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Myelodysplastic Syndromes clinical trials
Every US clinical trial registered for Myelodysplastic Syndromes — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
193 US clinical trials · 73 currently recruiting
The research picture
Myelodysplastic Syndromes has 193 registered US clinical trials, 73 of them open to new participants right now — about 38% of the total.
- 73
- recruiting participants now
- 38%
- of trials open to enrollment
- 11
- in Phase 3–4 (later-stage)
- 13
- top sponsor: Washington University School of Medicine
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
UNC Lineberger Comprehensive Cancer Center
NCT01137643
Study of MGUS, Smoldering Myeloma, Early MDS and CLL to Assess Molecular Events of Progression and Clinical Outcome
Dana-Farber Cancer Institute
NCT02269592
Registry of Older Patients With Cancer
UNC Lineberger Comprehensive Cancer Center
NCT01137825
The Prospective Collection, Storage and Reporting of Data on Patients Undergoing Hematopoietic Stem Cell Transplantation Utilizing a Standard Preparative Regimen
Wake Forest University Health Sciences
NCT01890486
Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS
National Cancer Institute (NCI)
NCT05350748
Collection of Blood, Bone Marrow, Skin, Saliva, and Stool Samples From Healthy Volunteers Used for Comparative Analysis of Myeloid Malignancies
National Cancer Institute (NCI)
NCT05588154
HEME Home Transfusion Program
Dana-Farber Cancer Institute
NCT06487247
Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants
National Cancer Institute (NCI)
NCT07019155
Leukemia and Lymphoma Society (LLS) Services Impact on Outcomes and Care
University of Miami
NCT05745285
ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
Bristol-Myers Squibb
NCT05949684
Mosaic Trial for Stem Cell Transplant Recipients
Northwestern University
NCT06960993
Whole Genome Sequencing (ChromoSeq) as an Adjunct to Conventional Genomic Profiling in AML and MDS
Washington University School of Medicine
NCT04986657
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
Taiho Oncology
NCT04256317
A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
Orca Biosystems
NCT03802695
Protocol For Genomically Profiling, Collecting, Archiving And Distributing Blood And Bone Marrow Specimens From Children And Young Adults With Hematologic Malignancy
Dana-Farber Cancer Institute
NCT04968834
Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
Institut de Recherches Internationales Servier
NCT02074839
A Study of BGB-11417 in Participants With Myeloid Malignancies
BeiGene
NCT04771130
A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes
Chordia Therapeutics
NCT05732103
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions
Takeda
NCT06499285
Pre-emptive Therapy With DEC-C to Improve Outcomes in MDS Patients With Measurable Residual Disease Post Allogeneic Hematopoietic Cell Transplant
Washington University School of Medicine
NCT04742634
A Telehealth Advance Care Planning Intervention
University of Rochester
NCT05875805
A Study of BH-30236 in Relapsed/ Refractory Acute Myelogenous Leukemia and Higher Risk Myelodysplastic Syndrome
BlossomHill Therapeutics
NCT06501196
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
Takeda
NCT04419649
Study of SX-682 Alone and in Combination With Oral or Intravenous Decitabine in Subjects With Myelodysplastic Syndrome
Syntrix Biosystems
NCT04245397
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
Disc Medicine
NCT05320198
A Study of MGD024 in Patients With Relapsed or Refractory Hematologic Malignancies
MacroGenics
NCT05362773
Study of REM-422 in Patients With AML or Higher Risk MDS
Remix Therapeutics
NCT06297941
Venetoclax Plus Intensive Chemotherapy in AML and Advanced MDS
Montefiore Medical Center
NCT05342584
Metabolically Optimized, Non-cytotoxic Low Dose Weekly Decitabine/Venetoclax in MDS and AML
Montefiore Medical Center
NCT05184842
A Study of Momelotinib in Participants With Low-risk Myelodysplastic Syndrome
GlaxoSmithKline
NCT06847867
Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
Orca Biosystems
NCT07216443
Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
Children's Hospital of Philadelphia
