Participants will take the study drug by mouth each day while doctors regularly check for side effects, collect blood samples, and monitor how the medicine moves through the body. Any serious side effects that limit the amount of drug that can be given (dose‑limiting toxicity) are recorded, and doctors look for signs that the disease is responding using accepted criteria. The study continues until sufficient information on safety and appropriate dosing is gathered.

The purpose of this study is to evaluate the safety and feasibility of using this specific combination of medications before receiving stem cells from a donor (called allogeneic stem cell transplantation). The treatment involves receiving these medications through different methods – some are given as tablets by mouth, while others are administered through an intravenous line directly into the bloodstream.

During the study, patients will receive a carefully planned sequence of treatments over several days. Treosulfan will be given for three days, Fludarabine for five days, and Venetoclax for seven days. After this preparation phase, patients will receive stem cells from their matched donor. The doctors will then monitor the patients’ progress and how well the transplanted cells are working in their body.

The purpose of the study is to compare the effectiveness and safety of the two treatments in reducing the need for red blood cell transfusions. Participants are randomly assigned to receive either elritercept or epoetin alfa and will be treated for up to 24 weeks. During this time, they will have regular clinic visits where blood samples are taken to check hemoglobin levels, which indicate how well the blood is carrying oxygen, and doctors will record whether transfusions are still needed.

The study involves a series of visits scheduled roughly every few weeks. At each visit, a healthcare professional will administer the injection, perform a quick blood test, and ask about any side effects. The overall goal is to see if participants can stay without transfusions for a sustained period while their hemoglobin improves.

The purpose of this study is to learn how safe AZD3632 is, how well the body tolerates it, how it moves through the body over time, and whether it might help treat these blood cancers. The study is divided into different modules or parts. The first module tests AZD3632 by itself to find the best dose that is both safe and effective. This module also includes a smaller part that looks at whether eating food affects how the body absorbs the medication. The second module tests AZD3632 when given together with posaconazole to see if this combination is safe and to understand how the two medications interact in the body.

During the study, participants will take the study medication and visit the clinic regularly for check-ups. These visits will include physical examinations, blood tests to check how the body is functioning, heart monitoring tests, and assessments to see how the disease is responding to treatment. The doctors will monitor for any side effects and measure various aspects of how the medication works in the body, including the levels of medication in the blood at different times. For participants with acute leukemia, the doctors will look for signs that the cancer is responding, such as a reduction in cancer cells in the bone marrow and blood. For those with myelodysplastic syndromes, the doctors will check whether blood cell counts improve and whether participants need fewer blood transfusions. The study will also track how long any positive responses last and overall survival.

The medication being studied, RVU120, comes in the form of capsules that are taken by mouth. The treatment can be given either alone or combined with other medications, depending on how the patient was treated in their previous study. The maximum daily amount that can be given is 150 milligrams, and treatment may continue for up to 5 months.

During the study, doctors will monitor how well patients tolerate the medication by checking for any side effects or health problems that may occur. The study will track how long patients can continue taking the medication before they need to stop treatment for any reason. This helps researchers understand both the long-term safety and effectiveness of RVU120 in treating these types of cancer.

The treatments used in this study include several anti-cancer medications: cytarabine, daunorubicin, etoposide, cladribine, mitoxantrone, obinutuzumab, ibrutinib, venetoclax, and fludarabine. Some patients may also receive cyclophosphamide, busulfan, or treosulfan as part of their treatment. The medications are given either through intravenous infusion or as oral tablets, depending on the specific drug.

The main purpose of this study is to determine if less intensive treatment can be as effective as standard treatment in patients who show no signs of remaining disease after initial therapy. The study will monitor patients’ survival, side effects, and whether the disease returns after treatment. Participants will be followed for several years to evaluate the long-term effectiveness of these treatment approaches.

The purpose of this research is to determine if momelotinib can help patients become less dependent on blood transfusions. The study will be conducted in two parts – first to find the right dose of the medication, and then to see how well it works in reducing the need for blood transfusions over a 24-week period.

During the study, participants will receive momelotinib tablets and will be monitored for how their body responds to the treatment. The researchers will track how many blood transfusions patients need and will also check for any side effects of the medication. The total treatment period may last up to 76 weeks, during which patients will have regular check-ups to monitor their health and blood counts.

