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Condition (MeSH)

Myelodysplastic Syndromes

ClinicalTrials.gov lists 2,297 trials for Myelodysplastic Syndromes or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 71.

MeSH D009190 Hemic and Lymphatic Diseases C15
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Phase 1 Phase 1 518 Phase 1/2 Phase 1/2 293 Phase 2 Phase 2 817 Phase 2/3 Phase 2/3 30 Phase 3 Phase 3 175 Phase 4 Phase 4 30 Not applicable Not applicable 145 Observational Observational 274 Phase 1 Phase 1 518 Phase 1/2 Phase 1/2 293 Phase 2 Phase 2 817 Phase 2/3 Phase 2/3 30 Phase 3 Phase 3 175 Phase 4 Phase 4 30 Not applicable Not applicable 145 Observational Observational 274

Trials per phase (log scale)

2 more sections with rows

1,964 of the 2,297 trials are tagged with Myelodysplastic Syndromes itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Myelodysplastic Syndromes trials start? (36)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 5 trials are registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational
  • Other (Expanded access, Unknown)

Top of scale: 105

1991: 1 trials (Observational 1) 1992: 3 trials (Not applicable 1, Phase 2 2) 1993: 4 trials (Phase 3 2, Phase 2 1, Phase 1 1) 1994: 1 trials (Phase 2 1) 1995: 5 trials (Observational 1, Phase 3 1, Phase 2/3 1, Phase 2 1, Phase 1/2 1) 1996: 15 trials (Observational 3, Phase 3 3, Phase 2 6, Phase 1/2 1, Phase 1 2) 1997: 18 trials (Not applicable 2, Phase 3 2, Phase 2 9, Phase 1/2 1, Phase 1 4) 1998: 38 trials (Observational 1, Phase 4 2, Phase 3 6, Phase 2/3 1, Phase 2 18, Phase 1/2 2, Phase 1 8) 1999: 35 trials (Observational 2, Not applicable 2, Phase 3 2, Phase 2 13, Phase 1/2 2, Phase 1 14) 2000: 37 trials (Not applicable 3, Phase 3 4, Phase 2/3 1, Phase 2 15, Phase 1/2 4, Phase 1 10) 2001: 40 trials (Observational 4, Not applicable 4, Phase 3 2, Phase 2/3 1, Phase 2 22, Phase 1/2 4, Phase 1 3) 2002: 52 trials (Observational 2, Not applicable 4, Phase 4 2, Phase 3 7, Phase 2/3 1, Phase 2 17, Phase 1/2 9, Phase 1 10) 2003: 44 trials (Observational 2, Not applicable 3, Phase 3 1, Phase 2 28, Phase 1/2 6, Phase 1 4) 2004: 58 trials (Observational 6, Not applicable 5, Phase 3 7, Phase 2/3 1, Phase 2 25, Phase 1/2 6, Phase 1 8) 2005: 81 trials (Observational 1, Not applicable 6, Phase 4 2, Phase 3 8, Phase 2/3 1, Phase 2 38, Phase 1/2 6, Phase 1 19) 2006: 77 trials (Observational 8, Not applicable 2, Phase 3 5, Phase 2/3 1, Phase 2 36, Phase 1/2 14, Phase 1 11) 2007: 103 trials (Other 1, Observational 17, Not applicable 5, Phase 4 4, Phase 3 5, Phase 2/3 1, Phase 2 37, Phase 1/2 10, Phase 1 23) 2008: 96 trials (Other 1, Observational 13, Not applicable 10, Phase 3 8, Phase 2 35, Phase 1/2 10, Phase 1 19) 2009: 88 trials (Other 1, Observational 10, Not applicable 4, Phase 4 4, Phase 3 3, Phase 2/3 1, Phase 2 32, Phase 1/2 11, Phase 1 22) 2010: 84 trials (Observational 15, Not applicable 2, Phase 4 2, Phase 3 8, Phase 2 33, Phase 1/2 9, Phase 1 15) 2011: 71 trials (Observational 9, Phase 4 2, Phase 3 5, Phase 2/3 1, Phase 2 29, Phase 1/2 9, Phase 1 16) 2012: 71 trials (Observational 7, Not applicable 4, Phase 3 2, Phase 2 35, Phase 1/2 8, Phase 1 15) 2013: 93 trials (Observational 13, Not applicable 6, Phase 4 3, Phase 3 3, Phase 2/3 2, Phase 2 31, Phase 1/2 20, Phase 1 15) 2014: 79 trials (Observational 9, Not applicable 8, Phase 4 1, Phase 3 4, Phase 2/3 2, Phase 2 21, Phase 1/2 12, Phase 1 22) 2015: 80 trials (Observational 6, Not applicable 5, Phase 3 3, Phase 2 34, Phase 1/2 12, Phase 1 20) 2016: 90 trials (Observational 11, Not applicable 8, Phase 4 1, Phase 3 4, Phase 2/3 3, Phase 2 24, Phase 1/2 18, Phase 1 21) 2017: 87 trials (Observational 10, Not applicable 6, Phase 4 2, Phase 3 7, Phase 2 28, Phase 1/2 13, Phase 1 21) 2018: 93 trials (Observational 10, Not applicable 2, Phase 3 12, Phase 2/3 3, Phase 2 26, Phase 1/2 10, Phase 1 30) 2019: 86 trials (Observational 10, Not applicable 4, Phase 4 1, Phase 3 13, Phase 2 24, Phase 1/2 7, Phase 1 27) 2020: 93 trials (Observational 16, Not applicable 3, Phase 3 7, Phase 2/3 1, Phase 2 21, Phase 1/2 13, Phase 1 32) 2021: 105 trials (Observational 12, Not applicable 11, Phase 4 1, Phase 3 8, Phase 2/3 3, Phase 2 29, Phase 1/2 21, Phase 1 20) 2022: 86 trials (Observational 10, Not applicable 7, Phase 3 5, Phase 2/3 2, Phase 2 23, Phase 1/2 15, Phase 1 24) 2023: 82 trials (Observational 11, Not applicable 8, Phase 4 2, Phase 3 10, Phase 2/3 1, Phase 2 24, Phase 1/2 10, Phase 1 16) 2024: 93 trials (Observational 20, Not applicable 4, Phase 3 5, Phase 2/3 2, Phase 2 31, Phase 1/2 6, Phase 1 25) 2025: 94 trials (Observational 14, Not applicable 10, Phase 3 6, Phase 2 32, Phase 1/2 11, Phase 1 21)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 88
2025 94
2024 93
2023 82
2022 86
2021 105
2020 93
2019 86
2018 93
2017 87

