Phase 2
N=40
Exemestane in Treating Patients With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial Neoplasia or Low Grade Endometrial Cancer
Atypical Hyperplasia · Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia · Endometrial Carcinoma · FIGO Grade 1 Endometrial Endometrioid Adenocarcinoma · FIGO Grade 2 Endometrial Endometrioid Adenocarcinoma
Bottom Line
View on ClinicalTrials.gov: NCT03300557 ↗Enrolled (actual)
40
Serious AEs
0.0%
Results posted
Jun 2025
Primary outcome: Primary: Change in Tumor Proliferation — -0.17 percent (represented by mean) of cells
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Exemestane (Drug); Laboratory Biomarker Analysis (Other); Pharmacokinetic Study (Other); Questionnaire Administration (Other)
- Age
- Adult, Older Adult · 45+ yrs
- Sex
- Female
- Sponsor
- National Cancer Institute (NCI)
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Tumor Proliferation |
-0.17 | — |
| SECONDARY Changes in Circulating Serum Estradiol |
4.08 | — |
| SECONDARY Changes in Circulating Serum Progesterone |
0.22 | — |
| SECONDARY Percent of Participants by Pathological Response Class at 2 Months |
2.5; 20; 57.5; 10; 10 | — |
| SECONDARY Change From Baseline in Percent of Cells Positive for Tissue Markers |
-0.17; -0.07; -0.12; -2.50 | — |
| SECONDARY Deoxyribonucleic Acid (DNA) Mutational Analysis |
NA | — |
| SECONDARY Protein Markers |
85 | — |
| SECONDARY Ki-67 Expression With Historic Controls |
-0.07 | — |
| SECONDARY Plasma Levels of Exemestane |
2.51 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Females with a histologically proven CAH/ EIN or low grade (grade 1 or grade 2) endometrial carcinoma (EC) for which surgery is planned; the pathologic report from the referring facility will be used to determine pathologic eligibility; this report must be within 45 days of their baseline (pre-surgical) clinic visit
- No prior treatment for CAH/EIN/EC
- Post-menopausal confirmed with one the following criteria:
- >= 60 years of age
- Age 56 to 59 years of age with >= 2 years of amenorrhea
- Age 56 to 59 years of age with = 9 g/dL
- Serum creatinine = = 60 mL/min using Cockcroft-Gault equation for patients with creatinine levels > 1.5 x institutional upper limit of normal (ULN)
- Total bilirubin = = 3000/mcl
- Platelets >= 100,000/mcl
- Able and willing to take oral medications
- Ability to understand and the willingness to sign a written informed consent document
- Body mass index (BMI) > 20
Exclusion Criteria
- Participants who had curatively treated invasive malignancies for which all treatments ended within 1 year prior to the study (with the exception of basal cell or squamous cell carcinoma of the skin)
- Not a surgical candidate or surgery is not scheduled within 43 days from starting the study drug
- Receiving any other investigational agents
- Any gastrointestinal condition causing malabsorption or obstruction (e.g. celiac sprue, gastric bypass surgery, strictures, adhesions, history of small bowel resection, blind loop syndrome)
- Has been on any hormonal treatment (including progestin-containing intrauterine device [IUD]) for CAH/EIN or low grade (grade 1 or grade 2) endometrial carcinoma in last 3 months
- Use hormone replacement therapy (including systemic or topical estrogen, progesterone, or testosterone based medication) or/and phytoestrogen supplements (i.e. black cohosh) or has been on progestin (including progestin containing IUD), tamoxifen or aromatase inhibitor within the prior 3 months
- Concomitant use of strong CYP3A4 inducers such as rifampicin, phenytoin, carbamazepine, phenobarbital or St. John's wort as these may significantly reduce the availability of exemestane
- Known hypersensitivity to exemestane or its excipients
- Known intercurrent illness or psychiatric illness/social situations that will limit compliance with study requirements
- Evidence or high suspicion of metastatic disease at enrollment
- Women with severe bone density issues/osteoporosis (defined as any medical treatment for osteoporosis, and/or a T-score of -2.5 or lower, and/or history of fracture of the hip or spine)
- Unwilling or unable to undergo research biopsy during the baseline (pre-surgical) clinic visit, or inadequate research biopsy obtained during the baseline (pre-surgical) clinic visit (determined by the gynecologic oncologist at the time of the subject's pelvic exam)
Data sourced from ClinicalTrials.gov (NCT03300557). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.