Summary
Eflornithine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Colorectal Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Colorectal Cancer. ClinicalTrials.gov lists 7 registered trials linking Eflornithine to Colorectal Cancer: 1 is active or not yet recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00118365). It plans or enrolled 375 participants. Registration activity spans 1998 to 2022. The literature layer holds 2 publications for this pair: 1 systematic review and 1 clinical trial publication. Publication years run from 2016 to 2023. 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 Colorectal Cancer ranks 1212 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; 5-year survival stage I: ~90% (resectable); stage IV: ~15% overall, ~20-30% with modern therapy and liver metastasis resection.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 49.0 (trials 24.0, literature 5.0, tier 15, approved bonus 5.0) |
| Registered trials | 7 total: 0 recruiting, 1 active / not yet recruiting, 4 completed, 2 other |
| Linked publications | 2 (1 systematic review, 1 clinical trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | APC |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01349881 | S0820, Adenoma and Second Primary Prevention Trial 2013 | active, not recruiting | Phase 3 | 354 |
| NCT00118365 | Eflornithine and Sulindac in Preventing Colorectal Cancer in Patients With Colon Polyps 1998 | completed | Phase 3 | 375 |
| NCT01483144 | Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP) 2013 | completed | Phase 3 | 171 |
| NCT01245816 | A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP) 2011 | withdrawn | Phase 3 | — |
| NCT00983580 | Acetylsalicylic Acid and Eflornithine in Treating Patients at High Risk for Colorectal Cancer 2009 | completed | Phase 2 | 107 |
| NCT00033371 | Celecoxib With or Without Eflornithine in Preventing Colorectal Cancer in Patients With Familial Adenomatous Polyposis 2001 | completed | Phase 2 | 205 |
| NCT05500508 | Oral AMXT 1501 Dicaprate in Combination With IV DFMO 2022 | terminated | PHASE1, PHASE2 | 15 |
Published literature
- Systematic review Eflornithine for chemoprevention in the high-risk population of colorectal cancer: a systematic review and meta-analysis with trial sequential analysisYang L, Wang Y, Hu S et al. · Frontiers in oncology · 2023 · PMID 38033490
- Clinical trial publication Efficacy and safety of eflornithine (CPP-1X)/sulindac combination therapy versus each as monotherapy in patients with familial adenomatous polyposis (FAP): design and rationale of a randomized, double-blind, Phase III trialBurke CA, Dekker E, Samadder NJ et al. · BMC gastroenterology · 2016 · PMID 27480131
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 DGIdb, in this case via the biomarker or target APC. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Eflornithine approved for Colorectal Cancer?
Eflornithine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Colorectal Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Colorectal Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Eflornithine in clinical trials for Colorectal Cancer?
ClinicalTrials.gov lists 7 registered trials linking Eflornithine to Colorectal Cancer: 1 is active or not yet recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00118365). It plans or enrolled 375 participants. Registration activity spans 1998 to 2022.
What does the evidence show for Eflornithine in Colorectal Cancer?
Eflornithine has both completed registered trials and synthesis-level publications linked to Colorectal 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 DGIdb, in this case via the biomarker or target APC. 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 2026-07-05. Page built 2026-10-07.