolimus) could prevent graft-versus-host disease (GVHD) in 40 patients receiving stem cell transplants for blood cancers. GVHD occurs when donor immune cells attack the recipient's …

This study tested an oral drug called ASTX727 in people with lower-risk myelodysplastic syndromes (MDS), a bone marrow disorder. The goal was to see if a lower dose given over a longer schedule could improve blood cell counts and reduce the need for transfusions. The trial enroll…

This phase II trial tested whether adding the drug itacitinib to standard medications (tacrolimus and sirolimus) can better prevent graft-versus-host disease (GVHD) in 59 patients with blood cancers receiving donor stem cell transplants. GVHD occurs when donor immune cells attack…

This study tested a new method of cord blood transplantation in 13 children and young adults with high-risk myeloid cancers. The approach uses a molecule called UM171 to grow more stem cells from a single cord blood unit, aiming to speed up recovery and reduce complications. Earl…

This study tested a new way to prepare cord blood for transplant in 30 people with high-risk leukemia or myelodysplasia. The cord blood was treated with a molecule called UM171 to help it grow more stem cells. The goal was to see if this approach is safe and helps patients recove…

This phase 2 trial tested a two-step stem cell transplant approach for 47 patients with high-risk blood cancers like leukemia and lymphoma. The method uses the drug cyclophosphamide to lower the chance of graft-versus-host disease (GVHD), a common complication. The goal was to se…

This early-phase trial tested a new type of stem cell transplant called a microtransplant in 25 people with myeloid blood cancers (like leukemia) who were too old or frail for a standard transplant. Unlike a regular transplant, this one used stem cells from a partially matched fa…

This early-phase trial tested a combination of two drugs, ibrutinib and lenalidomide, in 4 patients with myelodysplastic syndrome (MDS) who had not responded to or could not tolerate standard treatments. The main goal was to find the safest dose and identify side effects. While t…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This early-phase trial tested a cancer vaccine (CDX-1401) along with an immune booster, chemotherapy, and an immunotherapy drug in 8 people with myelodysplastic syndrome (MDS) or low-blast acute myeloid leukemia (AML). The goal was to see if the combination is safe and helps the …

This Phase 3 trial tested a special cord blood product called NiCord (omidubicel) against standard cord blood transplants in 125 people with blood cancers like leukemia and lymphoma. The goal was to see if NiCord helps the body rebuild its infection-fighting cells faster. Researc…

This early-phase trial tested a new treatment for people with myelodysplastic syndrome (MDS) or secondary acute myeloid leukemia (AML) who had not responded to prior therapy. The treatment involved giving specially prepared donor immune cells (with CD8 cells removed to reduce ris…

This early-stage study tested a new drug called PRT2527, alone or with other cancer drugs (zanubrutinib or venetoclax), in 86 people with various blood cancers that had returned or stopped responding to standard treatments. The main goals were to check safety, find the best dose,…

This early-phase study tested an experimental oral drug called LP-108, given alone or with another drug (azacitidine), in 32 adults with blood cancers (MDS, CMML, or AML) that returned or didn't respond to prior therapy. The main goals were to find the safest dose and understand …

This early-phase trial tested a combination of two chemotherapy drugs, bortezomib (Velcade) and lenalidomide (Revlimid), in 22 adults whose acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) had returned after a stem cell transplant. The main goal was to find the safe…

This early-phase study tested the drug regorafenib in 16 adults with advanced blood cancers like acute myeloid leukemia (AML) that had stopped responding to other treatments. The main goal was to find the safest dose and see how the drug affects cancer-related cell signals. While…

This study tested whether a low dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (GVHD) — a common and serious complication where donor cells attack the patient's body. The trial involved 35 adults with various blood dis…

This study tested whether the antibiotic levofloxacin can prevent fevers and infections in people with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) who are receiving the chemotherapy drug azacitidine. Infections are a major risk for these patients. The trial ran…

This study tested the drug dasatinib in 68 adults with several types of blood and bone marrow disorders, including certain leukemias, myelofibrosis, and mastocytosis. The goal was to see if dasatinib could help control the disease and to check its safety. Participants took dasati…

