Trial Outcomes & Findings for Exemestane in Treating Patients With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial Neoplasia or Low Grade Endometrial Cancer (NCT NCT03300557)

Last Updated: 2025-08-29

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

40 participants

Primary outcome timeframe

Baseline up to 2 months

Results posted on

2025-08-29

Participant Flow

We are aware that CTRP lists 46 as enrollment. PRS shall remain as 40 because: "Potential participants who are screened for the purpose of determining eligibility for the study, but do not participate in the study, are not considered enrolled." Thus, 40 is the proper number because the other 6 were screened and found ineligible, therefore not considered enrolled.

Participant milestones

Participant milestones
Measure	Treatment (Exemestane) 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Overall Study STARTED	40
Overall Study COMPLETED	40
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Exemestane) n=40 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Age, Customized 50-59 years	13 Participants n=5 Participants
Age, Customized 60-69 years	18 Participants n=5 Participants
Age, Customized 70-79 years	7 Participants n=5 Participants
Age, Customized Unknown	2 Participants n=5 Participants
Sex: Female, Male Female	40 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	38 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	38 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	40 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline up to 2 months

Population: There was not enough tissue to analyze from the other two participants

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=38 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Change in Tumor Proliferation	-0.17 percent (represented by mean) of cells Standard Deviation 0.22

SECONDARY outcome

Timeframe: Baseline up to 2 months

Population: Site did not collect data on the 3 participants omitted from the count

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

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=37 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Changes in Circulating Serum Estradiol	4.08 pg/mL Standard Deviation 13.81

SECONDARY outcome

Timeframe: Baseline up to 2 months

Population: Site did not collect the relevant information for the omitted 3 participants

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

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=37 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Changes in Circulating Serum Progesterone	0.22 ng/mL Standard Deviation 0.27

SECONDARY outcome

Timeframe: Up to 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.

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=40 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Percent of Participants by Pathological Response Class at 2 Months No visible lesion	2.5 Percent of participants
Percent of Participants by Pathological Response Class at 2 Months CAH/EIN	20 Percent of participants
Percent of Participants by Pathological Response Class at 2 Months Grade I (Low) EC	57.5 Percent of participants
Percent of Participants by Pathological Response Class at 2 Months Grade II (Low) EC	10 Percent of participants
Percent of Participants by Pathological Response Class at 2 Months Grade III (High) EC	10 Percent of participants

SECONDARY outcome

Timeframe: Up to 2 months

Population: N=37 for caspase because there was not enough tissue on slide for 2 and 1 block was not sent. N=36 for cyclin D1 because 3 were missing data and 1 block was not sent. N=35 for pAKT because 3 were missing data, 1 did not have enough tissue, and 1 block was not sent. N=22 for IGF-1R because 1 black was not sent, 5 did not have enough tissue, and the rest are missing data.

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.

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=37 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Change From Baseline in Percent of Cells Positive for Tissue Markers Cleaved capsase	-0.17 % positive Standard Deviation 0.18
Change From Baseline in Percent of Cells Positive for Tissue Markers Cyclin D1	-0.07 % positive Standard Deviation 0.22
Change From Baseline in Percent of Cells Positive for Tissue Markers pAKT	-0.12 % positive Standard Deviation 0.27
Change From Baseline in Percent of Cells Positive for Tissue Markers IGF-1R	-2.50 % positive Standard Deviation 3.00

SECONDARY outcome

Timeframe: Up to 2 months

Population: Samples were collected from participants. After enrollment was completed, the intended laboratory was no longer available to process the samples. There are no data to report and there is no intent to analyze these samples in the future.

Will be analyzed by next generation sequencing.

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=22 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Deoxyribonucleic Acid (DNA) Mutational Analysis	NA percentage of cells Samples were collected from participants. After enrollment was completed, the intended laboratory was no longer available to process the samples. There are no data to report.

SECONDARY outcome

Timeframe: Up to 2 months

Population: Tampon submission was optional and not all participants submitted tampon samples.

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

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=22 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Protein Markers	85 number of proteins

SECONDARY outcome

Timeframe: Up to 2 months

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

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=40 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Ki-67 Expression With Historic Controls	-0.07 percentage of cells Standard Deviation 0.10

SECONDARY outcome

Timeframe: Up to 2 months

Population: 40th specimen was not collected by the site.

