Disease × agent evidence record

Megestrol Acetate for Cancer: evidence, trials and status

Megestrol Acetate 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 1 recruiting 14 publications Evidence tier A · Strong Score 73.0
Research Tracker › Pairs › Cancer › Megestrol Acetate
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

Megestrol Acetate 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 Megestrol Acetate to Cancer: 1 is currently recruiting; 3 are active or not yet recruiting; 1 has completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT06963593). It plans or enrolled 220 participants. Registration activity spans 1987 to 2027. The literature layer holds 14 publications for this pair: 5 Cochrane reviews, 2 meta-analyses, 1 systematic review, 1 randomised controlled trial publication 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 score73.0 (trials 23.0, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 1 recruiting, 3 active / not yet recruiting, 1 completed, 3 other
Linked publications14 (5 Cochrane reviews, 5 clinical trial publications, 2 meta-analyses, 1 systematic review, 1 randomised controlled trial publication)
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
NCT07462663SHAPE-ENDO: Pilot Randomized Trial of Multimodal Pre-Surgical Optimization Versus Standard Surgery in Patients With Obesity and Early-Stage Endometrial Cancer
2027
not yet recruitingPhase 480
NCT06963593Clinical Study on the Treatment of Anorexia and Cachexia in Advanced Gastric and Esophageal Cancer With Nanosized Megestrol Acetate Combined With Immunochemotherapy
2025
not yet recruitingPhase 4220
NCT03283488Comparison Between Mirtazapine and Megestrol for the Control of Anorexia-cachexia in Cancer Patients in Palliative Care.
2019
completedPhase 252
NCT07092137Nano-crystalline Megestrol Acetate for Anorexia-Cachexia Syndrome in Advanced Lung Cancer
2025
not yet recruitingPhase 2116
NCT05913427Evaluation of the Efficacy of Addition of Progesterone to Standard Chemotherapy in Adrenocortical Carcinoma (ACC)
2022
recruitingPhase 280
NCT02980653Megestrol Acetate Against Cancer-related Critical Body Weight Loss in Patients With H&N Cancer Who Receiving CCRT
2015
status unknownPhase 2105
NCT04046185Programmed Death-1(PD-1) Inhibitor Combined With Progesterone Treatment in Endometrial Cancer
2019
status unknownEarly Phase 160
NCT00002465High-Dose Megestrol in Treating Patients With Metastatic Breast Cancer, Endometrial Cancer, or Mesothelioma
1987
status unknownPHASE1, PHASE2—

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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 Megestrol Acetate approved for Cancer?

Megestrol Acetate 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 Megestrol Acetate in clinical trials for Cancer?

ClinicalTrials.gov lists 8 registered trials linking Megestrol Acetate to Cancer: 1 is currently recruiting; 3 are active or not yet recruiting; 1 has completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT06963593). It plans or enrolled 220 participants. Registration activity spans 1987 to 2027.

What does the evidence show for Megestrol Acetate in Cancer?

Megestrol Acetate 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. Megestrol Acetate for Cancer: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/cancer/megestrol-acetate.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.