This study tested a new drug called VIC-1911 added to standard medications to prevent graft-versus-host disease (GVHD) in 16 adults with blood cancers receiving a stem cell transplant. The goal was to find a safe and effective dose that reduces GVHD and relapse. Results are still…

This study tested whether adding ready-made natural killer (NK) cells from a donor to a standard stem cell transplant could safely reduce the chance of cancer returning in people with high-risk myeloid cancers like acute myeloid leukemia, myelodysplastic syndrome, and chronic mye…

This early-stage trial tested a new oral drug, JNJ-64619178, in 114 people with advanced cancers (including solid tumors, lymphoma, and myelodysplastic syndromes) that had stopped responding to standard treatments. The main goals were to find a safe dose and check for side effect…

This study tested whether adding the drug abatacept to standard medications (calcineurin inhibitor and methotrexate) could better prevent severe acute graft-versus-host disease (aGVHD) after a stem cell transplant from an unrelated donor. A total of 186 participants aged 6 and ol…

This phase II trial tested two drugs—sirolimus and azacitidine—in 57 people with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that had come back or didn't respond to standard treatment. The goal was to see if the combination could shrink or control the…

This study tested a stem cell transplant from half-matched (haploidentical) family donors for 34 people with various blood cancers. Patients received a reduced-intensity conditioning regimen of fludarabine, melphalan, and total body irradiation before the transplant. The goal was…

This study tested adding a drug called thiotepa to the usual chemotherapy given before a donor stem cell transplant for people with leukemia. The goal was to see if this stronger mix could help more patients stay cancer-free one year after the transplant. The study included 40 ad…

This early-phase trial tested replacing the standard drug cyclophosphamide with bendamustine after a half-matched bone marrow transplant in 50 patients with various blood cancers. The goal was to see if this change could reduce side effects like graft-versus-host disease while st…

This study tested a drug called guadecitabine combined with donor lymphocyte infusions in 55 patients whose acute myeloid leukemia or myelodysplastic syndrome came back after a stem cell transplant. The goal was to see if the drug could help the donor immune cells fight the cance…

This study tested a two-step stem cell transplant for people with high-risk blood cancers like leukemia and lymphoma. First, patients got low-dose chemotherapy and radiation to prepare the body. Then, they received donor stem cells and immune cells to help destroy remaining cance…

This study tested whether adding pevonedistat to the standard drug azacitidine helps people with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia, or low-blast acute myeloid leukemia live longer without their disease getting worse. About 454 adults took part…

This phase 1 study tested the drug enasidenib as a maintenance therapy after a stem cell transplant in 23 people with IDH2-mutant acute myeloid leukemia or chronic myelomonocytic leukemia. The goal was to find the safest dose and see how well patients tolerated it. Enasidenib wor…

This early-stage study tested a new drug called JNJ-74856665 in 153 people with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML). The main goals were to find safe doses and check for side effects when given alone or with oth…

This study tested whether a combination of two drugs, cyclophosphamide and abatacept, could lower the risk of chronic graft-versus-host disease (GVHD) after a stem cell transplant in people with high-risk blood cancers. GVHD happens when donor cells attack the patient's body. The…

This study tested whether the drug palifermin could make stem cell transplants safer and reduce chronic graft-versus-host disease (GVHD), a serious complication where donor cells attack the patient's body. Thirty-four adults with blood cancers received palifermin before transplan…

Researchers are testing whether giving azacitidine, a chemotherapy drug, together with donor lymphocyte infusions after a stem cell transplant can prevent relapse in people with high-risk acute myeloid leukemia or myelodysplastic syndrome. About 30 patients will receive the combi…

This study tested a drug called abatacept, given as a shot under the skin, to prevent a serious complication called graft-versus-host disease (GVHD) in 29 adults with blood cancers. Participants received a stem cell transplant from a half-matched donor. The goal was to see if aba…

This study tests an electronic program that combines mindfulness and music therapy for people undergoing stem cell transplants for blood cancers. The goal is to see if it improves quality of life and reduces symptoms like pain. About 60 adults will participate, and the program in…

