Summary
Cedazuridine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). ClinicalTrials.gov lists 8 registered trials linking Cedazuridine to Myelodysplastic Syndromes (MDS): 3 are currently recruiting; 4 are active or not yet recruiting; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT05201066). The largest enrolment is 102 participants (NCT03613532). Registration activity spans 2018 to 2025. The literature layer holds 4 publications for this pair: 1 systematic review and 3 clinical trial publications. Publication years run from 2024 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Myelodysplastic Syndrome ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 53.5 (trials 24.5, literature 9.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 3 recruiting, 4 active / not yet recruiting, 0 completed, 1 other |
| Linked publications | 4 (3 clinical trial publications, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | inhibitor; inhibitor |
| Data sources | DGIdb |
| Linked via biomarker / target | CDA |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT05201066 | 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. 2023 | active, not recruiting | Phase 2 | 33 |
| NCT03613532 | Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN 2018 | active, not recruiting | Phase 1 | 102 |
| NCT03906695 | Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes 2019 | active, not recruiting | Phase 1 | 30 |
| NCT06484062 | Testing the Anti-cancer Drug, Cirtuvivint, and Its Combination With ASTX727 to Improve Outcomes in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes 2025 | recruiting | Phase 1 | 54 |
| NCT04953897 | Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment 2021 | recruiting | Phase 1 | 18 |
| NCT04655755 | Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia 2021 | active, not recruiting | PHASE1, PHASE2 | 52 |
| NCT05010122 | ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome 2021 | recruiting | PHASE1, PHASE2 | 42 |
| NCT04013880 | ASTX727 and FT-2102 in Treating IDH1-Mutated Recurrent/Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia 2019 | withdrawn | PHASE1, PHASE2 | — |
Published literature
- Clinical trial publication Venetoclax/FluBu2 RIC transplant followed by all-oral venetoclax/decitabine maintenance for poor-risk MDS/AMLGarcia JS, Kim HT, Murdock HM et al. · Blood advances · 2026 · PMID 41348911
- Systematic review Health-related quality of life of patients with acute myeloid leukemia and myelodysplastic syndromes/neoplasms treated with decitabine: a systematic literature reviewCannella L, Venditti A, Palmieri R et al. · Annals of hematology · 2026 · PMID 41995852
- Clinical trial publication Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trialRobin M, D'Aveni M, Stamatoullas A et al. · The Lancet. Haematology · 2025 · PMID 40784355
- Clinical trial publication Oral decitabine and cedazuridine plus venetoclax for older or unfit patients with acute myeloid leukaemia: a phase 2 studyBazinet A, Garcia-Manero G, Short N et al. · The Lancet. Haematology · 2024 · PMID 38452788
Mechanism and notes
Recorded mechanism or class: inhibitor; inhibitor.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CDA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Cedazuridine approved for Myelodysplastic Syndromes (MDS)?
Cedazuridine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Cedazuridine in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Cedazuridine to Myelodysplastic Syndromes (MDS): 3 are currently recruiting; 4 are active or not yet recruiting; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT05201066). The largest enrolment is 102 participants (NCT03613532). Registration activity spans 2018 to 2025.
What does the evidence show for Cedazuridine in Myelodysplastic Syndromes (MDS)?
Trials of Cedazuridine in Myelodysplastic Syndromes (MDS) are registered but none has completed, so there is no outcome evidence from those studies yet; the record is a signal of research interest. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CDA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.