The study medication will be given as tablets taken by mouth. The treatment period will last up to 48 weeks, during which patients will receive momelotinib at doses up to 300 mg per day. The study consists of two parts: first, a safety phase to find the right dose, followed by a second phase to test how well the medication works.

During the study, doctors will monitor patients’ symptoms and response to treatment, particularly focusing on improvements in VEXAS syndrome symptoms and any changes in blood cell production. They will also track how well patients can reduce their use of steroids while taking the study medication. Throughout the treatment period, patients will have regular check-ups to monitor their health and any side effects.

The purpose of the study is to assess the safety of metformin and how feasible it is to use this treatment in patients with CCUS and low-risk MDS. Participants in the study will take metformin in the form of a coated tablet, which is taken orally. The study will last for up to 52 weeks, during which time researchers will monitor the participants’ health and any changes in their condition. The study will also look at how metformin might work to control the progression of these diseases.

Throughout the study, researchers will collect information on various health markers, such as changes in blood cell counts and other indicators of disease progression. This information will help determine if metformin could be a viable option for preventing leukemia in patients with CCUS and low-risk MDS. The findings from this study will inform the design of future research to further explore the potential benefits of metformin for these conditions.

The purpose of the study is to see if the new oral treatment is as effective as the injection form. Participants in the study will receive either the oral treatment or the injection, and then switch to the other form during the study. This is known as a crossover study. The study will take place over several phases, starting with a dose-escalation phase to determine the best dose of the oral treatment, followed by a phase where the effectiveness of the oral treatment is compared to the injection.

Throughout the study, researchers will monitor participants for any side effects and measure how well the treatment works in controlling the diseases. The study aims to provide a more convenient treatment option for patients with these conditions, potentially improving their quality of life by offering an oral medication instead of injections. The study is expected to continue until 2027.

Participants in the study will receive CCS1477 in the form of a capsule taken by mouth. In some cases, it will be combined with other medications, such as Dexamethasone or Pomalidomide. The trial will monitor the safety and tolerability of these treatments, which means checking for any side effects and how well patients can handle the medication. The study will also look at how the drug is processed in the body and its potential effects on the cancer.

The trial will take place over several years, with regular check-ups and assessments to track the progress of the treatment. This includes looking at changes in the cancer through various tests and evaluations. The goal is to gather information on the drug’s safety and its impact on the cancer, which could help in developing new treatment options for these serious conditions.

The purpose of the study is to assess how well the combination of Lisaftoclax and Azacitidine works in patients who have been newly diagnosed with HR-MDS. Participants in the study will receive either the combination of Lisaftoclax and Azacitidine or a placebo with Azacitidine. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment until the study is completed. This helps ensure that the results are unbiased and reliable.

Throughout the study, participants will be monitored regularly to track their response to the treatment and any side effects they may experience. The study aims to provide valuable information on the potential benefits and safety of using Lisaftoclax in combination with Azacitidine for treating HR-MDS, which could lead to improved treatment options for patients with this condition in the future.

The purpose of the study is to evaluate how effective Ivosidenib is in improving the time patients remain free from events like disease relapse or death. Participants in the study will receive Ivosidenib as a maintenance therapy for up to 24 months after undergoing a procedure called allogeneic stem cell transplantation (alloSCT), which involves receiving healthy stem cells from a donor. The study will compare the outcomes of patients taking Ivosidenib with those who do not receive this treatment.

Throughout the study, participants will be monitored for various outcomes, including their overall survival and the rate at which they remain free from disease events. The study will also track any side effects or complications that may arise from the treatment. This research aims to provide valuable insights into the potential benefits of Ivosidenib for patients with AML or HR-MDS who have the IDH1 mutation.

The purpose of the study is to evaluate how effective elritercept is in reducing the number of red blood cell transfusions needed by participants. The study will involve participants receiving either the treatment or a placebo, and their progress will be monitored over a period of time. The main goal is to see if participants can achieve transfusion independence, meaning they do not need transfusions for at least eight weeks during the study period.

Participants will be closely monitored for any side effects or changes in their health, including laboratory tests, vital signs, and heart function tests. The study aims to provide valuable information on the safety and effectiveness of elritercept for treating transfusion-dependent anemia in people with myelodysplastic syndromes.

