Exemestane in Treating Patients With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial Neoplasia or Low Grade Endometrial Cancer

NCT03300557 | Completed Phase 2 Results DMC FDA Drug

• 7 other identifiers: NCI-2017-01782UW17010N01-CN-2012-000332016LS183 / UWI17010/UAB1788UWI2016-08-01N01CN00033P30CA014520
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Atypical Hyperplasia; Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia; Endometrial Carcinoma; FIGO Grade 1 Endometrial Endometrioid Adenocarcinoma; FIGO Grade 2 Endometrial Endometrioid Adenocarcinoma

Outcome Measures

Primary Outcomes (1)

• Change in Tumor Proliferation

  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.

  Baseline up to 2 months

Secondary Outcomes (8)

• Changes in Circulating Serum Estradiol

  Baseline up to 2 months

• Changes in Circulating Serum Progesterone

  Baseline up to 2 months

• Percent of Participants by Pathological Response Class at 2 Months

  Up to 2 months

• Change From Baseline in Percent of Cells Positive for Tissue Markers

  Up to 2 months

• Deoxyribonucleic Acid (DNA) Mutational Analysis

  Up to 2 months

Study Arms (1)

Treatment (exemestane)

EXPERIMENTAL

Patients receive exemestane PO QD over 21-42 days in the absence of disease progression or unaccepted toxicity. Patients undergo standard of care surgery between days 22-43.

Drug: Exemestane; Other: Laboratory Biomarker Analysis; Other: Pharmacokinetic Study; Other: Questionnaire Administration

Interventions

Exemestane DRUG

Given PO

Also known as: Aromasin, FCE-24304

Treatment (exemestane)

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (exemestane)

Pharmacokinetic Study OTHER

Correlative studies

Also known as: PHARMACOKINETIC, PK Study

Treatment (exemestane)

Questionnaire Administration OTHER

Ancillary studies

Treatment (exemestane)

Eligibility Criteria

• Age: 45 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 \< 2 years of amenorrhea and follicle stimulating hormone (FSH) within institutional post-menopausal range.
• Age 45 to 55 years of age with FSH within institutional post-menopausal range. The Ki-67 expression changes based on menopausal status and specifically varies based on what phase of the menstrual cycle the sample is collected. Therefore, in order to eliminate this source of variability, only postmenopausal women will be included in this trial. In addition, exemestane is currently approved for use in post-menopausal women only.
• Eastern Cooperative Oncology Group (ECOG) performance status =\< 1
• Hemoglobin \>= 9 g/dL
• Serum creatinine =\< 1.5 x upper limit of normal or calculated creatinine clearance \>= 60 mL/min using Cockcroft-Gault equation for patients with creatinine levels \> 1.5 x institutional upper limit of normal (ULN)
• Total bilirubin =\< 1.5 x ULN OR direct bilirubin =\< 1 x ULN
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =\< 2.5 x ULN
• White blood cell (WBC) \>= 3000/mcl
• Platelets \>= 100,000/mcl
• Able and willing to take oral medications

You may not qualify if:

• 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)

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (3)

University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States

Location

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States

Location

MeSH Terms

Conditions

Endometrial Hyperplasia; Endometrial Neoplasms

Interventions

exemestane; Pharmacogenomic Variants

Condition Hierarchy (Ancestors)

Uterine Diseases; Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases; Uterine Neoplasms; Genital Neoplasms, Female; Urogenital Neoplasms; Neoplasms by Site; Neoplasms

Intervention Hierarchy (Ancestors)

Polymorphism, Genetic; Genetic Variation; Genetic Phenomena

Results Point of Contact

• Title: Britt Erickson
• Organization: University of Minnesota/Masonic Cancer Center

bkeric@umn.edu

612-626-6283

Study Officials

• Britt K Erickson

  University of Wisconsin, Madison

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2017
• First Posted: October 3, 2017
• Study Start: November 15, 2017
• Primary Completion: December 13, 2023
• Study Completion: August 14, 2025
• Last Updated: August 29, 2025
• Results First Posted: June 3, 2025

Record last verified: 2025-08

Locations

US (3)