Disease × agent evidence record

Denileukin Diftitox for Cancer: evidence, trials and status

Denileukin Diftitox 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 13 publications Evidence tier A · Strong Score 70.5
Research Tracker › Pairs › Cancer › Denileukin Diftitox
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

Denileukin Diftitox 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ClinicalTrials.gov lists 8 registered trials linking Denileukin Diftitox to Cancer: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00051012). It plans or enrolled 86 participants. Registration activity spans 1995 to 2009. The literature layer holds 13 publications for this pair: 3 Cochrane reviews, 1 meta-analysis, 4 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 2001 to 2025. 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 score70.5 (trials 20.5, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 3 other
Linked publications13 (5 clinical trial publications, 4 randomised controlled trial publications, 3 Cochrane reviews, 1 meta-analysis)
Agent typeBiologic
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
NCT00051012Study of ONTAK (Denileukin Diftitox) in Previously Treated Cutaneous T-Cell Lymphoma Patients
1995
completedPhase 486
NCT00493129Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)
2004
completedPhase 28
NCT00003615Denileukin Diftitox in Treating Patients With Non-Hodgkin's Lymphoma
1999
completedPhase 277
NCT00880360A Trial of Intravenous Denileukin Diftitox in Stage III or IV Ovarian Cancer
2007
completedPhase 219
NCT00726037A Pilot Study, Evaluating the Efficacy of Regulatory T-cell Suppression by Ontak in Metastatic Pancreatic Cancer
2008
terminatedPhase 27
NCT00278369Pilot Study of Denileukin Diftitox Plus High-Dose IL-2 for Patients With Metastatic Renal Cancer
2005
completedEarly Phase 120
NCT00945269Therapeutic Autologous Lymphocytes, Aldesleukin, and Denileukin Diftitox in Treating Patients With Stage III-IV Melanoma
2009
terminatedPHASE1, PHASE23
NCT00513188Combination Chemotherapy and Denileukin Diftitox in Treating Patients With Newly Diagnosed T-Cell Non-Hodgkin Lymphoma
2007
withdrawnNot applicable—

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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 Denileukin Diftitox approved for Cancer?

Denileukin Diftitox 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 Denileukin Diftitox in clinical trials for Cancer?

ClinicalTrials.gov lists 8 registered trials linking Denileukin Diftitox to Cancer: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00051012). It plans or enrolled 86 participants. Registration activity spans 1995 to 2009.

What does the evidence show for Denileukin Diftitox in Cancer?

Denileukin Diftitox 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. Denileukin Diftitox for Cancer: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/cancer/denileukin-diftitox.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.