P4, N=100, Recruiting, University of California, San Francisco

Emerging evidence suggests aberrant progesterone signalling contributes to TNBC risk, while preclinical studies indicate progesterone receptor modulators such as mifepristone may substantially reduce cancer development. DNA methylation-based markers reflecting field cancerization and replicative age could enable real-time monitoring of preventive efficacy and support an individualised primary prevention approach.

P1, N=20, Completed, Nova Southeastern University | Active, not recruiting --> Completed

P3, N=615, Active, not recruiting, Leiden University Medical Center | Recruiting --> Active, not recruiting | N=1186 --> 615 | Trial completion date: Apr 2030 --> Jan 2028 | Trial primary completion date: Apr 2029 --> Jun 2027

Of the 10 patients, 60% experienced adverse events, including one grade 3 increase in gamma-glutamyltransferase. In conclusion, ONA-XR showed early tumor proliferation suppression, particularly in high PgR-expressing tumors, supporting its potential for further clinical evaluation.

P=N/A, N=637, Completed, University of California, San Francisco

In vivo, ACC-bearing mice were treated with immune checkpoint inhibitors and mifepristone...Moreover, the percentage of macrophages detected by immunohistochemistry positively correlated with response to immunotherapy. Collectively, this study identifies a glucocorticoid-induced C1Q+ CD163 + macrophage phenotype that may contribute to T cell recruitment and enhance the efficacy of immunotherapy in ACC.

P=N/A, N=108, Completed, Affiliated Hospital of Nantong University; Affiliated Hospital of Nantong University | Not yet recruiting --> Completed

These findings demonstrate that M. longistyla extract possesses significant anti-hemorrhagic activity evidenced by reduced uterine bleeding, suppression of pro-inflammatory cytokine (TNF-α) and upregulation of VEGF. These effects suggest that the extract promotes endometrial repair and restoration of uterine homeostasis.

P3, N=1000, Not yet recruiting, University of Pennsylvania

Short-term mifepristone exposure does not produce molecular changes linked to endometrial carcinogenesis in BRCA1/2 pathogenic variant carriers. These findings provide important safety data for the future development of progesterone receptor modulators in cancer prevention. Long-term studies are needed to confirm these observations.

The study found that mifepristone treatment significantly reduced serum CA125 and PRL levels, and the 20 mg/kg/day dose was most effective in improving pregnancy outcomes. These findings suggest that mifepristone can reduce key biomarkers associated with uterine adenomyosis and improve fertility outcomes, offering a promising treatment option for women suffering from this condition.