Disease × agent evidence record

Decitabine for Cancer: evidence, trials and status

Decitabine has both completed registered trials and synthesis-level publications linked to Cancer. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit.

8 registered trials 0 recruiting 18 publications Evidence tier A · Strong Score 70.0
Research Tracker › Pairs › Cancer › Decitabine
Research map, not treatment advice. This page aggregates registry and literature records. It does not evaluate efficacy, dosing or safety for any individual. Discuss any treatment decision with a qualified clinician.

Summary

Decitabine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. ClinicalTrials.gov lists 8 registered trials linking Decitabine to Cancer: 1 is active or not yet recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00867672). The largest enrolment is 222 participants (NCT04485052). Registration activity spans 2004 to 2022. The literature layer holds 18 publications for this pair: 5 Cochrane reviews, 4 meta-analyses, 1 systematic review, 3 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 2021 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 Cancer ranks 1226 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: Spontaneous regression is rare (<0.5% of cases); 5-year survival without treatment: <5% for advanced NSCLC; ~20% for stage I-II with surgery.

Open in Repurposing ExplorerCancer hubChEMBLOpen Targets

Evidence table

Evidence tierA · Strong
Evidence score70.0 (trials 20.0, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 0 recruiting, 1 active / not yet recruiting, 4 completed, 3 other
Linked publications18 (5 Cochrane reviews, 5 clinical trial publications, 4 meta-analyses, 3 randomised controlled trial publications, 1 systematic review)
Agent typeDrug (Small molecule)
Development stage (any indication)Approved
Mechanism / classNot recorded
Data sourcesOpen Targets, ChEMBL
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 IDTitleStatusPhaseEnrolment
NCT01893372Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS)
2013
completedPhase 229
NCT00867672Study of Decitabine Alone or in Combination With Valproic Acid and All-trans Retinoic Acid in Acute Myeloid Leukemia
2011
completedPhase 2204
NCT00085293Decitabine in Treating Patients With Metastatic Papillary Thyroid Cancer or Follicular Thyroid Cancer Unresponsive to Iodine I 131
2004
completedPhase 212
NCT00740181Decitabine, Cytarabine, GCSF for Refractory AML/MDS
2008
terminatedPhase 29
NCT03613532Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN
2018
active, not recruitingPhase 1102
NCT05829226A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
2022
completedPhase 1101
NCT02412475Epigenetic Reprogramming in Relapse AML
2015
terminatedPhase 13
NCT04485052Efficacy and Safety Evaluation of IBI188 in Combination With Demethylating Agents in Treatment of Patients With Acute Myeloid Leukemia
2020
suspendedPHASE1, PHASE2222

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Published literature

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Mechanism and notes

No mechanism of action is recorded for this pair in the source databases.

OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.

Frequently asked questions

Is Decitabine approved for Cancer?

Decitabine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.

Is Decitabine in clinical trials for Cancer?

ClinicalTrials.gov lists 8 registered trials linking Decitabine to Cancer: 1 is active or not yet recruiting; 4 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00867672). The largest enrolment is 222 participants (NCT04485052). Registration activity spans 2004 to 2022.

What does the evidence show for Decitabine in Cancer?

Decitabine has both completed registered trials and synthesis-level publications linked to Cancer. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.

Cite this page

Open Source Medicine Foundation. Decitabine for Cancer: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/cancer/decitabine.html

Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.

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This is a research map, not treatment advice. Evidence tiers and scores summarise what has been studied, not whether a treatment works or is safe for you.