Participants in the study will receive an infusion of RNK001 natural killer cells, which are specially prepared outside the body, and may also receive aldesleukin to help boost the immune response. The study will be conducted in two phases. In the first phase, the focus will be on assessing the safety and any side effects of the treatment. In the second phase, the study will look at how well the treatment works in controlling the disease.

The treatment involves a non-myeloablative immunosuppressive conditioning regimen, which means it is designed to suppress the immune system without completely destroying the bone marrow. This approach helps the body accept the new cells. The study will monitor participants closely to understand how the treatment affects their condition and to ensure their safety throughout the process. The trial aims to provide insights into the potential benefits of combining RNK001 and aldesleukin for patients with AML and related conditions.

The purpose of the study is to evaluate the effectiveness of a combination of medications used during the transplantation process. The medications being studied are Fludarabine, Treosulfan, and Melphalan. Fludarabine is used in combination with either Treosulfan or Melphalan to prepare the body for the transplant. These medications are given through an intravenous infusion, which means they are delivered directly into the bloodstream through a vein. The study will compare the outcomes of using Treosulfan versus Melphalan in combination with Fludarabine to see which is more effective in helping patients with AML and MDS.

Participants in the study will receive one of the two treatment combinations and will be monitored for their response to the treatment. The study will also look at the prevention of a condition called Graft-versus-Host Disease (GvHD), which can occur after a transplant when the donor cells attack the recipient’s body. The study aims to find the best treatment approach to improve survival rates and reduce complications for patients undergoing allogeneic transplantation for AML and MDS.

The purpose of the study is to evaluate how well Luspatercept can help patients achieve a period of at least eight weeks without needing a blood transfusion within the first 24 weeks of treatment. The study will also look at the safety and tolerability of Luspatercept, as well as its effects on reducing the number of transfusions needed, increasing hemoglobin levels, and improving quality of life. Participants will receive either Luspatercept or a placebo, and their progress will be monitored over a period of time to assess these outcomes.

Participants in the study will receive regular injections and attend scheduled visits to monitor their health and response to the treatment. The study aims to provide valuable information on whether Luspatercept can be an effective treatment option for patients with anemia due to MDS with del5q, who are resistant or intolerant to previous treatments and require regular blood transfusions.

Participants in the study will be randomly assigned to receive either ivosidenib or azacitidine. Ivosidenib is taken orally as a film-coated tablet, while azacitidine is administered as a powder mixed into a liquid for injection. The study will last for a period of up to 48 weeks, during which the effects of the treatments will be monitored. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatments.

The study aims to observe how well the treatments work in achieving remission, which means reducing or eliminating signs of the disease. It will also look at other outcomes such as overall survival, quality of life, and the rate at which the disease progresses to a more severe form called acute myeloid leukemia (AML). Participants’ health and response to the treatments will be closely monitored throughout the study to gather comprehensive data on the safety and effectiveness of ivosidenib and azacitidine in treating myelodysplastic syndromes with an IDH1 mutation.

The study is designed to be conducted in two parts. In the first part, participants will receive multiple doses of ION440 to assess how their bodies respond to the treatment. This includes monitoring for any side effects and changes in health indicators such as vital signs and neurological function. The second part of the study will continue to monitor participants who have completed the first part, ensuring that the treatment remains safe and well-tolerated over a longer period.

Throughout the study, researchers will collect information on how ION440 is processed in the body, including its concentration in the blood and spinal fluid. This will help determine the appropriate dosage and frequency of administration. The study aims to provide valuable insights into the potential benefits and risks of using ION440 as a treatment for MECP2 Duplication Syndrome, ultimately contributing to better management of this condition.

The main treatment being studied is Azacitidine, which is given as an injection under the skin. Other medications that may be used in the study include Fludarabine, Daunorubicin, Busulfan, Cytarabine, and Thiotepa. These medications are given through an intravenous line directly into the bloodstream.

The purpose of this research is to determine whether it is better to perform stem cell transplantation immediately or to first treat patients with medications like Azacitidine or traditional chemotherapy, depending on the number of abnormal cells in their bone marrow. The study will monitor patients’ health and recovery throughout their treatment period and follow-up care.

