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Atypical HyperplasiaPhase 2NCT03300557

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

A Phase 2 study testing Exemestane, Laboratory Biomarker Analysis and Pharmacokinetic Study in people with Atypical Hyperplasia and Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia, run by National Cancer Institute (NCI) at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 45 years and older, women only
What is being tested?
Exemestane, Laboratory Biomarker Analysis and Pharmacokinetic Study
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
3 sitesUnited States
Study dates
15 Nov 2017to 14 Aug 2025
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 45 years and older
  • Women only
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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
  • Ability to understand and the willingness to sign a written informed consent document
  • Body mass index (BMI) > 20

Who cannot join

  • 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
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  • 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)

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • University of Alabama at Birmingham Cancer Center

    Birmingham, Alabama, 35233

  • University of Wisconsin Carbone Cancer Center - University Hospital

    Madison, Wisconsin, 53792

  • University of Minnesota/Masonic Cancer Center

    Minneapolis, Minnesota, 55455

Other trials in United States

About this study

Official title: Pilot Study of Daily Exemestane in Women With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial Neoplasia or Low Grade Endometrial Cancer

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To determine if there is a decrease in proliferation index, measured by Ki-67 expression, in complex atypical hyperplasia (CAH)/endometrial intraepithelial neoplasia (EIN) or low grade (grade 1 and grade 2) endometrial cancer cells from baseline to post-exemestane treatment.

SECONDARY OBJECTIVES:

  1. Circulating serum estradiol and progesterone. II. Pathological response (regression of CAH/EIN or low grade [grade 1 and grade 2] endometrial carcinoma).

III. Tissue biomarkers. IV. Deoxyribonucleic acid (DNA) mutational analysis through next generation sequencing and methylation status of endometrial tumor.

  1. Protein markers via tampon recovery before and after treatment. VI. DNA markers via tampon recovery. VII. Safety and adverse effects of treatment. VIII. Comparison of Ki-67 expression changes between study subjects and a historical cohort.

IX. Evaluation of the levels of exemestane in the plasma samples pre and post treatment.

OUTLINE:

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

After completion of study treatment, patients with unresolved adverse events on day of surgery are followed up periodically.

Being tested
Exemestane, Laboratory Biomarker Analysis, Pharmacokinetic Study, Questionnaire Administration
Medical subject headings
Endometrial Hyperplasia, Endometrial Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ExemestaneDrug

    Also called Aromasin, FCE-24304

    Given PO

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • Pharmacokinetic StudyOther

    Also called PHARMACOKINETIC, PK Study

    Correlative studies

  • Questionnaire AdministrationOther

    Ancillary studies

Study groups in NCT03300557
Study groupRoleWhat they receive
Treatment (exemestane)ExperimentalPatients 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.
Receives: Drug: Exemestane; Other: Laboratory Biomarker Analysis; Other: Pharmacokinetic Study; Other: Questionnaire Administration

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Baseline up to 2 months

Secondary outcomes (8)

  1. Changes in Circulating Serum Estradiol

    Circulating serum estradiol pre and post treatment to determine the effect of daily dose of 25mg of exemestane for 21-42 days.

    Measured: Baseline up to 2 months

  2. Changes in Circulating Serum Progesterone

    Circulating serum progesterone pre and post treatment to determine the effect of daily dose of 25mg of exemestane for 21-42 days.

    Measured: Baseline up to 2 months

  3. Percent of Participants by Pathological Response Class at 2 Months

    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.

    Measured: Up to 2 months

  4. Change From Baseline in Percent of Cells Positive for Tissue Markers

    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.

    Measured: Up to 2 months

  5. Deoxyribonucleic Acid (DNA) Mutational Analysis

    Will be analyzed by next generation sequencing.

    Measured: Up to 2 months

  6. Protein Markers

    Perform pre- and post-treatment proteomic analysis of vaginal proteins from tampon recovery to identify biomarkers that may predict response to exemestane treatment.

    Measured: Up to 2 months

  7. Ki-67 Expression With Historic Controls

    Will compare Ki-67 expression between participants samples and historically matched samples.

    Measured: Up to 2 months

  8. Plasma Levels of Exemestane

    Will evaluate plasma levels of exemestane pre and post treatment.

    Measured: Up to 2 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Oct 2017
  2. Study start15 Nov 2017
  3. Primary completion13 Dec 2023
  4. Study completion14 Aug 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
40 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03300557
Sponsor's protocol ID
NCI-2017-01782
Other IDs
NCI-2017-01782, UW17010, N01-CN-2012-00033, 2016LS183 / UWI17010/UAB1788, UWI2016-08-01, N01CN00033, P30CA014520
Status last verified
Aug 2025
First submitted
2 Oct 2017
Last updated
29 Aug 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Exemestane, Laboratory Biomarker Analysis and Pharmacokinetic Study in people with Atypical Hyperplasia and Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia, run by National Cancer Institute (NCI) at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Tumor Proliferation (time frame: Baseline up to 2 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03300557.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Aug 2025; AzTrials last refreshed its copy on 7 Nov 2026.

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