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Home › Conditions › Myelodysplastic Syndromes

Myelodysplastic Syndromes

5 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Myelodysplastic Syndromes

272 trials tracked for Myelodysplastic Syndromes: 32 in phase 3 or 4 and 10 with published results. The most-cited published study has 533 citations.

272Trials tracked
32Phase 3 & 4
0Recruiting
10With published results
Phase distribution
Phase 4 5 Phase 3 27 Phase 2 200 Phase 1 21 Other / NA 19
  1. Phase 3 A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes Completed · 533 cited
  2. Phase 3 Baclofen-Amitriptyline Hydrochloride-Ketamine Gel in Treating Peripheral Neuropathy Caused by Chemotherapy in Patients With Cancer Completed · 251 cited
  3. Phase 3 Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder Completed · 153 cited
  4. Phase 3 Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML) Completed · 120 cited
  5. Phase 3 Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant Completed · 111 cited
  6. Phase 3 A Study of Galunisertib in Participants With Myelodysplastic Syndromes Completed · 86 cited
Show 44 more trials
  1. Phase 3 Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML Completed · 82 cited
  2. Phase 3 The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS) Completed · 77 cited
  3. Phase 3 Study of US-ATG-F to Prevent Chronic Graft Versus Host Disease (GVHD) Completed · 24 cited
  4. Phase 3 Tac, Mini-MTX, MMF Versus Tac, MTX for GVHD Prevention Completed · 10 cited
  5. Phase 4 Posaconazole Versus Micafungin for Prophylaxis Against Invasive Fungal Infections During Neutropenia in Patients Undergoing Chemotherapy for Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia or Myelodysplastic Syndrome Completed
  6. Phase 4 A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS) Completed
  7. Phase 4 Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients Completed
  8. Phase 4 Study of Azacitidine in Adult Taiwanese Subjects With Higher-Risk Myelodysplastic Syndromes (MDS) Completed
  9. Phase 4 Busulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT) Completed
  10. Phase 3 IV Busulfan With Allo-BMT: Study for Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome Completed
  11. Phase 3 Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer Completed
  12. Phase 3 Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs Completed
  13. Phase 3 Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML Completed
  14. Phase 3 Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Completed
  15. Phase 3 Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia Completed
  16. Phase 3 Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain Completed
  17. Phase 3 Single vs Double Umbilical Cord Blood Transplants in Children With High Risk Leukemia and Myelodysplasia (BMT CTN 0501) Completed
  18. Phase 3 Methotrexate or Pentostatin for Graft-versus-host Disease Prophylaxis in Risk-adapted Allogeneic Bone Marrow Transplantation for Hematologic Malignancies Completed
  19. Phase 3 An Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic Syndrome Completed
  20. Phase 3 American Ginseng in Treating Patients With Fatigue Caused by Cancer Completed
  21. Phase 3 A Randomized Trial of Procrit vs. No Procrit in AML and High Risk MDS Completed
  22. Phase 3 Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality Completed
  23. Phase 3 Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402) Completed
  24. Phase 3 A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care Completed
  25. Phase 3 An Extension to Study AZA PH GL 2003 CL 001 Allowing for Continuation of Azacitidine Treatment in Patients With Myelodysplastic Syndromes (MDS) Completed
  26. Phase 3 Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes Completed
  27. Phase 2 Pilot Study of Leuprolide to Improve Immune Function After Allogeneic Bone Marrow Transplantation Completed
  28. Phase 2 Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer Completed
  29. Phase 2 T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts Completed
  30. Phase 2 Salsalate, Venetoclax, and Decitabine or Azacitidine for the Treatment of Acute Myeloid Leukemia or Advanced Myelodysplasia/Myeloproliferative Disease Completed
  31. Phase 2 NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders Completed
  32. Phase 2 Allo BMT Using Matched Related/Unrelated Donors With FluBu and HiCY Completed
  33. Phase 2 Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen Completed
  34. Phase 2 CD8 Depleted, Non-engrafting, HLA Mismatched Unrelated Infusion With MDS and Secondary AML Completed
  35. Phase 2 Off-the-shelf NK Cells + SCT for Myeloid Malignancies Completed
  36. Phase 2 Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies Completed
  37. Phase 2 Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer Completed
  38. Phase 2 Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS) Completed
  39. Phase 2 Study to Separately Evaluate the Activity of Talacotuzumab (JNJ-56022473) or Daratumumab in Transfusion-Dependent Participants With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) Who Are Relapsed or Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment Completed
  40. Phase 2 2018-0674 - IL-7 for T-Cell Recovery Post Haplo and CB Transplant - Phase I/II Completed
  41. Phase 2 Multi-Ctr PII Cmb.Modality Tx Ruxolitinib, Decitabine, and DLI for Post HSCT in AML/MDS Completed
  42. Phase 2 Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders Completed
  43. Phase 2 Decitabine and Gemtuzumab Ozogamicin in Acute Myelogenous Leukemia and High-risk Myelodysplastic Syndrome Completed
  44. Phase 2 Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies Completed