Will evaluate plasma levels of exemestane pre and post treatment.

Outcome measures

Outcome measures
Measure	Treatment (Exemestane) n=39 Participants 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Plasma Levels of Exemestane	2.51 ng/mL Standard Deviation 1.23

Adverse Events

Treatment (Exemestane)

Serious events: 0 serious events

Other events: 38 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment (Exemestane) n=40 participants at risk 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. Exemestane: Given PO Laboratory Biomarker Analysis: Correlative studies Pharmacokinetic Study: Correlative studies Questionnaire Administration: Ancillary studies
Vascular disorders Hot flashes	52.5% 21/40 • 3 months
Vascular disorders Hypertension	42.5% 17/40 • 3 months
Vascular disorders Flushing	2.5% 1/40 • 3 months
Gastrointestinal disorders Diarrhea	25.0% 10/40 • 3 months
Gastrointestinal disorders Abdominal pain	20.0% 8/40 • 3 months
Gastrointestinal disorders Constipation	12.5% 5/40 • 3 months
Gastrointestinal disorders Nausea	10.0% 4/40 • 3 months
Gastrointestinal disorders Vomiting	10.0% 4/40 • 3 months
Gastrointestinal disorders Bloating	5.0% 2/40 • 3 months
Gastrointestinal disorders Dyspepsia	2.5% 1/40 • 3 months
Gastrointestinal disorders Stomach pain	2.5% 1/40 • 3 months
Eye disorders Blurred vision	2.5% 1/40 • 3 months
Eye disorders Eye disorders - Other, specify	2.5% 1/40 • 3 months
Musculoskeletal and connective tissue disorders Myalgia	12.5% 5/40 • 3 months
Musculoskeletal and connective tissue disorders Arthralgia	7.5% 3/40 • 3 months
Musculoskeletal and connective tissue disorders Back pain	7.5% 3/40 • 3 months
Musculoskeletal and connective tissue disorders Pain in extremity	7.5% 3/40 • 3 months
Musculoskeletal and connective tissue disorders Arthritis	5.0% 2/40 • 3 months
Musculoskeletal and connective tissue disorders Chest wall pain	2.5% 1/40 • 3 months
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, specify	2.5% 1/40 • 3 months
Nervous system disorders Headache	20.0% 8/40 • 3 months
Nervous system disorders Dizziness	10.0% 4/40 • 3 months
Nervous system disorders Concentration impairment	2.5% 1/40 • 3 months
Nervous system disorders Dysgeusia	2.5% 1/40 • 3 months
Nervous system disorders Paresthesia	2.5% 1/40 • 3 months
General disorders Fatigue	22.5% 9/40 • 3 months
General disorders Edema limbs	10.0% 4/40 • 3 months
General disorders Chills	2.5% 1/40 • 3 months
General disorders Fever	2.5% 1/40 • 3 months
General disorders Flu like symptoms	2.5% 1/40 • 3 months
Skin and subcutaneous tissue disorders Pruritus	15.0% 6/40 • 3 months
Skin and subcutaneous tissue disorders Hyperhidrosis	10.0% 4/40 • 3 months
Skin and subcutaneous tissue disorders Alopecia	2.5% 1/40 • 3 months
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	2.5% 1/40 • 3 months
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	5.0% 2/40 • 3 months
Respiratory, thoracic and mediastinal disorders Cough	5.0% 2/40 • 3 months
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.0% 2/40 • 3 months
Respiratory, thoracic and mediastinal disorders Sneezing	5.0% 2/40 • 3 months
Psychiatric disorders Anxiety	5.0% 2/40 • 3 months
Psychiatric disorders Insomnia	5.0% 2/40 • 3 months
Investigations Alanine aminotransferase increased	2.5% 1/40 • 3 months
Investigations Blood bilirubin increased	2.5% 1/40 • 3 months
Renal and urinary disorders Urinary frequency	5.0% 2/40 • 3 months
Renal and urinary disorders Urinary urgency	2.5% 1/40 • 3 months
Renal and urinary disorders Urine discoloration	2.5% 1/40 • 3 months

Additional Information

Britt Erickson

University of Minnesota/Masonic Cancer Center

Phone: 612-626-6283

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60