MDS Clinical Trials

32 recruiting

MDS Trials at a Glance

79 actively recruiting trials for mds are listed on ClinicalTrialsFinder across 6 cities in 29 countries. The largest study group is Phase 2 with 32 trials, with the heaviest enrollment activity in New York, Boston, and Houston. Lead sponsors running mds studies include National Heart, Lung, and Blood Institute (NHLBI), St. Jude Children's Research Hospital, and Institute of Hematology & Blood Diseases Hospital, China.

Treatments under study

About MDS Clinical Trials

Looking for clinical trials for MDS? There are currently 32 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 MDS 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 MDS 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 79 trials

Recruiting
Phase 2

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

MDSImmunodeficiencyGATA2
National Cancer Institute (NCI)144 enrolled1 locationNCT01861106
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

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

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

Severe Aplastic AnemiaSingle Lineage Cytopenias, T-LGLHypoplastic MDS
National Heart, Lung, and Blood Institute (NHLBI)145 enrolled1 locationNCT05998408
Recruiting

Familial Investigations of Childhood Cancer Predisposition

Hereditary Breast and Ovarian CancerHodgkin LymphomaAdrenocortical Carcinoma+30 more
St. Jude Children's Research Hospital1,500 enrolled1 locationNCT03050268
Recruiting
Phase 1Phase 2

Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)

MDS/MPN
Michael Savona94 enrolled4 locationsNCT04061421
Recruiting
Phase 2

MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases

Acute Myeloid LeukemiaAcute LeukemiaAML+2 more
Masonic Cancer Center, University of Minnesota132 enrolled1 locationNCT07493538
Recruiting
Phase 2

Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA

MDS (Myelodysplastic Syndrome)Aplastic Anemia (AA)
The First Affiliated Hospital of Soochow University66 enrolled1 locationNCT07400341
Recruiting
Not Applicable

A Single-arm, Prospective Study of a Clad-LABU Conditioning Regimen in HSCT for R/R MDS/AML in Elderly Patients

Relapsed or Refractory Acute Myeloid Leukemia (AML)MDS (Myelodysplastic Syndrome)
The First Affiliated Hospital of Soochow University30 enrolled1 locationNCT07500753
Recruiting
Phase 1Phase 2

Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML/MDS

Proton TherapyAcute Myeloid Leukemia (AML)Myelodysplastic Neoplasm+2 more
Institute of Hematology and Blood Transfusion, Czech Republic16 enrolled2 locationsNCT07532824
Recruiting
Phase 1

ICP-248 in Combination With Azacitidine for the Treatment in Patients With Myeloid Malignancies

Acute Myelogenous LeukemiaMyelodysplastic Syndromes (MDS)
Beijing InnoCare Pharma Tech Co., Ltd.266 enrolled18 locationsNCT06656494
Recruiting
Phase 1

First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)

Acute Myeloid LeukemiaR/R AMLR/R HR-MDS+1 more
Genmab78 enrolled5 locationsNCT07384715
Recruiting
Phase 1Phase 2

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS)Severe Aplastic AnemiaHypo-Plastic MDS
National Heart, Lung, and Blood Institute (NHLBI)37 enrolled1 locationNCT03173937
Recruiting
Phase 3

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

Myelodysplastic Syndromes (MDS)Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS)
Institut de Recherches Internationales Servier48 enrolled62 locationsNCT06465953
Recruiting
Phase 2

BMT-06: Study of Intensity Modulated Total Marrow Irradiation (IM-TMI)

Acute LeukemiaMDS
University of Illinois at Chicago27 enrolled1 locationNCT04187105
Recruiting

WearAble Technology for Collecting Health Data in People Who Are the Transfused (WATCH Transfused) - A UK Exploratory Study to Improve Quality of Life and the Efficacy of Transfusion Supportive Care in People With Blood Cancers Undergoing Treatment

MDS (Myelodysplastic Syndrome)AML (Acute Myeloid Leukemia)
University of Oxford80 enrolled1 locationNCT07511829
Recruiting
Phase 1

CD123-Directed T-Cell Therapy for Acute Myelogenous Leukemia (CATCHAML)

B-ALLAML/MDST-ALL+1 more
St. Jude Children's Research Hospital108 enrolled2 locationsNCT04318678
Recruiting

Replacing Bone Marrow Diagnostics With Peripheral Blood Analysis in Cytopenia Patients

MDSCytopenia
Weizmann Institute of Science1,500 enrolled2 locationsNCT07081087
Recruiting
Phase 2

Luspatercept + Darbepoetin in MDS

MDS (Myelodysplastic Syndrome)
Yale University60 enrolled3 locationsNCT07096297
Recruiting
Phase 2

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Acute Myeloid LeukemiaMDSMyelodysplastic Syndromes+3 more
Franziska Wachter37 enrolled2 locationsNCT05796570
Recruiting
Phase 2

A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation

LymphomaMyelofibrosisAML (Acute Myelogenous Leukemia)+8 more
Center for International Blood and Marrow Transplant Research358 enrolled13 locationsNCT06859424