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Myelodysplastic Syndromes clinical trials

Directory of U.S. ClinicalTrials.gov public records for Myelodysplastic Syndromes. Browse Myelodysplastic Syndromes recruiting trials, filter by phase, sponsor, location, or intervention, and open the official ClinicalTrials.gov study record for each result.

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ClinicalTrials.gov public records Last synced Oct 16, 2026, 11:27 AM EDT

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Official matching trials
1797
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12
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Recently updated
Condition scope
Myelodysplastic Syndromes
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How Myelodysplastic Syndromes studies are organized in the registry

MDS listings rely on risk-stratum vocabulary — “lower-risk” and “higher-risk MDS” describe different study populations and appear in titles. The portfolio overlaps with AML research, and some records list both conditions because higher-risk MDS studies often allow or track progression to AML.

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Showing 13–24 of 1,797 matching trials for Myelodysplastic Syndromes.
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Live official records

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Conditions
Myelodysplastic Syndromes
Interventions
pacritinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 120 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
GATA2, Immunodeficiency, MDS
Interventions
Allogeneic HSCT, Busulfan Test dose, Fludarabine (Fludara, Berlex Laboratories), Busulfan (Busulfex), Cyclophosphamide (CTX, Cytoxan), Total Body Irradiation (TBI), Mycophenolate mofetil (MMF), Tacrolimus, Equine Anti-Thymocyte Globulin
Procedure · Drug · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
6 Years to 70 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Myelodysplastic Syndrome With Excess Blasts, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Recurrent Myelodysplastic Syndrome/Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Refractory Myelodysplastic Syndrome, Refractory Myelodysplastic Syndrome/Acute Myeloid Leukemia
Interventions
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Echocardiography Test, Multigated Acquisition Scan, UAE Inhibitor TAK-243
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
4
States / cities
Tampa, Florida • Chicago, Illinois • Columbus, Ohio + 1 more
Conditions
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Interventions
Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
Device · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
2
States / cities
Baltimore, Maryland • Bethesda, Maryland
Conditions
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia, Myelodysplasia-Related, Myelodysplastic Syndrome
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Azacitidine, Best Practice, Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Busulfan, Chest Radiography, Computed Tomography, Cytarabine, Daunorubicin, Daunorubicin Hydrochloride, Decitabine and Cedazuridine, Echocardiography Test, Emavusertib, Enasidenib, Epoetin Alfa, Fludarabine, Gemtuzumab Ozogamicin, Gilteritinib, Liposome-encapsulated Daunorubicin-Cytarabine, Luspatercept, Melphalan, Multigated Acquisition Scan, Mutation Carrier Screening, Olutasidenib, Placebo Administration, Positron Emission Tomography, Quizartinib, Total-Body Irradiation, Venetoclax
Procedure · Drug · Other + 2 more
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
342
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 262 more
Conditions
Adult Acute Myeloid Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Untreated Adult Acute Myeloid Leukemia
Interventions
5-fluoro-2-deoxycytidine, tetrahydrouridine, laboratory biomarker analysis, reverse transcriptase-polymerase chain reaction, DNA methylation analysis
Drug · Other · Genetic
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2022
U.S. locations
1
States / cities
Duarte, California
Conditions
Immunodeficiency, Hematopoietic Stem Cell Transplantation
Interventions
Allogeneic HSCT, Busulfan test dose, Mycophenolate mofetil (MMF), Tacrolimus, Busulfan, Total Body Irradiation (TBI), Fludarabine, Cyclophosphamide (CY), Post-Transplant Cyclophosphamide (PTCY)
Procedure · Drug · Radiation
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Acute Myeloid Leukaemia, High Risk Myelodysplastic Syndrome
Interventions
AMX-883, Posaconazole
Drug
Lead sponsor
Amphista Therapeutics Ltd
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Fairfax, Virginia
Conditions
Myeloid Neoplasm
Interventions
CPX-351, MHA, Allogeneic Hematopoietic Stem Cell Transplantation
Drug · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
1 Year to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
AML/MDS, Acute Myeloid Leukemia, Myelodysplastic Syndromes
Interventions
Danvatirsen, Danvatirsen + Venetoclax
Drug · Combination Product
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
2
States / cities
The Bronx, New York • Houston, Texas
Conditions
Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes, Bone Marrow Diseases
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 120 Years
Enrollment
1,400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome
Interventions
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Dual-Energy Computed Tomography, Echocardiography, Fludarabine, Magnetic Resonance Imaging, Melphalan, Multigated Acquisition Scan, Partially Engineered T-regulatory Cell Donor Graft TRGFT-201, Tacrolimus, Thiotepa, Total Marrow and Lymphoid Irradiation
Procedure · Drug · Biological + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
60 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
1
States / cities
Duarte, California

About this Myelodysplastic Syndromes clinical trials directory page

What this page lists
U.S. ClinicalTrials.gov public records that list Myelodysplastic Syndromes as a study condition, including recruiting, not yet recruiting, and active not recruiting trials across phase 1, phase 2, and phase 3.
How to narrow results
Use the filters above to scope by intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, age, or NCT ID within Myelodysplastic Syndromes.
Where the data comes from
Records are sourced from the official ClinicalTrials.gov public API. Each result links back to the official study page, which is the source of truth for eligibility, enrollment, and contact information.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, ranking of trials by quality, or AI-generated medical summaries.

Related: all condition pages, browse by drug or therapy, browse by U.S. city, browse by sponsor, or open the full search for Myelodysplastic Syndromes.