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Exemestane in Treating Patients With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial Neoplasia or Low Grade Endometrial Cancer
Phase 2 trial testing Exemestane in Atypical Hyperplasia in 40 participants. Completed in 14 August 2025.
| Lead sponsor | National Cancer Institute (NCI) |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | prevention |
| Enrollment | 40 |
| Start date | 15 November 2017 |
| Primary completion | 13 December 2023 |
| Estimated completion | 14 August 2025 |
| Sites | 3 locations across United States |
National Cancer Institute (NCI)
45 and older, female only, with Atypical Hyperplasia or Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Will be measured by change in Ki-67 expression. Will evaluate the change from baseline to post-exposure in absolute change in percent Ki-67 using one-sample Student's t-test or Wilcoxon signed-rank test, as appropriate.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -0.17 | ± 0.22 |
Circulating serum estradiol pre and post treatment to determine the effect of daily dose of 25mg of exemestane for 21-42 days.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 4.08 | ± 13.81 |
Circulating serum progesterone pre and post treatment to determine the effect of daily dose of 25mg of exemestane for 21-42 days.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 0.22 | ± 0.27 |
This measure assesses change in categories in pathological response. As pathological response is an ordered categorical variable with classes of No visible lesion, CAH/EIN, Grade I, Grade II, and Grade III in this study, a change in class from baseline to time of surgery represents a decrease or increase in disease severity.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 2.5 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 20 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 57.5 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 10 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 10 |
Assessment of change from baseline for apoptosis (cleaved caspase 3), proliferation (cyclin D1), insulin pathway (pAKT, IGF-1R), and endocrine regulation (estrogen receptor/progesterone receptor/androgen receptor). The units for absolute change in is % Positive.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -0.17 | ± 0.18 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -0.07 | ± 0.22 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -0.12 | ± 0.27 |
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -2.50 | ± 3.00 |
Will be analyzed by next generation sequencing.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | NA |
Perform pre- and post-treatment proteomic analysis of vaginal proteins from tampon recovery to identify biomarkers that may predict response to exemestane treatment.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 85 |
Will compare Ki-67 expression between participants samples and historically matched samples.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | -0.07 | ± 0.10 |
Will evaluate plasma levels of exemestane pre and post treatment.
| Group | Value | 95% CI |
|---|---|---|
| Treatment (Exemestane) | 2.51 | ± 1.23 |
Time frame: 3 months. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Treatment (Exemestane) |
|---|---|---|
| Hot flashes | Vascular disorders | — |
| Hypertension | Vascular disorders | — |
| Diarrhea | Gastrointestinal disorders | — |
| Fatigue | General disorders | — |
| Abdominal pain | Gastrointestinal disorders | — |
| Headache | Nervous system disorders | — |
| Pruritus | Skin and subcutaneous tissue disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — |
| Nausea | Gastrointestinal disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| Dizziness | Nervous system disorders | — |
| Edema limbs | General disorders | — |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — |
| Back pain | Musculoskeletal and connective tissue disorders | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — |
| Bloating | Gastrointestinal disorders | — |
| Arthritis | Musculoskeletal and connective tissue disorders | — |
| Allergic rhinitis | Respiratory, thoracic and mediastinal disorders | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — |
| Sneezing | Respiratory, thoracic and mediastinal disorders | — |
| Anxiety | Psychiatric disorders | — |
| Insomnia | Psychiatric disorders | — |
| Urinary frequency | Renal and urinary disorders | — |
| Flushing | Vascular disorders | — |
| Dyspepsia | Gastrointestinal disorders | — |
| Stomach pain | Gastrointestinal disorders | — |
| Blurred vision | Eye disorders | — |
| Eye disorders - Other, specify | Eye disorders | — |
| Chest wall pain | Musculoskeletal and connective tissue disorders | — |
| Musculoskeletal and connective tissue disorder - Other, specify | Musculoskeletal and connective tissue disorders | — |
| Concentration impairment | Nervous system disorders | — |
| Dysgeusia | Nervous system disorders | — |
| Paresthesia | Nervous system disorders | — |
| Chills | General disorders | — |
| Fever | General disorders | — |
| Flu like symptoms | General disorders | — |
| Alopecia | Skin and subcutaneous tissue disorders | — |
Data from ClinicalTrials.gov NCT03300557 adverse events section.
This pilot phase IIa trial studies how well exemestane works in treating patients with complex atypical hyperplasia of the endometrium/endometrial intraepithelial neoplasia or low grade endometrial cancer. Exemestane may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
4 peer-reviewed publications reference this trial (live from Europe PMC):
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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing