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Ongoing Clinical Trials for Myelodysplastic Syndrome

This article provides detailed information about 62 ongoing clinical trials focused on myelodysplastic syndrome, a group of blood disorders where the bone marrow doesn’t produce enough healthy blood cells. These trials are testing various treatments including new medications, drug combinations, and stem cell transplantation approaches across multiple countries in Europe and beyond. (Also known as: MDS, Myelodysplastic Syndromes)

Clinical trial locations

Study Comparing Luspatercept and Epoetin Alfa for Treating Anemia in Patients with Low-Risk Myelodysplastic Syndrome Who Are Not Dependent on Blood Transfusions

This trial is comparing two medications for treating anemia in patients with low-risk myelodysplastic syndrome who don’t need regular blood transfusions. Patients will receive either luspatercept or epoetin alfa, both given as subcutaneous injections.

Main inclusion criteria: Patients must be at least 18 years old with confirmed MDS diagnosis, have low to intermediate risk according to IPSS-R, and have a serum erythropoietin level of 500 U/L or less. They must have baseline hemoglobin of 9.5 g/dL or lower and show symptoms of anemia.

Main exclusion criteria: Patients with other blood disorders, those who have received erythropoiesis-stimulating agents before (with certain exceptions), or those with conditions that would make treatment unsafe.

Primary goal: The study aims to evaluate if luspatercept can achieve transfusion independence for at least 8 weeks within the first 24 weeks while also increasing hemoglobin levels by 1.5 g/dL or more.

Treatment approach: Luspatercept works by helping increase red blood cell production, while epoetin alfa stimulates the bone marrow to produce more red blood cells. The trial will monitor patients’ hemoglobin levels and transfusion needs throughout the treatment period.

Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia

This trial compares a new oral medication combination (ASTX030) with the standard injectable form of azacitidine for treating blood disorders including MDS, CMML, and AML. The oral form combines azacitidine with cedazuridine to improve how the body absorbs the medication.

Main inclusion criteria: Patients must be 18 years or older with confirmed diagnosis of MDS, CMML, or AML. They should have adequate kidney and liver function, and their white blood cell count must be 10 G/L or less.

Main exclusion criteria: Patients with other serious medical conditions, those who have recently had major surgery, or those with active uncontrolled infections.

Primary goal: To establish that the oral form is as effective as the injectable form and to find the best dosage schedule for the oral medication.

Treatment approach: The trial will test different doses of the oral medication and compare its effects to subcutaneous azacitidine. Patients will undergo regular monitoring to assess their response and any side effects.

Study Comparing Thymoglobulin and Grafalon for Preventing Graft Versus Host Disease in Elderly Patients with Acute Myeloid Leukemia or Myelodysplastic Syndrome

This trial compares two medications (ATG-thymoglobulin and ATLG-grafalon) used to prevent graft-versus-host disease in older patients with AML or MDS who are undergoing stem cell transplantation. Both medications help suppress the immune system to prevent it from attacking the transplanted cells.

Main inclusion criteria: Patients must be between 50 and 70 years old with AML or MDS requiring stem cell transplant. They must have a matching unrelated donor (10/10 HLA match) and meet specific health criteria for heart and lung function.

Main exclusion criteria: Patients with severe uncontrolled infections, serious heart problems, or those who don’t meet the transplant eligibility criteria.

Primary goal: To determine which medication is more effective at preventing graft-versus-host disease while maintaining good survival rates.

Treatment approach: Patients will receive fludarabine and treosulfan as conditioning therapy, followed by stem cell transplant and prophylactic treatment with either ATG-thymoglobulin or ATLG-grafalon. Regular monitoring will track for signs of graft-versus-host disease and other complications.

Study of Azacitidine and Drug Combination Before Stem Cell Transplantation in Patients with High-Risk Myelodysplastic Syndrome

This trial evaluates different treatment approaches before stem cell transplantation for higher-risk MDS patients. It compares immediate transplantation with transplantation after receiving either azacitidine or conventional chemotherapy.

Main inclusion criteria: Patients must be 18-70 years old with higher-risk MDS that hasn’t been treated before. They must be eligible for stem cell transplantation and have adequate organ function.

Main exclusion criteria: Previous bone marrow transplant, active uncontrolled infections, severe heart or organ problems, and certain other medical conditions.

Primary goal: To determine whether immediate transplantation or transplantation after azacitidine or chemotherapy provides better outcomes for patients with higher-risk MDS.

Treatment approach: Depending on the number of blast cells in bone marrow, patients will either proceed directly to transplant or receive pre-treatment with azacitidine or chemotherapy. All patients will ultimately undergo stem cell transplantation with busulfan and thiotepa conditioning.

