Disease × agent evidence record

Diethylstilbestrol for Prostate Cancer: evidence, trials and status

Diethylstilbestrol has both completed registered trials and synthesis-level publications linked to Prostate 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.

5 registered trials 0 recruiting 10 publications Evidence tier A · Strong Score 68.5
Research Tracker › Pairs › Prostate Cancer › Diethylstilbestrol
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

Diethylstilbestrol has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Prostate Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Prostate Cancer. ClinicalTrials.gov lists 5 registered trials linking Diethylstilbestrol to Prostate Cancer: 5 have completed. The most advanced is Phase 3 (NCT00699751). It plans or enrolled 921 participants. Registration activity spans 2002 to 2020. The literature layer holds 10 publications for this pair: 3 Cochrane reviews, 2 randomised controlled trial publications and 5 clinical trial publications. Publication years run from 1999 to 2012. 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 Prostate Cancer 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: Low-grade (Gleason 6/Grade Group 1): active surveillance appropriate in 40-50% — many will never need treatment; high-grade: lethal without treatment within 5-10 years.

Open in Repurposing ExplorerProstate Cancer hubProstate Cancer biomarkers & interventionsChEMBLOpen Targets

Evidence table

Evidence tierA · Strong
Evidence score68.5 (trials 18.5, literature 30.0, tier 15, approved bonus 5.0)
Registered trials5 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 0 other
Linked publications10 (5 clinical trial publications, 3 Cochrane reviews, 2 randomised controlled trial publications)
Agent typeDrug (Small molecule)
Development stage (any indication)Approved
Mechanism / classNot recorded
Data sourcesOpen Targets, ChEMBL
Linked via biomarker / targetACP3
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
NCT00316927Dexamethasone, Aspirin, and Diethylstilbestrol in Treating Patients With Locally Advanced or Metastatic Prostate Cancer
2002
completedPhase 3260
NCT00699751A Phase III Study of Radium-223 Dichloride in Patients With Symptomatic Hormone Refractory Prostate Cancer With Skeletal Metastases
2008
completedPhase 3921
NCT02217566Study of Abiraterone Acetate in Participants With Metastatic Castration-Resistant Prostate Cancer (mCRPC), Chemo-Naive, Who Received a Prior Diethylstilbestrol Therapy
2014
completedPhase 246
NCT00136526Docetaxel and Diethylstilbestrol in the Treatment of Androgen Independent Prostate Cancer
2002
completedPhase 230
NCT04268628A Study of Pharmacodynamic and Genetic Parameters of Abira-DES Study Participants (NCT02217566) - Participants With Metastatic Castration-Resistant Prostate Cancer Treated With Abiraterone Acetate Following Unresponsive Treatment With Diethylstilbestrol
2020
completedNot applicable42

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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, in this case via the biomarker or target ACP3. The tier describes how well the drug-disease link is documented, not how well the drug works.

Frequently asked questions

Is Diethylstilbestrol approved for Prostate Cancer?

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

Is Diethylstilbestrol in clinical trials for Prostate Cancer?

ClinicalTrials.gov lists 5 registered trials linking Diethylstilbestrol to Prostate Cancer: 5 have completed. The most advanced is Phase 3 (NCT00699751). It plans or enrolled 921 participants. Registration activity spans 2002 to 2020.

What does the evidence show for Diethylstilbestrol in Prostate Cancer?

Diethylstilbestrol has both completed registered trials and synthesis-level publications linked to Prostate 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, in this case via the biomarker or target ACP3. The tier describes how well the drug-disease link is documented, not how well the drug works.

Cite this page

Open Source Medicine Foundation. Diethylstilbestrol for Prostate Cancer: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-06. https://research.opensourcemed.info/pairs/prostate-cancer/diethylstilbestrol.html

Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. 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.