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ClinicalTrials.gov 4 recruiting now official registry

Myelodysplastic Syndrome (MDS) clinical trials

Every US clinical trial registered for Myelodysplastic Syndrome (MDS) — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.

18 US clinical trials · 4 currently recruiting

The research picture

Myelodysplastic Syndrome (MDS) has 18 registered US clinical trials, 4 of them open to new participants right now — about 22% of the total.

4
recruiting participants now
22%
of trials open to enrollment
2
in Phase 3–4 (later-stage)
3
top sponsor: St. Jude Children's Research Hospital

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

RECRUITING Phase 2 70 participants

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Children's Hospital Medical Center, Cincinnati

NCT02143830

RECRUITING Phase 1 60 participants

CLN-049 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

Cullinan Therapeutics

NCT05143996

RECRUITING Phase 1 37 participants

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

National Heart, Lung, and Blood Institute (NHLBI)

NCT03173937

RECRUITING Phase 1 36 participants

Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics

medac

NCT05534620

ACTIVE NOT RECRUITING Phase 3 531 participants

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

AbbVie

NCT04401748

ACTIVE NOT RECRUITING Phase 1 260 participants

Study of Oral LY3410738 in Patients With Advanced Hematologic Malignancies With IDH1 or IDH2 Mutations

Eli Lilly and Company

NCT04603001

ACTIVE NOT RECRUITING Phase 2 170 participants

Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation

St. Jude Children's Research Hospital

NCT02790515

ACTIVE NOT RECRUITING Phase 1 102 participants

Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN

Jacqueline Garcia, MD

NCT03613532

ACTIVE NOT RECRUITING Phase 1 14 participants

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Diane George

NCT02061800

COMPLETED Phase 2 80 participants

HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

Center for International Blood and Marrow Transplant Research

NCT02793544

COMPLETED Phase 3 57 participants

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

St. Jude Children's Research Hospital

NCT00186823

COMPLETED Phase 1 43 participants

Improving Blood Stem Cell Collection and Transplant Procedures

National Heart, Lung, and Blood Institute (NHLBI)

NCT01517035

COMPLETED Phase 2 40 participants

Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT

Center for International Blood and Marrow Transplant Research

NCT01772953

COMPLETED Phase 1 36 participants

Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM

Gamida Cell

NCT01816230

COMPLETED Phase 1 19 participants

Selinexor With Fludarabine and Cytarabine for Treatment of Refractory or Relapsed Leukemia or Myelodysplastic Syndrome

St. Jude Children's Research Hospital

NCT02212561

COMPLETED Phase 2 10 participants

Peptide Vaccinations to Treat Patients With Low-Risk Myeloid Cancers

National Heart, Lung, and Blood Institute (NHLBI)

NCT00488592

COMPLETED NA 10 participants

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders

Massachusetts General Hospital

NCT01758042

COMPLETED Phase 1 3 participants

Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies

Joanne Kurtzberg, MD

NCT00673114

Phase Distribution

PhaseTrial count
Phase 1 10
Phase 2 5
Phase 3 2

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

Reading the Myelodysplastic Syndrome (MDS) Trial Landscape

ClinicalTrials.gov lists 18 US studies indexed under Myelodysplastic Syndrome (MDS), and 4 of those are currently open to recruitment — roughly 22% 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 Syndrome (MDS) shows 2 late-stage studies (Phase 3 and Phase 4 combined) alongside 15 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 Syndrome (MDS) is led by St. Jude Children's Research Hospital with 3 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 18 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 Syndrome (MDS)?

PlainTrial tracks 18 US clinical trials for Myelodysplastic Syndrome (MDS), of which 4 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Myelodysplastic Syndrome (MDS)?

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.

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.

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