Study of Elritercept for Treating Anemia in Adults with Low to Intermediate Risk Myelodysplastic Syndromes

This trial tests elritercept (KER-050) for treating transfusion-dependent anemia in patients with very low, low, or intermediate-risk MDS. The medication is given as a subcutaneous injection and compared to placebo.

Main inclusion criteria: Patients must be 18 or older with confirmed MDS diagnosis, have less than 5% blasts in bone marrow, and be dependent on red blood cell transfusions. They must have an ECOG performance status of 0-2.

Main exclusion criteria: Other types of cancer, recent heart attack, uncontrolled high blood pressure, severe liver or kidney disease, and pregnancy or breastfeeding.

Primary goal: To evaluate if elritercept can help patients achieve transfusion independence for at least 8 weeks within the first 24 weeks of treatment.

Treatment approach: Participants will receive either elritercept or placebo through subcutaneous injection. Regular monitoring will assess hemoglobin levels, transfusion requirements, and any side effects. The study aims to determine the optimal dose and evaluate long-term safety.

Study of luspatercept in patients with low-risk myelodysplastic syndrome (MDS) who do not require blood transfusions and have not received erythropoiesis treatment

This trial evaluates luspatercept (Reblozyl) in patients with low-risk MDS who have anemia but don’t need blood transfusions and haven’t received erythropoiesis-stimulating treatment before.

Main inclusion criteria: Patients must have confirmed MDS with very low, low, or intermediate risk according to IPSS-R, symptomatic anemia with hemoglobin below 10 g/dL, and be at least 18 years old. They must not be dependent on blood transfusions.

Main exclusion criteria: Prior ESA treatment, high-risk MDS, current need for regular blood transfusions, and various other medical conditions.

Primary goal: To assess if luspatercept can increase hemoglobin levels by at least 1.5 g/dL and maintain this improvement for at least 8 weeks within the first 24 weeks of treatment.

Treatment approach: Patients receive luspatercept as subcutaneous injections. The treatment period may continue for up to 18 months for patients who show improvement. Regular monitoring tracks hemoglobin levels and overall health status.

Study of momelotinib tablets in patients with anemia caused by low-risk myelodysplastic syndrome

This trial tests momelotinib, an oral medication, for treating anemia in patients with lower-risk MDS who require regular blood transfusions. The medication is a JAK inhibitor that may help improve blood cell production.

Main inclusion criteria: Patients must be at least 18 years old with very low, low, or intermediate-risk MDS according to IPSS-R. They must have previously received ESA or luspatercept treatment without response or lost response, and require at least 4 units of red blood cell transfusions over 8 weeks.

Main exclusion criteria: Previous momelotinib treatment, active infections, severe heart conditions, recent cancer history, pregnancy or breastfeeding, and various other medical conditions.

Primary goal: To determine if patients can achieve transfusion independence for at least 12 weeks during the study period.

Treatment approach: Patients take momelotinib tablets by mouth. The study monitors blood cell counts, transfusion requirements, and any side effects. Regular assessments track response to treatment and overall health status.

Study of MP0533 for Patients with Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Using Sodium Dihydrogen Phosphate Dihydrate, Polysorbate 20, and Sodium Chloride

This trial tests MP0533, an investigational medication, for patients with relapsed or refractory AML or MDS. The medication is given as an intravenous infusion.

Main inclusion criteria: Patients must be 18 or older with confirmed diagnosis of relapsed or refractory AML or MDS according to WHO 2016 classification. They must have at least 5% bone marrow blasts and adequate organ function.

Main exclusion criteria: Patients who haven’t recovered from previous treatments, those with severe heart problems, uncontrolled infections, pregnancy or breastfeeding, and inability to follow study procedures.

Primary goal: To assess the safety and tolerability of MP0533 and find the most appropriate dose for treating these blood cancers.

Treatment approach: Patients receive MP0533 through intravenous infusion. The trial monitors for side effects and measures treatment response, including complete response and overall survival rates.

Study on Azacitidine for Patients with Myelodysplastic Syndrome After Stem Cell Transplantation

This trial evaluates azacitidine as a preventive treatment for patients with MDS who show minimal residual disease after stem cell transplant. The goal is to prevent relapse and improve survival.

Main inclusion criteria: Patients must be at least 18 years old with confirmed MDS, MDS/MPN, or AML with myelodysplasia-related features. They must have received a stem cell transplant and be on stable medication doses. Female patients of childbearing potential must have a negative pregnancy test.