The treatment involves taking EP0042 in capsule form, either by itself or together with two other medications: venetoclax (taken as tablets) and azacitidine (given as an injection). The study aims to find the right dose of EP0042 that is both safe and potentially effective for treating these blood cancers.

During the study, patients will receive the medications and be monitored regularly to check how well they tolerate the treatment and how their disease responds. Doctors will track any side effects and measure how the medications work in the body. The study will also look at how long patients respond to the treatment and their overall survival.

The medication being tested is called luspatercept (Reblozyl), which is given as an injection under the skin. This treatment aims to improve the body’s ability to produce red blood cells and increase hemoglobin levels in patients with MDS. The medication will be given to patients for up to 24 weeks to see if it can help improve their anemia.

The study will measure how well the treatment works by checking if patients’ hemoglobin levels increase by a certain amount and if this improvement lasts for at least 8 weeks. Patients will receive subcutaneous injections of luspatercept at doses that may be adjusted during the study period. The treatment period may continue for up to 18 months for patients who show improvement in their condition.

The purpose of the study is to evaluate the safety and effectiveness of CA-4948 when used alone in patients with relapsed or refractory AML and higher-risk MDS. The study will be conducted in two phases. In the first phase, the focus is on determining the safest dose of the medication and understanding how well patients tolerate it. The second phase aims to assess the anti-cancer activity of CA-4948 in patients with specific genetic mutations, such as FMS-like tyrosine kinase-3 (FLT-3) mutations and spliceosome mutations like SF3B1 or U2AF1.

Participants in the study will receive the medication and be monitored for any side effects or changes in their condition. The study will also look at how the medication affects the body over time, including its concentration in the blood and how long it stays active. The trial is expected to continue until July 2025, allowing researchers to gather comprehensive data on the treatment’s impact on these serious blood disorders.

The purpose of the study is to evaluate the safety and tolerability of different doses of elritercept. The study is divided into two main parts. In the first part, participants will receive increasing doses of elritercept to determine the most suitable dose for further testing. In the second part, the selected dose will be confirmed for its safety and tolerability. There is also a long-term extension phase to assess the long-term safety of elritercept in these patients.

Participants in the study will receive elritercept through subcutaneous injections, which means the medication is injected under the skin. The study will monitor participants for any side effects and how well they tolerate the treatment. The trial aims to find out if elritercept can help reduce the need for blood transfusions and improve the overall blood cell production in patients with MDS. The study will also look at how the treatment affects the progression of the disease over time.

The purpose of this study is to evaluate the safety of continuing treatment with sabatolimab for patients who have already participated in a previous study involving this medication and are considered by their doctors to benefit from ongoing treatment. The study will monitor the frequency and severity of any side effects experienced by the participants. Participants will receive sabatolimab through an intravenous infusion, which means the medication is given directly into a vein.

In addition to sabatolimab, the study may involve other medications such as azacitidine, a drug used to treat certain types of blood disorders, and venetoclax, a medication that helps to kill cancer cells by blocking a protein that allows them to survive. Azacitidine is administered as a powder mixed into a liquid for injection, while venetoclax is taken orally as a tablet. The study will continue for a period of up to 60 months, during which the participants’ health and response to the treatment will be closely monitored by the research team.

The purpose of this study is to evaluate different doses and schedules of ASTX727 in people with lower-risk MDS. The study is divided into two phases. The first phase will determine the safety of different doses and schedules, while the second phase will assess how well the selected doses work in treating the disease.

During the study, participants will take ASTX727 tablets according to a specified schedule. The treatment period may last up to 84 days, during which doctors will monitor the participants’ blood counts and overall health. The study will look at how the medication affects blood cell production and whether it helps reduce the need for blood transfusions. Researchers will also track how the body processes the medication and observe any side effects that may occur.

The purpose of this research is to gather long-term safety information about ASTX727 in patients who have already been using this medication in previous studies and have shown benefit from the treatment. The study will continue providing ASTX727 to these patients while monitoring their health and any effects of the medication.

During the study, participants will continue taking ASTX727 tablets according to their established treatment schedule. The maximum daily dose is 135 milligrams, and treatment may continue for up to 6 months. The research team will regularly check participants’ health status and record any side effects that may occur during the treatment period.