NCT05194397
A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation
TScan Therapeutics
NCT05473910
Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms
H. Lee Moffitt Cancer Center and Research Institute
NCT05732961
Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft
Stanford University
NCT05088356
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
M.D. Anderson Cancer Center
NCT05365035
Whole Genome Sequencing (ChromoSeq) as an Adjunct to Conventional Genomic Profiling in MDS
Washington University School of Medicine
NCT05434598
Allo HSCT Using RIC and PTCy for Hematological Diseases
Masonic Cancer Center, University of Minnesota
NCT05805605
UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy
University of Alabama at Birmingham
NCT06047886
A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation
Dana-Farber Cancer Institute
NCT04024761
CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT
Children's Hospital of Philadelphia
NCT03810196
A Study of GLB-001 in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher Risk Myelodysplastic Syndromes
GluBio Therapeutics
NCT06146257
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Masonic Cancer Center, University of Minnesota
NCT03579875
A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
Lixte Biotechnology Holdings
NCT03886662
HM2023-05: GTB-3650 Trike for High Risk MDS and R/R AML
Masonic Cancer Center, University of Minnesota
NCT06594445
Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation
Sawa Ito, MD
NCT06529731
A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
M.D. Anderson Cancer Center
NCT06113302
Ascorbate in Myelodysplastic Syndrome
Prajwal Dhakal
NCT07283900
Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML
Montefiore Medical Center
NCT05986240
Intensity Modulated Total Marrow Irradiation in Fully Human Leukocyte Antigen (HLA)-Matched and Partially-HLA Mismatched Allogeneic Transplantation Patients With High-Risk Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), and Myelodysplastic Syndrome (MDS)
University of Illinois at Chicago
NCT06802315
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
Franziska Wachter
NCT05796570
Prevention of GvHD in Participants With Hematological Malignancies Undergoing Hematopoietic Stem Cell Transplant (HSCT)
Tr1X
NCT06462365
Co-Transplant of an Unmodified Haplo-Identical Graft With Cord Blood
Case Comprehensive Cancer Center
NCT06904482
IS-free Treg HaploHCT
Dana-Farber Cancer Institute
NCT04678401
Umbilical Cord Blood Transplantation From Unrelated Donors
University of Rochester
NCT03016806
Home Blood Transfusions
Thomas Jefferson University
NCT07121140
Trial of 2 Step ATG for Acute GVHD Prevention Post Myeloablative Allogeneic Stem Cell Transplant
University of Alabama at Birmingham
NCT06315309
Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Hepatic Impairment
Taiho Oncology
NCT04953910
Leflunomide in Combination With Decitabine for Treatment of Relapsed or Refractory Myelodysplastic Syndromes
West Virginia University
NCT06923488
Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
Abhay Singh, MD MPH
NCT06630221
Hyperbaric Oxygen Therapy and Allogeneic Peripheral Blood Stem Cell (PBSC) Transplant
Omar Aljitawi
NCT03964506
Phase I Trial for Patients w/ Advanced Hematologic Malignancies Undergoing Allogeneic HCT
Stanford University
NCT06551584
Phase Ib Study of CD33 FPBMC in Patients With MRD+ AML or MDS
University of Virginia
NCT07270978
Trial of Novel Anti-leukemia Agents in Flu/Mel RIC Transplant for Myeloid Malignancies
University of Alabama at Birmingham
NCT07044544
ABBA CORD: dCBT w/ Abatacept for aGVHD Prophylaxis
Leland Metheny
NCT06680661
5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies
Benjamin Tomlinson
NCT04187703
Alpha/Beta T Cell and CD19+ B Cell Depletion in Allogeneic Stem Cell Transplantation in Patients With Malignant Diseases
University of Florida
NCT05800210
Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment
Taiho Oncology
NCT04953897
Statins in Patients With Clonal Cytopenia of Undetermined Significance (CCUS) and Myelodysplastic Syndromes (MDS)
Washington University School of Medicine
NCT05483010
A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.
