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ClinicalTrials.gov 8 recruiting now official registry

Refractory Myelodysplastic Syndrome clinical trials

8 of 14 indexed Refractory Myelodysplastic Syndrome trials are currently recruiting.

14 US clinical trials · 8 currently recruiting

Refractory Myelodysplastic Syndrome is the subject of 14 registered US clinical trials on ClinicalTrials.gov, 8 of them currently open to new participants. 0 are in Phase 3-4 (later-stage) and 14 in Phase 1-2 (earlier-stage). The most active sponsor is National Cancer Institute (NCI), running 5 of these trials.

The research picture

Refractory Myelodysplastic Syndrome has 14 registered US clinical trials, 8 of them open to new participants right now, about 57% of the total.

8
recruiting participants now
57%
of trials open to enrollment
0
in Phase 3–4 (later-stage)
5
top sponsor: National Cancer Institute (NCI)
Phase 112Phase 22

Active & Recent Trials

14 total, page 1 of 1

RECRUITING Phase 1 120 participants

CLAG-M or FLAG-Ida Chemotherapy and Reduced-Intensity Conditioning Donor Stem Cell Transplant for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelomonocytic Leukemia

Fred Hutchinson Cancer Center

NCT04375631

RECRUITING Phase 1 120 participants

ONC201 in Treating Patients With Relapsed or Refractory Acute Leukemia or High-Risk Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT02392572

RECRUITING Phase 2 76 participants

A Phase II, Open-Label, Study of Subcutaneous Canakinumab, an Anti-IL-1β Human Monoclonal Antibody, for Patients With Low or Int-1 Risk IPSS/IPSS-R Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia

M.D. Anderson Cancer Center

NCT04239157

RECRUITING Phase 1 60 participants

Eltanexor and Venetoclax in Relapsed or Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia

Vanderbilt-Ingram Cancer Center

NCT06399640

RECRUITING Phase 1 54 participants

Pembrolizumab and Decitabine With or Without Venetoclax in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome That Is Newly-Diagnosed, Recurrent, or Refractory

City of Hope Medical Center

NCT03969446

RECRUITING Phase 1 54 participants

Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes

National Cancer Institute (NCI)

NCT06484062

RECRUITING Phase 1 52 participants

Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

M.D. Anderson Cancer Center

NCT04128748

RECRUITING Phase 1 42 participants

TAK-243 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts

National Cancer Institute (NCI)

NCT03816319

ACTIVE NOT RECRUITING Phase 1 54 participants

Ipilimumab and Decitabine in Treating Patients With Relapsed or Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

National Cancer Institute (NCI)

NCT02890329

ACTIVE NOT RECRUITING Phase 1 34 participants

Venetoclax and Azacitidine for the Treatment of Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

M.D. Anderson Cancer Center

NCT04550442

ACTIVE NOT RECRUITING Phase 2 14 participants

Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation

National Cancer Institute (NCI)

NCT03953898

ACTIVE NOT RECRUITING Phase 1 8 participants

Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory That Expresses E-selectin Ligand on the Cell Membrane

National Cancer Institute (NCI)

NCT05146739

ACTIVE NOT RECRUITING Phase 1 6 participants

Navitoclax in Relapsed or Refractory High-Risk Myelodysplastic Syndrome

Thomas Jefferson University

NCT05564650

ACTIVE NOT RECRUITING Phase 1 2 participants

Ivosidenib and Combination Chemotherapy for the Treatment of IDH1 Mutant Relapsed or Refractory Acute Myeloid Leukemia

Northwestern University

NCT04250051

Phase Distribution

PhaseTrial count
Phase 1 12
Phase 2 2

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

Reading the Refractory Myelodysplastic Syndrome Trial Landscape

14 indexed Refractory Myelodysplastic Syndrome studies · 8 recruiting (57%).

Phase mix: 0 late (III/IV) vs 14 early (I/II).

Top sponsor: National Cancer Institute (NCI) (5 trials), 6 others listed. 14 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 Refractory Myelodysplastic Syndrome?

PlainTrial tracks 14 US clinical trials for Refractory Myelodysplastic Syndrome, of which 8 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Refractory 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.