Showing 10 of 36 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 100 trials starting in 2026 and about 92 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 243 trials still running at the 2026-10-09 snapshot; registered median of 41 months across 1,523 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Myelodysplastic Syndromes trials in? (10)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 518
Phase 1/2 293
Phase 2 817
Phase 2/3 30
Phase 3 175
Phase 4 30
Not applicable 145
Observational 274
Expanded access 14
Unknown 1

How many Myelodysplastic Syndromes trials are recruiting? (12)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 61
Recruiting 298
Enrolling by invitation 10
Active, not recruiting 148
Available 5

Showing 5 of 12 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Myelodysplastic Syndromes trials? (5)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
National Cancer Institute (NCI) 71
Novartis Pharmaceuticals 52
Celgene 45
National Heart, Lung, and Blood Institute (NHLBI) 38
Alexion Pharmaceuticals, Inc. 32

Which drugs are being tested for Myelodysplastic Syndromes? (4)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
Cytoxan 229
Busulfan 142
Decitabine 98
Melphalan 92

Interventions as named in the registry (11)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 281
Azacitidine MTD 194
Mofetil Mycophenolate 137
IR Tacrolimus 133
Total Body Irradiation 125

Showing 5 of 11 interventions.

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These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Myelodysplastic Syndromes trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Febrile neutropenia 2,053 180
Pneumonia 1,506 174
Thrombocytopenia 1,013 90
Sepsis 977 208
Neutropenia 858 62

Showing 5 of 10 adverse events.

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Which publications cover Myelodysplastic Syndromes? (895)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Phase 1 study of rogocekib in patients with relapsed or refractory hematologic malignancies. Blood advances 2026 Yes
Transplantation in patients with lower-risk MDS: a prospective phase 2 trial based on donor availability. Blood advances 2026 Yes
Estimating the Causal Effect of Realistic Treatment Strategies Using Longitudinal Observational Data. Medical decision making : an international journal of the Society for Medical Decision Making 2026 Yes
NCCN Guidelines® Insights: Myelodysplastic Syndromes, Version 2.2025. Journal of the National Comprehensive Cancer Network : JNCCN 2025 Yes
A Phase I Study of FHD-286, a Dual BRG1/BRM (SMARCA4/SMARCA2) Inhibitor, in Patients with Advanced Myeloid Malignancies. Clinical cancer research : an official journal of the American Association for Cancer Research 2025 Yes

Showing 5 of 895 publications.

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Prevalence (34)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Myelodysplastic syndrome Annual incidence 1-9 / 100 000 United Kingdom GB Exact
Myelodysplastic syndrome Annual incidence 1-9 / 100 000 United Kingdom GB Exact
Myelodysplastic syndrome Point prevalence 1-5 / 10 000 United States US Exact
Myelodysplastic syndrome Annual incidence 1-9 / 100 000 United States US Exact
Myelodysplastic syndrome Point prevalence 1-5 / 10 000 United States US Exact

Showing 5 of 34 prevalence observations.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Myelodysplastic syndrome 52688 Exact Orphanet Rare Disease Ontology (ORDO)
Myelodysplastic syndrome 52688 Exact Orphadata Science knowledge files, Product 1

Sub-conditions in MeSH (4)

MeSH descendants one level down with at least one trial, most trials first.

Not on this page

Not measured: delivery record.

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