M.D. Anderson Cancer Center
NCT06566742
Preemptive CIML NK Cell Therapy After Hematopoietic Stem Cell Transplantation
Dana-Farber Cancer Institute
NCT06138587
Iadademstat With Hypomethylating Agent in Patients With Myelodysplastic Syndrome
Medical College of Wisconsin
NCT06502145
3'-Deoxy-3'-[18F] Fluorothymidine PET Imaging in Patients With Cancer
Barbara Ann Karmanos Cancer Institute
NCT00935090
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
Celgene
NCT03682536
A Collaborative Palliative and Leukemia Care Model for Patients With AML and MDS Receiving Non-Intensive Therapy
Massachusetts General Hospital
NCT03310918
HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
Center for International Blood and Marrow Transplant Research
NCT06001385
HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide
Center for International Blood and Marrow Transplant Research
NCT04904588
Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Geron Corporation
NCT02598661
Precision-T: A Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
Orca Biosystems
NCT04013685
A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)
Bristol-Myers Squibb
NCT05469737
A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia
St. Jude Children's Research Hospital
NCT03164057
A Study to Assess Safety, Tolerability and Preliminary Efficacy of Bexmarilimab in Combination With Standard of Care in Patients With Hematological Malignancies
Faron Pharmaceuticals
NCT05428969
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
Bellicum Pharmaceuticals
NCT03301168
Epigenetics, Vitamin C, and Abnormal Blood Cell Formation - Vitamin C in Patients With Low-Risk Myeloid Malignancies
Rigshospitalet, Denmark
NCT03682029
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
Bristol-Myers Squibb
NCT06045689
Palliative Care Oncology in Patients With Relapsed, Refractory, and High-Risk Leukemias or MDS
University of Virginia
NCT04482894
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Massachusetts General Hospital
NCT04902833
PRGN-3006 Adoptive Cellular Therapy for CD33-Positive Relapsed or Refractory AML, MRD Positive AML or Higher Risk MDS
Precigen
NCT03927261
A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Agios Pharmaceuticals
NCT05490446
AML/MDS Drug Sensitization by in Vivo Chemotherapy Administration
Washington University School of Medicine
NCT04263181
Helical Tomotherapy, Fludarabine Phosphate, and Melphalan Followed By Allo-HSCT in Hematological Malignancies
City of Hope Medical Center
NCT00544466
A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML
Treadwell Therapeutics
NCT04730258
Electronic Health Mindfulness-based Music Therapy Intervention for Patients Undergoing Allogeneic Stem Cell Transplantation
University of Miami
NCT05968963
A Pilot Trial of Atorvastatin in Tumor Protein 53 (p53) -Mutant and p53 Wild-Type Malignancies
Joaquina Baranda
NCT03560882
Vorinostat for Graft vs Host Disease Prevention in Children, Adolescents and Young Adults Undergoing Allogeneic Blood and Marrow Transplantation
University of Michigan Rogel Cancer Center
NCT03842696
A Phase 1, Open-Label, Sequential Cross-over, Bioavailability/Bioequivalence Study to Compare the Pharmacokinetics of Oral Cladribine With the Reference Listed Drug, Intravenous Cladribine
M.D. Anderson Cancer Center
NCT06021600
Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes
University of Illinois at Chicago
NCT03121014
Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant
Bellicum Pharmaceuticals
NCT01744223
Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab.
Novartis Pharmaceuticals
NCT05201066
Itacitinib With High-dose Posttransplantation Cyclophosphamide in Older Patients
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
NCT05823571
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 80 |
| Phase 2 | 68 |
| Phase 3 | 9 |
| Phase 4 | 2 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Myelodysplastic Syndromes Trial Landscape
ClinicalTrials.gov lists 193 US studies indexed under Myelodysplastic Syndromes, and 73 of those are currently open to recruitment — roughly 38% of the total volume on the registry. 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.
The phase distribution for Myelodysplastic Syndromes shows 11 late-stage studies (Phase 3 and Phase 4 combined) alongside 148 earlier-phase entries (Phase 1 through Phase 2). 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.
Top sponsor activity for Myelodysplastic Syndromes is led by Washington University School of Medicine with 13 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 100 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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 Syndromes?
PlainTrial tracks 193 US clinical trials for Myelodysplastic Syndromes, of which 73 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Myelodysplastic Syndromes?
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.
Related
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.
Read our methodology — how this data is sourced, computed, and verified.
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.