Main exclusion criteria: Patients with different blood disorders, those outside the age range, or those unable to follow study procedures.

Primary goal: To reduce the rate of relapse or death at 12 months after detecting minimal residual disease following transplantation.

Treatment approach: Patients receive azacitidine subcutaneously after transplant. Regular monitoring includes checking for minimal residual disease markers and assessing for graft-versus-host disease and other complications.

Study on Ivosidenib and Azacitidine for Adults with Myelodysplastic Syndromes and IDH1 Mutation

This trial tests the combination of ivosidenib (AG-120) and azacitidine for patients with MDS who have an IDH1 mutation. Ivosidenib is taken as tablets while azacitidine is given as injection.

Main inclusion criteria: Patients must be 18 or older with confirmed diagnosis of higher-risk MDS with IDH1 mutation. They must not have received hypomethylating agents before and should have specific blood cell counts and risk scores.

Main exclusion criteria: Patients who have received previous treatment, those with higher blast counts, or those eligible for intensive chemotherapy or stem cell transplant.

Primary goal: To evaluate if the combination treatment can improve remission rates and overall survival compared to azacitidine alone.

Treatment approach: Participants are randomly assigned to receive either the combination of ivosidenib and azacitidine or azacitidine with placebo. Regular monitoring assesses blood counts, side effects, and disease progression over a 48-week period.

Summary

These clinical trials represent a comprehensive effort to improve treatment options for patients with myelodysplastic syndrome across different risk levels and stages of disease. Several notable patterns emerge from this collection of studies.

A significant number of trials focus on lower-risk MDS with anemia, testing various approaches including luspatercept, elritercept, and oral azacitidine. These studies particularly target patients who are transfusion-dependent or show symptoms of anemia, aiming to reduce transfusion needs and improve quality of life.

For higher-risk MDS, trials are exploring combinations of existing and novel therapies, including venetoclax with azacitidine, lisaftoclax combinations, and various targeted therapies for patients with specific genetic mutations (IDH1, IDH2, RARA).

A notable concentration of trials is testing treatments before stem cell transplantation, including studies on conditioning regimens with different drug combinations and radiation protocols. Several trials also focus on post-transplant care, particularly preventing graft-versus-host disease and managing disease relapse.

The trials span multiple European countries, with particularly strong representation in France, Germany, Spain, and Italy. Several studies include multiple countries, indicating international collaboration in MDS research.

Many trials focus on oral medications as alternatives to injectable treatments, potentially offering more convenient administration for patients. The studies also place significant emphasis on quality of life measures alongside traditional clinical endpoints.

Ongoing Clinical Trials on Myelodysplastic syndrome

  • A study testing AZD3632 alone or with other anticancer drugs in adults with acute leukemia or myelodysplastic syndromes with HOX gene overexpression

    Recruiting

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    Denmark Germany Italy

  • Study of RVU120 treatment in patients with myelodysplastic syndrome, solid tumors, or acute myeloid leukemia who benefited from previous RVU120 therapy

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    France Italy Poland Spain

  • Study comparing standard and reduced intensity treatment in patients with acute myeloid leukemia or chronic lymphocytic leukemia who have no detectable disease after initial therapy

    Recruiting

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    Investigated drugs:
    France Germany Poland

  • Study of momelotinib tablets in patients with anemia caused by low-risk myelodysplastic syndrome

    Recruiting

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    France Germany Italy Poland Spain

  • Study of momelotinib in patients with VEXAS syndrome and myelodysplastic syndrome who are dependent on or do not respond to steroid treatment

    Recruiting

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    France

  • Study on Metformin for Preventing Leukemia in Patients with Clonal Cytopenia and Low-Risk Myelodysplastic Syndromes

    Recruiting

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    Denmark

  • Study Comparing Oral Azacitidine and Cedazuridine with Subcutaneous Azacitidine for Patients with Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Acute Myeloid Leukemia

    Recruiting

    1 1 1 1
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    Investigated drugs:
    Czechia France Germany Hungary Italy Poland +1

  • Study on the Safety and Effects of Inobrodib, Betamethasone Sodium Phosphate, and Dimethyl Fumarate in Patients with Advanced Blood and Bone Marrow Cancers

    Recruiting

    1 1 1
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    Spain

  • Study on the Effectiveness of Lisaftoclax and Azacitidine in Adults with Newly Diagnosed Higher Risk Myelodysplastic Syndrome

    Recruiting

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    Belgium Bulgaria Czechia Finland France Germany +7

  • Study on Ivosidenib for Patients with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome with IDH1 Mutation After Stem Cell Transplant

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

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