The main purpose of this research is to determine how well patients respond to different doses of Busulfan when combined with the other medications as preparation therapy before stem cell transplantation. This preparation therapy, also known as conditioning therapy, helps prepare the body to receive the donor’s stem cells.

During the study, patients will receive these medications through intravenous infusion. Busulfan will be given at different dose levels, while Fludarabine and Thymoglobuline will be administered at set doses. After receiving these medications, patients will undergo the stem cell transplantation procedure. The study will monitor how patients respond to the treatment over time, particularly focusing on whether the disease returns or progresses.

The purpose of the study is to see if the combination of these two drugs can effectively target and inhibit the cancer cells in patients with the IDH1 mutation. The study will be conducted in two phases. The first phase will determine the best dose of Molidustat Sodium to use in combination with Ivosidenib. The second phase will assess how well this combination works in achieving complete remission, which means the disappearance of all signs of cancer in response to treatment.

Participants in the study will take the medications as prescribed and will be monitored for their response to the treatment over several months. The study will also look at the safety of the treatment and any side effects that may occur. The goal is to find a new effective treatment option for patients with these specific types of blood cancer who have limited options available. A placebo may be used in some parts of the study to compare the effects of the treatment. The study is expected to continue until early 2026.

The trial will test two medications, ivosidenib and enasidenib, which are taken as film-coated tablets. These medications are designed to target the specific gene mutations in the cancer cells. The study will compare the effects of these medications when used alongside standard chemotherapy treatments. Some patients will receive a placebo instead of the active medication to help researchers understand the true effects of the drugs being tested.

The purpose of the study is to see if these treatments can improve the time patients live without the cancer getting worse, known as event-free survival. Participants will go through different phases of treatment, including induction therapy, consolidation therapy, and maintenance therapy. These phases are designed to first reduce the number of cancer cells, then stabilize the condition, and finally maintain the results over time. The study aims to provide valuable information on how effective these treatments are for patients with these specific gene mutations in AML or MDS.

The purpose of this study is to evaluate the long-term safety of luspatercept, including monitoring for any progression to more serious conditions such as acute myeloid leukemia (AML) or the development of other types of cancer. Participants in this study will receive luspatercept as a solution for injection, which is administered under the skin. The study is designed as an open-label, single-arm, rollover study, meaning all participants will receive the treatment without a comparison group receiving a placebo.

Throughout the study, participants will be monitored for any adverse events or changes in their condition. The study will also track overall survival and any new growths that may develop. The maximum treatment period for participants is up to 360 days, during which they will continue to receive the treatment and be regularly assessed by healthcare professionals. This study aims to provide valuable information on the long-term effects and safety of luspatercept for individuals with these blood disorders.

The purpose of the study is to find the most suitable dose of that can be safely given to patients and to evaluate its effectiveness in treating these blood cancers. The study is divided into two parts. In the first part, the focus is on determining the best dose for both adults and children. In the second part, the study aims to assess how well the treatment works at the recommended dose. Participants will receive the treatment and be monitored for any side effects and improvements in their condition.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how the treatment affects their cancer. The study will help researchers understand more about the potential benefits and risks of for treating these serious conditions. The information gathered from this trial could contribute to developing new treatment options for patients with these types of blood cancers.

The purpose of this study is to prevent clinical events such as relapse or death in patients who have minimal residual disease (MRD) after undergoing a procedure called allogeneic stem cell transplantation. This procedure involves replacing diseased bone marrow with healthy stem cells from a donor. The study will monitor patients to see if the treatment with Azacitidine can help reduce the risk of these events.

Participants in the study will receive Azacitidine and will be closely monitored over a period of time to assess their response to the treatment. The study aims to determine the effectiveness of Azacitidine in preventing relapse and improving survival rates in patients with MDS who are MRD positive. The trial will also look at the safety of the treatment and its impact on the overall health of the participants.

The treatment involves taking tamibarotene tablets by mouth and receiving azacitidine through injections under the skin or into a vein. Azacitidine is given at a dose of 75 mg per square meter of body surface for 7 days, while tamibarotene is given as a daily tablet. Some patients will receive a placebo tablet instead of tamibarotene, but all patients will receive azacitidine.