This study looked at whether providing free blood transfusions to blood cancer patients would encourage them to enroll in hospice care earlier. Many patients delay hospice because they need transfusions for symptom relief, but hospices often cannot afford to provide them. The stu…

This study looks back at medical records of people who had an allogeneic stem cell transplant to see whether romiplostim helped their platelets recover faster. Platelets are blood cells that help clotting, and low counts are common after transplant. The researchers will compare p…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This completed study looked at how immune cells recover in 61 adults who received a stem cell transplant for blood cancers. Researchers measured the function of T and NK cells by testing their cytokine profiles after stimulation. The goal was to see if these measurements could he…

This completed study in Japan looked at how low-risk myelodysplastic syndrome (MDS) or unexplained anemia affects patients' daily lives. Researchers used questionnaires and interviews with 56 participants to understand symptoms, quality of life, and unmet needs. The goal was to c…

This study looked at how kidney function and race/ethnicity affect the drug treosulfan in 20 patients with AML or MDS getting a stem cell transplant. The goal was to find safe doses for those with kidney problems. It was a small, early-phase study that did not test a new treatmen…

This study looked at how often people with certain blood cancers or related conditions have an acquired deficiency of an enzyme called pyruvate kinase, which can cause anemia. Researchers took a single blood sample from 18 participants to measure enzyme activity and check for rel…

This study looked at 308 people with myelodysplastic syndromes (MDS) who had a stem cell transplant. Researchers identified each patient's unique genetic mutations and then monitored those mutations in blood and bone marrow samples after transplant. The goal was to see if rising …

This study collected blood, bone marrow, skin, and cheek cell samples from 650 adults with acute leukemia, myelodysplastic syndromes, or myeloproliferative disorders. Researchers analyzed the samples to find genetic, epigenetic, and drug sensitivity patterns that might predict ho…

This study tested whether a telehealth program could help older patients (60+) with acute myeloid leukemia or myelodysplastic syndrome discuss and document their future medical wishes. Researchers enrolled 36 patients, caregivers, and clinicians to see if the program was easy to …

This study looked back at medical records of 418 patients with lower-risk myelodysplastic syndromes (MDS) who had not previously used erythropoiesis-stimulating agents (ESAs). Researchers examined how luspatercept was used in everyday practice, including dosing, treatment changes…

This study measured how often iron builds up in the heart muscle of people who receive many blood transfusions. Researchers used MRI scans to check heart iron levels in 110 patients with thalassemia, sickle cell disease, or myelodysplasia. The goal was to understand how common th…

This completed study enrolled over 2,100 people with or suspected of having myelodysplastic syndromes (MDS), a group of blood disorders. Researchers collected medical data, blood, and tissue samples over time to track how the disease develops and progresses. The goal was to creat…

This study looked at 1,753 patients needing a stem cell transplant for blood cancers or other serious blood disorders. Researchers compared two strategies: first searching for a matched unrelated donor, then trying an alternative donor if needed, versus going straight to an alter…

This early-stage study tested the safety and tolerability of a new drug called LP-001 in 68 healthy adults aged 18 to 50. The goal was to see how the body handles single and multiple doses, not to treat any disease. Results will help guide future research for myelodysplastic synd…

This study looked at how 177 people with lower-risk myelodysplastic syndromes (MDS) are treated in real-world clinics across Japan. Researchers collected data on treatments, blood transfusions, and healthcare costs to understand current practice patterns. The goal was to describe…

This study looked at 126 people aged 60 and older who had a bone marrow transplant for blood cancers like leukemia or lymphoma. Researchers measured physical abilities, mental health, and stress levels before and after transplant to find traits that help people recover better. Th…

This study enrolled 349 people with myelodysplastic syndromes (MDS) to see how certain gene mutations affect survival. Researchers collected blood samples at diagnosis and tracked patients over time. The goal was to understand which mutations are linked to better or worse outcome…

This completed study involved 146 patients with blood cancers who received a stem cell transplant from a donor. Researchers collected blood and tissue samples over a year to study immune cells linked to graft-versus-host disease (GVHD), a common complication where donor cells att…