MDS Clinical Trials

93 recruitingLast updated: June 17, 2026

There are 93 actively recruiting mds clinical trials across 30 countries. Studies span Phase 2, Phase 1, Not Applicable, Phase 3, Early Phase 1. Top locations include New York, New York, United States, Houston, Texas, United States, Boston, Massachusetts, United States. Updated daily from ClinicalTrials.gov.


MDS Trials at a Glance

93 actively recruiting trials for mds are listed on ClinicalTrialsFinder across 6 cities in 30 countries. The largest study group is Phase 2 with 39 trials, with the heaviest enrollment activity in New York, Houston, and Boston. Lead sponsors running mds studies include National Heart, Lung, and Blood Institute (NHLBI), Institute of Hematology & Blood Diseases Hospital, China, and St. Jude Children's Research Hospital.

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 93 trials

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
Not Applicable

Haplo-Cord HSCT for AML/MDS

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

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Hodgkin LymphomaNon-Hodgkin LymphomaHematologic Malignancies+14 more
Center for International Blood and Marrow Transplant Research99,999 enrolled142 locationsNCT01351545
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 enrolled9 locationsNCT07384715
Recruiting
Phase 1Phase 2

Phase 1/ Phase 2 Study to Assess Safety and Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis

Myeloproliferative Neoplasms (MPN)Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN)Essential Thrombocythemia, ET
Jubilant Therapeutics Inc.30 enrolled7 locationsNCT07612280
Recruiting
Phase 2

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

MDSImmunodeficiencyGATA2
National Cancer Institute (NCI)144 enrolled1 locationNCT01861106
Recruiting
Phase 2

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

MDS (Myelodysplastic Syndrome)Severe Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)120 enrolled2 locationsNCT01174108
Recruiting
Phase 3

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-ThalassemiaMyelodysplastic Syndromes (MDS)Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
Celgene665 enrolled143 locationsNCT04064060
Recruiting

Creation of a Biocollection of Patients With Acute Myeloid Leukemia (AML) or Lymphoid Leukemia (ALL) or High-risk Myelodysplastic Syndrome (MDS) Monitored at the Nantes University Hospital

High risk myelodysplastic syndrome (MDS)Acute Lymphoid LeukemiaAcute Myeloid Leucemia
Nantes University Hospital1,000 enrolled1 locationNCT06681363
Recruiting
Phase 2

Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS

MDS (Myelodysplastic Syndrome)AML (Acute Myeloid Leukemia)
Ruijin Hospital20 enrolled1 locationNCT07607418
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
Early Phase 1

5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies

Myelodysplastic SyndromesMDS/MPN Crossover Syndromes
Benjamin Tomlinson20 enrolled1 locationNCT04187703
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
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
Not Applicable

Peloid Therapy in Temporomandibular Joint Dysfunction

PainTemporomandibular Disorders (TMDs)Otalgia
Uşak University40 enrolled1 locationNCT07576582
Recruiting

Familial Investigations of Childhood Cancer Predisposition

Pancreatic CancerHereditary Breast and Ovarian CancerHodgkin Lymphoma+43 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 1Phase 2

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

LeukemiaInborn Errors of MetabolismHLH+11 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT06839456