The study will track how many patients achieve complete remission (when signs of cancer disappear) and will also monitor how long patients survive, whether they need fewer blood transfusions, and how the treatment affects their quality of life. Doctors will regularly check patients’ blood counts and bone marrow to assess how well the treatment is working and watch for any side effects.

The purpose of the study is to evaluate the safety and effectiveness of the combination of ALX148 and Azacitidine in treating patients with higher-risk MDS. The study is divided into two phases. In the first phase, researchers will determine the best dose of ALX148 to use with Azacitidine. In the second phase, they will compare the effects of the combination treatment to Azacitidine alone. Participants will receive the study treatments and be monitored for their response to the treatment and any side effects they may experience.

Throughout the study, participants will have regular visits to the clinic for treatment and monitoring. The study aims to understand how well the combination of ALX148 and Azacitidine works in improving the condition of patients with higher-risk MDS and to gather information on any potential side effects. The trial will help determine if this combination can be a more effective treatment option for patients with this type of blood disorder.

The purpose of the study is to evaluate how well RVU120 can help improve anemia in patients with lower-risk MDS. Participants in the study will receive the medication over a period of time, and their response to the treatment will be monitored. The study will look at how the drug affects the production of red blood cells and whether it can reduce the need for blood transfusions. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of RVU120.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment. The study aims to gather information on the safety and effectiveness of RVU120 in treating anemia associated with MDS. This research could potentially lead to new treatment options for patients with this condition.

The purpose of the study is to assess how well canakinumab works and how safe it is for patients with these conditions. Participants in the study will receive canakinumab over a period of time, and their response to the treatment will be monitored. The study will look at whether there is an improvement in hemoglobin levels, which is a measure of red blood cells in the blood, and whether this improvement lasts for at least eight weeks. The study will also observe if patients can avoid needing blood transfusions during this time.

This trial is open-label, meaning both the researchers and participants know that canakinumab is being administered. The study is designed to gather information on the effectiveness of canakinumab in treating anemia associated with lower-risk MDS or MDS/MPN, providing valuable insights into potential new treatment options for these conditions.

In this study, patients with AML or MDS will receive a stem cell transplant from a donor who is a close match. Before the transplant, patients will undergo a reduced intensity conditioning regimen, which is a less aggressive form of treatment to prepare the body for the transplant. This regimen includes the use of two medications, Fludarabine and Treosulfan. The purpose of the study is to see which of the two treatments, Thymoglobulin or Grafalon, is more effective in preventing GVHD in these patients.

Participants in the study will receive either Thymoglobulin or Grafalon as part of their treatment plan. The study will monitor the occurrence of GVHD and other health outcomes for a period of time after the transplant. This includes checking for any signs of GVHD, monitoring blood cell recovery, and assessing overall health and quality of life. The study will help determine the best approach to prevent GVHD in patients with AML or MDS undergoing stem cell transplants.

The purpose of the study is to see how well these treatments work in people with lower-risk MDS who are not dependent on blood transfusions. Participants in the study will receive either Luspatercept or Epoetin Alfa through injections under the skin. The study will last for a period of up to 96 weeks, during which participants will be monitored to see if they need fewer blood transfusions and if their hemoglobin levels, which measure the amount of red blood cells, improve.

Throughout the study, participants will be regularly assessed to ensure their safety and to track the effectiveness of the treatments. The study will help determine which treatment is more effective in managing anemia in patients with MDS who have not previously been treated with erythropoiesis-stimulating agents, which are medications that help the body produce more red blood cells. This research is important for improving treatment options for people living with MDS.

The purpose of the study is to explore the safety and how well patients tolerate MP0533, as well as to find the best dose to use in future studies. The study will also look at how effective the treatment is in fighting the cancer. Participants will receive the treatment and be monitored closely to see how their bodies respond. The study will take place over several phases, starting with a small number of patients to determine the safest dose, and then expanding to include more patients to further assess the treatment’s effects.

Throughout the study, researchers will keep track of any side effects and changes in the patients’ health. The goal is to gather information that could lead to new treatment options for people with AML and MDS. This study is an important step in understanding how MP0533 can be used to help patients with these challenging conditions.