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ClinicalTrials.gov 46 recruiting now official registry

Myelodysplastic Syndrome clinical trials

46 of 135 indexed Myelodysplastic Syndrome trials are currently recruiting.

135 US clinical trials · 46 currently recruiting

Myelodysplastic Syndrome is the subject of 135 registered US clinical trials on ClinicalTrials.gov, 46 of them currently open to new participants. 7 are in Phase 3-4 (later-stage) and 121 in Phase 1-2 (earlier-stage). The most active sponsor is M.D. Anderson Cancer Center, running 33 of these trials.

The research picture

Myelodysplastic Syndrome has 135 registered US clinical trials, 46 of them open to new participants right now, about 34% of the total.

46
recruiting participants now
34%
of trials open to enrollment
7
in Phase 3–4 (later-stage)
33
top sponsor: M.D. Anderson Cancer Center
Phase 157Phase 264Phase 37

Active & Recent Trials

135 total, page 2 of 3

ACTIVE NOT RECRUITING Phase 2 282 participants

Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia

National Cancer Institute (NCI)

NCT01522976

ACTIVE NOT RECRUITING Phase 3 280 participants

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

Children's Oncology Group

NCT02521493

ACTIVE NOT RECRUITING Phase 3 247 participants

Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

National Cancer Institute (NCI)

NCT00843882

ACTIVE NOT RECRUITING Phase 2 204 participants

Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant

M.D. Anderson Cancer Center

NCT02861417

ACTIVE NOT RECRUITING Phase 3 187 participants

Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

Orca Biosystems

NCT05316701

ACTIVE NOT RECRUITING Phase 1 140 participants

Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms

Incyte Corporation

NCT04279847

ACTIVE NOT RECRUITING Phase 1 135 participants

Vorinostat and Azacitidine in Treating Patients With Myelodysplastic Syndromes or Acute Myeloid Leukemia

National Cancer Institute (NCI)

NCT00392353

ACTIVE NOT RECRUITING Phase 2 132 participants

Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm

National Cancer Institute (NCI)

NCT05600894

ACTIVE NOT RECRUITING Phase 2 116 participants

Venetoclax and Sequential Busulfan, Cladribine, and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT02250937

ACTIVE NOT RECRUITING Phase 2 102 participants

Multi-antigen CMV-MVA Triplex Vaccine in Reducing CMV Complications in Patients Previously Infected With CMV and Undergoing Donor Hematopoietic Cell Transplant

City of Hope Medical Center

NCT02506933

ACTIVE NOT RECRUITING Phase 2 99 participants

Nivolumab and/or Ipilimumab With or Without Azacitidine in Treating Patients With Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT02530463

ACTIVE NOT RECRUITING Phase 2 82 participants

Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation

Memorial Sloan Kettering Cancer Center

NCT01746849

ACTIVE NOT RECRUITING Phase 2 72 participants

Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia

City of Hope Medical Center

NCT01885689

ACTIVE NOT RECRUITING Phase 2 59 participants

Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation

City of Hope Medical Center

NCT04339101

ACTIVE NOT RECRUITING Phase 1 52 participants

Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

M.D. Anderson Cancer Center

NCT04655755

ACTIVE NOT RECRUITING Phase 1 52 participants

AZD6738 for Patients With Progressive MDS or CMML

Massachusetts General Hospital

NCT03770429

ACTIVE NOT RECRUITING Phase 2 50 participants

Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT03672539

ACTIVE NOT RECRUITING Phase 2 50 participants

Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes

National Cancer Institute (NCI)

NCT04802161

ACTIVE NOT RECRUITING Phase 1 44 participants

Administration of Donor Multi TAA-Specific T Cells for AML or MDS (ADSPAM)

Baylor College of Medicine

NCT02494167

ACTIVE NOT RECRUITING Phase 1 40 participants

Azacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

M.D. Anderson Cancer Center

NCT03862157

ACTIVE NOT RECRUITING Phase 1 35 participants

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

Roswell Park Cancer Institute

NCT03192397

ACTIVE NOT RECRUITING Phase 1 34 participants

CMV-MVA Triplex Vaccination of Stem Cell Donors in Preventing CMV Viremia in Participants With Allogeneic Transplant

City of Hope Medical Center

NCT03560752

ACTIVE NOT RECRUITING Phase 2 33 participants

Study of a Geriatric Assessment to Plan a Treatment Approach for Older People With Various Blood Disorders

Memorial Sloan Kettering Cancer Center

NCT04761770

ACTIVE NOT RECRUITING Phase 2 33 participants

Total Marrow and Lymphoid Irradiation as Conditioning Regimen Before Hematopoietic Cell Transplantation in Patients With Myelodysplastic Syndrome or Acute Leukemia

City of Hope Medical Center

NCT04262843

ACTIVE NOT RECRUITING Phase 1 33 participants

Guadecitabine and Atezolizumab in Treating Patients With Advanced Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia That Is Refractory or Relapsed

