Myelodysplastic Syndrome Clinical Trials

290 recruitingLast updated: June 17, 2026

There are 290 actively recruiting myelodysplastic syndrome clinical trials across 48 countries. Studies span Phase 2, Phase 1, Not Applicable, Phase 3, Early Phase 1. Top locations include Houston, Texas, United States, Boston, Massachusetts, United States, Duarte, California, United States. Updated daily from ClinicalTrials.gov.


Myelodysplastic Syndrome Trials at a Glance

290 actively recruiting trials for myelodysplastic syndrome are listed on ClinicalTrialsFinder across 6 cities in 48 countries. The largest study group is Phase 2 with 136 trials, with the heaviest enrollment activity in Houston, Boston, and Duarte. Lead sponsors running myelodysplastic syndrome studies include M.D. Anderson Cancer Center, Fred Hutchinson Cancer Center, and City of Hope Medical Center.

Browse myelodysplastic syndrome trials by phase

Treatments under study

About Myelodysplastic Syndrome Clinical Trials

Looking for clinical trials for Myelodysplastic Syndrome? There are currently 70 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Myelodysplastic Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Myelodysplastic Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 290 trials

Recruiting
Phase 1Phase 2

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms

Myelodysplastic Syndromes
National Cancer Institute (NCI)160 enrolled1 locationNCT06303193
Recruiting

Collection of Blood, Bone Marrow, Skin, Saliva, and Stool Samples From Healthy Volunteers Used for Comparative Analysis of Myeloid Malignancies

Myelodysplastic Syndromes
National Cancer Institute (NCI)1,000 enrolled1 locationNCT05588154
Recruiting
Phase 2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Paroxysmal Nocturnal Hemoglobinuria, PNHSevere Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)
National Heart, Lung, and Blood Institute (NHLBI)56 enrolled1 locationNCT03520647
Recruiting
Phase 1Phase 2

211At-BC8-B10 Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory High-Risk Acute Leukemia or Myelodysplastic Syndrome

Hematopoietic and Lymphoid Cell NeoplasmAcute Myeloid Leukemia in RemissionChronic Myelomonocytic Leukemia+9 more
Fred Hutchinson Cancer Center30 enrolled1 locationNCT03670966
Recruiting
Phase 3

A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Myelodysplastic SyndromeAnemia
Takeda300 enrolled154 locationsNCT07422480
Recruiting
Phase 2

MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)

Acute Myeloid LeukemiaMyelodysplastic SyndromeAcute Myeloid Leukemia Post Cytotoxic Therapy+2 more
National Cancer Institute (NCI)2,000 enrolled347 locationsNCT05564390
Recruiting
Phase 1

Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation

Myelodysplastic SyndromesRelapsed or Refractory Acute Myeloid Leukemia (AML)Untreated AML+1 more
Institut de Recherches Internationales Servier291 enrolled30 locationsNCT02074839
Recruiting
Phase 2

Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)

Myelodysplastic Syndrome
National Cancer Institute (NCI)54 enrolled140 locationsNCT06577441
Recruiting
Phase 2

CPX-351 and Ivosidenib for the Treatment of IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Myelodysplastic SyndromeMyeloproliferative NeoplasmRecurrent Acute Myeloid Leukemia+2 more
M.D. Anderson Cancer Center30 enrolled1 locationNCT04493164
Recruiting
Phase 1

Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes

Acute Myeloid LeukemiaMyelodysplastic SyndromeRecurrent Myelodysplastic Syndrome+6 more
National Cancer Institute (NCI)54 enrolled22 locationsNCT06484062
Recruiting
Phase 1Phase 2

Cladribine, Idarubicin, Cytarabine, and Quizartinib in Treating Patients With Newly Diagnosed, Relapsed, or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Acute Myeloid LeukemiaHigh Risk Myelodysplastic SyndromeRecurrent Acute Myeloid Leukemia+5 more
M.D. Anderson Cancer Center80 enrolled1 locationNCT04047641
Recruiting
Phase 1Phase 2

ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Acute Myeloid LeukemiaMyelodysplastic SyndromeRecurrent Acute Myeloid Leukemia+1 more
M.D. Anderson Cancer Center42 enrolled1 locationNCT05010122
Recruiting
Not Applicable

Haplo-Cord HSCT for AML/MDS

MDS (Myelodysplastic Syndrome)AML (Acute Myeloid Leukemia)
Fujian Medical University Union Hospital82 enrolled4 locationsNCT07588360
Recruiting
Phase 1

A Vaccine (VSV-hIFNβ-NIS) With or Without Cyclophosphamide and Combinations of Ipilimumab, Nivolumab, and Cemiplimab in Treating Relapsed or Refractory Multiple Myeloma, Acute Myeloid Leukemia or Lymphoma

Myelodysplastic SyndromeB-cell Non Hodgkin LymphomaRefractory Acute Myeloid Leukemia+16 more
Mayo Clinic99 enrolled2 locationsNCT03017820
Recruiting
Phase 2

Faecal Microbiota Transplantation After Allogeneic Stem Cell Transplantation

Chronic Lymphocytic LeukemiaHodgkin LymphomaMyeloma+4 more
University Hospital, Clermont-Ferrand150 enrolled20 locationsNCT04935684
Recruiting

Investigation of the Genetics of Hematologic Diseases

Diamond-Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+10 more
St. Jude Children's Research Hospital1,716 enrolled1 locationNCT02720679
Recruiting
Phase 1Phase 2

Phase I/II Study of CAR.70- Engineered IL15-transduced Cord Blood-derived NK Cells in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Hematological Malignances

Hodgkin LymphomaMultiple MyelomaAcute Myeloid Leukemia (AML)+7 more
M.D. Anderson Cancer Center80 enrolled1 locationNCT05092451
Recruiting
Phase 2

A Phase II Study of Cladribine and Low Dose Cytarabine in Combination With Venetoclax, Alternating With Azacitidine and Venetoclax, in Patients With Higher-risk Myeloproliferative Chronic Myelomonocytic Leukemia or Higher-risk Myelodysplastic Syndromes With Excess Blasts

Myelodysplastic SyndromesMyeloproliferative Chronic Myelomonocytic Leukemia
M.D. Anderson Cancer Center60 enrolled1 locationNCT05365035
Recruiting
Phase 1

DR-18 for the Treatment of Relapsed or Persistent Acute Myeloid Leukemia or Myelodysplastic Syndrome After Hematopoietic Cell Transplantation, the DR. DREAM Trial

Acute Myeloid LeukemiaMyelodysplastic SyndromeRecurrent Myelodysplastic Syndrome+1 more
Fred Hutchinson Cancer Center20 enrolled1 locationNCT06492707
Recruiting
Phase 2

Alpha/Beta T Cell and CD19+ B Cell Depletion in Allogeneic Stem Cell Transplantation in Patients With Malignant Diseases

Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaChronic Myeloid Leukemia+4 more
University of Florida20 enrolled1 locationNCT05800210