Showing the 50 most-cited and recently-updated of 272 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
FDA Approves Revlimid (lenalidomide) for Multiple Myeloma and Other Indications
Oncology FDA Approval
FDA Approves Revlimid (lenalidomide) for Multiple Myeloma and Other Indications FDA approves Revlimid for several blood cancers and related conditions.
FDA approved Revlimid (lenalidomide) for multiple myeloma (in combination with dexamethasone and as maintenance after auto-HSCT), transfusio…
The FDA approved Revlimid (lenalidomide) for multiple myeloma, certain myelodysplastic syndromes, mantle cell lymphoma, and other lymphomas.…
FDA May 2, 2026
Donor-derived anti-CD33 CAR T-cell therapy showed 20% overall response in 15 adults with relapsed AML/MDS
Oncology Phase I
Donor-derived anti-CD33 CAR T-cell therapy showed 20% overall response in 15 adults with relapsed AML/MDS Donor-Derived Cells Show Promise Against Leukemia Relapse
This Phase 1/2 study enrolled 15 adults with relapsed or MRD-positive CD33+ AML/MDS after allogeneic hematopoietic cell transplantation.
A new treatment using donor immune cells shows early signs of fighting leukemia after transplant.
Blood Apr 24, 2026
Meta-analysis identifies germline susceptibility loci for rare cancers including MDS and GIST
Genetics & Precision Medicine Cohort
Meta-analysis identifies germline susceptibility loci for rare cancers including MDS and GIST Genes linked to rare cancers found in large study of over 480,000 people
This meta-analysis of observational cohorts from over 480,000 individuals identified novel germline susceptibility loci for rare cancers.
A huge study of over 480,000 people found new genes that raise the risk of several rare cancers, offering clues for families and doctors.
medRxiv Apr 22, 2026
FDA Approves Rytelo (telomerase inhibitor oligonucleotide) for transfusion-dependent anemia in low- to intermediate-1 risk MDS
Hematology FDA Approval
FDA Approves Rytelo (telomerase inhibitor oligonucleotide) for transfusion-dependent anemia in low- to intermediate-1 risk MDS FDA approves new drug called Rytelo for certain blood disorder patients who need frequent transfusions
Rytelo is approved for adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have transfusion-dependent anemia…
FDA approves Rytelo to help adults with low-risk blood disorders who need frequent transfusions after other treatments fail.
FDA Apr 17, 2026
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation
Oncology Phase II
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation Early trial explores itacitinib for stem cell transplant patients with blood disorders
This Phase IIa trial evaluated itacitinib combined with tacrolimus and sirolimus in patients with acute leukemia, myelodysplastic syndrome, …
A new trial adds itacitinib to standard care for stem cell transplant patients with blood disorders to help prevent graft-versus-host diseas…
CT.gov Apr 13, 2026

Questions about Myelodysplastic Syndromes

Are there new drugs approved specifically for Myelodysplastic Syndromes anemia?

Yes, newer drugs like luspatercept, imetelstat, and lenalidomide are approved for anemia in lower-risk MDS, with more in development.

Full answer →
Can Revlimid help with anemia in low-risk Myelodysplastic Syndromes?

Yes, Revlimid (lenalidomide) is FDA-approved for transfusion-dependent anemia in low- or intermediate-1-risk MDS with a deletion 5q abnormality.

Full answer →
Is Rytelo approved for patients with transfusion-dependent anemia in Myelodysplastic Syndromes?

Yes, Rytelo (imetelstat) is FDA-approved for adults with low- to intermediate-1 risk MDS and transfusion-dependent anemia who need ≥4 RBC units over 8 weeks and have not responded to or are ineligible for ESAs.

Full answer →
What genetic factors might increase Myelodysplastic Syndromes risk?

Genetic factors that may increase MDS risk include inherited variants near the API5 gene (OR=2.21) and acquired mutations in genes like TET2, ASXL1, DNMT3A, and SF3B1.

Full answer →
Does the FDA approve Rytelo for low-risk Myelodysplastic Syndromes?

Yes, the FDA approved Rytelo (imetelstat) in June 2024 for adults with low- to intermediate-1 risk MDS who have transfusion-dependent anemia and have not responded to or are ineligible for ESAs.

Full answer →
Can Revlimid be used to treat anemia in Myelodysplastic Syndromes?

Yes, Revlimid (lenalidomide) is FDA-approved for transfusion-dependent anemia in low- or intermediate-1-risk MDS with a deletion 5q abnormality.

Full answer →
See all 6 questions about Myelodysplastic Syndromes →