University of Southern California

NCT02935361

ACTIVE NOT RECRUITING Phase 2 31 participants

Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes

Fred Hutchinson Cancer Center

NCT03399773

ACTIVE NOT RECRUITING NA 30 participants

Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study

Ohio State University Comprehensive Cancer Center

NCT05972577

ACTIVE NOT RECRUITING Phase 1 29 participants

RAD001 in Combination With PKC412 in Patients With Relapsed, Refractory or Poor Prognosis AML or MDS

Richard Stone, MD

NCT00819546

ACTIVE NOT RECRUITING Phase 2 23 participants

Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia

Fred Hutchinson Cancer Center

NCT04994808

ACTIVE NOT RECRUITING Phase 1 20 participants

TBI Using IMRT (VMAT or Tomotherapy) for the Prevention of Pul Toxicities in Patients Undergoing Donor SCT

City of Hope Medical Center

NCT04281199

ACTIVE NOT RECRUITING Phase 2 20 participants

Adding Itacitinib to Cyclophosphamide and Tacrolimus for the Prevention of Graft Versus Host Disease in Patients Undergoing Hematopoietic Stem Cell Transplants

City of Hope Medical Center

NCT05364762

ACTIVE NOT RECRUITING Phase 1 16 participants

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

M.D. Anderson Cancer Center

NCT03622788

ACTIVE NOT RECRUITING Phase 1 15 participants

Administration of Donor T Cells With the Caspase-9 Suicide Gene

Baylor College of Medicine

NCT01494103

ACTIVE NOT RECRUITING Phase 1 10 participants

CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene

Baylor College of Medicine

NCT00710892

ACTIVE NOT RECRUITING Phase 2 7 participants

Triplex Vaccine in Preventing CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplantation

City of Hope Medical Center

NCT04060277

ACTIVE NOT RECRUITING Phase 1 7 participants

90Y-DOTA-anti-CD25 Basiliximab, Fludarabine, Melphalan, and Total Marrow and Lymphoid Irradiation for the Treatment of High-Risk Acute Leukemia or Myelodysplastic Syndrome

City of Hope Medical Center

NCT05139004

ACTIVE NOT RECRUITING Phase 1 6 participants

Navitoclax in Relapsed or Refractory High-Risk Myelodysplastic Syndrome

Thomas Jefferson University

NCT05564650

COMPLETED Phase 3 260 participants

Study of US-ATG-F to Prevent Chronic Graft Versus Host Disease (GVHD)

Neovii Biotech

NCT01295710

COMPLETED Phase 3 233 participants

IV Busulfan With Allo-BMT: Study for Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT00469144

COMPLETED Phase 2 202 participants

Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow

National Heart, Lung, and Blood Institute (NHLBI)

NCT00003838

COMPLETED Phase 2 153 participants

Decitabine (DAC) w/ or w/o Valproic Acid (VPA) in Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

M.D. Anderson Cancer Center

NCT00414310

COMPLETED Phase 2 150 participants

Nivolumab and Azacitidine With or Without Ipilimumab in Treating Patients With Refractory/Relapsed or Newly Diagnosed Acute Myeloid Leukemia

M.D. Anderson Cancer Center

NCT02397720

COMPLETED Early Phase 1 130 participants

Evaluation of Antibody Response to High-Dose Seasonal Influenza Vaccination in Patients With Myeloid Malignancy Receiving Chemotherapy and Healthy Volunteers

Roswell Park Cancer Institute

NCT04484532

COMPLETED Phase 2 128 participants

Study of Three Different Schedules of Low-Dose Decitabine in Myelodysplastic Syndrome (MDS)

M.D. Anderson Cancer Center

NCT00067808

COMPLETED Phase 2 102 participants

The Role of Cyclosporine in Blood Cell Transplants With T-Cell Add-Back for Blood Cancers

National Heart, Lung, and Blood Institute (NHLBI)

NCT00001873

COMPLETED Phase 1 96 participants

A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome

Opsona Therapeutics

NCT02363491

COMPLETED Phase 2 96 participants

Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor

Memorial Sloan Kettering Cancer Center

NCT00582933

COMPLETED Phase 1 92 participants

Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System

National Cancer Institute (NCI)

NCT00520130

COMPLETED Phase 2 84 participants

Cord Blood With T-Cell Depleted Haplo-identical Peripheral Blood Stem Cell Transplantation for Hematological Malignancies

Memorial Sloan Kettering Cancer Center

NCT01682226

COMPLETED Phase 2 73 participants

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Fred Hutchinson Cancer Center

NCT00723099

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Phase Distribution

PhaseTrial count
Phase 1 57
Phase 2 64
Phase 3 7

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Myelodysplastic Syndrome Trial Landscape

135 indexed Myelodysplastic Syndrome studies · 46 recruiting (34%).

Phase mix: 7 late (III/IV) vs 121 early (I/II).

Top sponsor: M.D. Anderson Cancer Center (33 trials), 9 others listed. 50 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Myelodysplastic Syndrome?

PlainTrial tracks 135 US clinical trials for Myelodysplastic Syndrome, of which 46 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Myelodysplastic Syndrome?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.