A Phase 2 interventional study of Exemestane and Laboratory Biomarker Analysis in Atypical Hyperplasia, Endometrial Atypical Hyperplasia/Endometrioid Intraepithelial Neoplasia and Endometrial Carcinoma, sponsored by National Cancer Institute (NCI). Completed at 3 sites in United States. Open to female participants aged 45 Years and older. Per ClinicalTrials.gov, last updated 2025-08-29.
Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Prevention
Among 29 studies in Endometrial Hyperplasia with a site in United States — 10 open to participants now, 16 under way, none of them phase 3. Endometrial Hyperplasia · United States.
Among the 29 studies in Endometrial Hyperplasia with a site in United States, this one is the 14th largest by enrollment of the 23 interventional ones, is the only one that started in 2017, is one of 13 in phase 2, and was the 12th registered.
110 studies on the registry are indexed under Endometrial Hyperplasia; 38 are open to participants now.
This study's enrollment of 40 is below the median of 80 across 71 interventional studies indexed under Endometrial Hyperplasia.
Browse Endometrial Hyperplasia studies →10 of the 29 studies in Endometrial Hyperplasia with a site in United States are open now, in 1 country. Each count lists the studies.
Of the 21 Endometrial Hyperplasia studies recruiting now, 9 (43%) set no upper age limit and 11 (52%) stop at 75 or younger; 7 take children under 18; 3 take healthy volunteers.
This study: 45 Years and older · female · no healthy volunteers.
The full table on the hub →29 studies; the count open now beside each. This study's row is marked.
First in 2002; this study’s year is marked. 1 started in 2017.
Median enrollment 48 across the 23 interventional studies, 1,928 across the 6 observational. Of the 11 that completed or were terminated, 3 (27%) have results posted.
3 of 29 have an industry lead sponsor. The 10 most frequent:
National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.
National Cancer Institute (NCI) has 3 studies filed under Endometrial Hyperplasia — fewer than ten, so there is no comparison to draw.
1 of the studies in Endometrial Hyperplasia with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05051722Mayo ClinicLeveraging Methylated DNA Markers (MDMs) in the Detection of Endometrial Cancer, Ovarian Cancer, and Cervical Cancer | 2027-07-30 |
Among the 29 studies in Endometrial Hyperplasia with a site in United States, from their registry updates:
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.
PRIMARY OBJECTIVE:
I. 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:
I. 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.
V. 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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
confirmed with one the following criteria:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Given PO
Also known as: Aromasin, FCE-24304
Correlative studies
Correlative studies
Also known as: PHARMACOKINETIC, PK Study
Ancillary studies
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.
Time frame: Baseline up to 2 months
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.
Time frame: Baseline up to 2 months
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.
Time frame: Baseline up to 2 months
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.
Time frame: Up to 2 months
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.
Time frame: Up to 2 months
Deoxyribonucleic Acid (DNA) Mutational Analysis
Will be analyzed by next generation sequencing.
Time frame: Up to 2 months
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.
Time frame: Up to 2 months
Ki-67 Expression With Historic Controls
Will compare Ki-67 expression between participants samples and historically matched samples.
Time frame: Up to 2 months
Plasma Levels of Exemestane
Will evaluate plasma levels of exemestane pre and post treatment.
Time frame: Up to 2 months
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.
| Milestone | Treatment (Exemestane) |
|---|---|
| Started | 40 |
| Completed | 40 |
| Not completed | 0 |
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.
| percent (represented by mean) of cells | Treatment (Exemestane) |
|---|---|
| Change in Tumor Proliferation | -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.
| pg/mL | Treatment (Exemestane) |
|---|---|
| Changes in Circulating Serum Estradiol | 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.
| ng/mL | Treatment (Exemestane) |
|---|---|
| Changes in Circulating Serum Progesterone | 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.
| Percent of participants | Treatment (Exemestane) |
|---|---|
| No visible lesion | 2.5 |
| CAH/EIN | 20 |
| Grade I (Low) EC | 57.5 |
| Grade II (Low) EC | 10 |
| Grade III (High) EC | 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.
| % positive | Treatment (Exemestane) |
|---|---|
| Cleaved capsase | -0.17 ± 0.18 |
| Cyclin D1 | -0.07 ± 0.22 |
| pAKT | -0.12 ± 0.27 |
| IGF-1R | -2.50 ± 3.00 |
Will be analyzed by next generation sequencing.
| percentage of cells | Treatment (Exemestane) |
|---|---|
| Deoxyribonucleic Acid (DNA) Mutational Analysis | NA |
Perform pre- and post-treatment proteomic analysis of vaginal proteins from tampon recovery to identify biomarkers that may predict response to exemestane treatment.
| number of proteins | Treatment (Exemestane) |
|---|---|
| Protein Markers | 85 |
Will compare Ki-67 expression between participants samples and historically matched samples.
| percentage of cells | Treatment (Exemestane) |
|---|---|
| Ki-67 Expression With Historic Controls | -0.07 ± 0.10 |
Will evaluate plasma levels of exemestane pre and post treatment.
| ng/mL | Treatment (Exemestane) |
|---|---|
| Plasma Levels of Exemestane | 2.51 ± 1.23 |
Collected over 3 months. Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (Exemestane) | 0/40 (0%) | 0/40 (0%) | 38/40 (95%) |
| Event | Treatment (Exemestane) |
|---|---|
| Hot flashesVascular disorders | 21/40 |
| HypertensionVascular disorders | 17/40 |
| DiarrheaGastrointestinal disorders | 10/40 |
| FatigueGeneral disorders | 9/40 |
| Abdominal painGastrointestinal disorders | 8/40 |
| HeadacheNervous system disorders | 8/40 |
| PruritusSkin and subcutaneous tissue disorders | 6/40 |
| ConstipationGastrointestinal disorders | 5/40 |
| MyalgiaMusculoskeletal and connective tissue disorders | 5/40 |
| NauseaGastrointestinal disorders | 4/40 |
| Age, Customized(Participants) | Treatment (Exemestane) |
|---|---|
| 50-59 years | 13 |
| 60-69 years | 18 |
| 70-79 years | 7 |
| Unknown | 2 |
| Sex: Female, Male(Participants) | Treatment (Exemestane) |
|---|---|
| Female | 40 |
| Male | 0 |
| Ethnicity (NIH/OMB)(Participants) | Treatment (Exemestane) |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 38 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Treatment (Exemestane) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 38 |
| More than one race | 1 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Treatment (Exemestane) |
|---|---|
| United States | 40 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Nov 2017 to primary completion in Dec 2023, 72 months.
Results were first posted in Jun 2025, 1 year 5 months after primary completion. A results delay was requested in Dec 2024.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Exemestane
National Cancer Institute (NCI) →