Atypical Endometrial HyperplasiaPhase 2NCT03671811
Megestrol Acetate With or Without Pterostilbene in Treating Patients With Endometrial Cancer Undergoing Hysterectomy
A Phase 2 study testing Megestrol Acetate and Pterostilbene in people with Atypical Endometrial Hyperplasia and Endometrial Carcinoma, run by City of Hope Medical Center at 1 site in United States.
Trial summary
This phase II trial studies how well megestrol acetate with or without pterostilbene works in treating patients with endometrial cancer undergoing hysterectomy. Drugs used in chemotherapy, such as megestrol acetate, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them… Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Atypical Endometrial Hyperplasia, Endometrial Carcinoma
- What is being tested?
- Megestrol Acetate and Pterostilbene
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Jan 2019to 28 Feb 2027
- 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 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented informed consent of the participant and/or legally authorized representative
- Willing to undergo an intraoperative biopsy/or standard of care tissue collection during surgery, following completion of treatment with MA +/- PTE
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Histologically confirmed EC or complex atypical hyperplasia of the endometrium
- Candidate for a total hysterectomy with or without bilateral salpingo-oophorectomy
- About to initiate preoperative window period, with planned hysterectomy scheduled
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- Platelets >= 100,000/mm^3
- NOTE: Platelet transfusions are not permitted within 14 days of platelet assessment unless cytopenia is secondary to disease involvement
- Total bilirubin =< 1.5 X upper limit of normal (ULN)
- Aspartate aminotransferase (AST) =< 1.5 x ULN
- Alanine aminotransferase (ALT) =< 1.5 x ULN
- Creatinine clearance of >= 60 mL/min per 24 hour urine test or the Cockcroft-Gault formula
- Women of childbearing potential: negative urine or serum pregnancy test in premenopausal women. Postmenopausal women do not need to undergo a pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
Who cannot join
- Pterostilbene supplements within 30 days prior to day 1 of protocol therapy
- Any of the following phytochemical-based supplements within 30 days prior to day 1 of protocol therapy: resveratrol, genistein, and quercetin
- Chemotherapy for EC
- Allergic reaction/hypersensitivity to similar agents, excipients
- Unstable cardiac disease as defined by one of the following:
- Cardiac events such as myocardial infarction (MI) within the past 6 months
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- NYHA (New York Heart Association) heart failure class III-IV
- Uncontrolled atrial fibrillation or hypertensive emergency/urgency (defined as systolic blood pressure >= 180 mmHg and/or diastolic blood pressure >= 120 mmHg)
- Active or history of recent thromboembolism or stroke, within the past 6 months
- Cushing's syndrome
- Acute infection requiring systemic (intravenous) treatment
- Known history of human immunodeficiency virus (HIV) infection
- Known active hepatitis B or C infection
- Inability to swallow tablets/capsules
- Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns or compliance with clinical study procedures, e.g., infection/inflammation, intestinal obstruction, unable to swallow medication, social/ psychological issues, etc
- Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: Open-Label Randomized Phase II Trial of Megestrol Acetate With or Without Pterostilbene in Patients With Endometrial Cancer Scheduled for Hysterectomy
This phase II trial studies how well megestrol acetate with or without pterostilbene works in treating patients with endometrial cancer undergoing hysterectomy. Drugs used in chemotherapy, such as megestrol acetate, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Pterostilbene is an antioxidant found in blueberries or grapes, and it has been shown to be effective in killing tumor cells and reducing cancer burden. It is not yet known whether giving megestrol acetate with or without pterostilbene may work better in treating patients with endometrial cancer.
Read the detailed description
PRIMARY OBJECTIVE:
- Determine the effect of megestrol acetate (MA) plus pterostilbene (PTE) versus MA alone on tumor proliferation (Ki-67) during the preoperative window in patients with endometrial cancer (EC) who are scheduled for hysterectomy.
EXPLORATORY OBJECTIVES:
- Determine the effect of MA plus PTE versus MA alone on histologic response during the preoperative window in patients with EC or endometrial complex atypical hyperplasia who are scheduled for hysterectomy.
II. Explore biological characteristics of tumors to determine potential biomarkers which could select for treatment eligibility in future studies.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive pterostilbene orally (PO) twice daily (BID) and megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity.
ARM II: Patients receive megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity.
After completion of study treatment, patients are followed up at 6 weeks.
- Being tested
- Megestrol Acetate, Pterostilbene
- 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.
Megestrol AcetateDrug
Also called 17-Hydroxy-6-methylpregna-4,6-diene-3,20-dione acetate, 17.alpha.-Acetoxy-6-methylpregna-4,6-diene-3,20-dione, 6-Dehydro-6-methyl-17.alpha.-acetoxyprogesterone, 6-Methyl-6-dehydro-17.alpha.-acetoxyprogesterone
Given PO
PterostilbeneBiological
Also called 3'',5''-Dimethoxy-4-stilbenol, 3,5-Dimethoxy-4''-hydroxystilbene, Trans-3,5-dimethoxy-4-hydroxystilbene
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Arm I (pterostilbene, megestrol acetate) | Experimental | Patients receive 100mg pterostilbene BID and 80mg megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity. Receives: Drug: Megestrol Acetate; Biological: Pterostilbene |
| Arm II (megestrol acetate) | Experimental | Patients receive megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity. Receives: Drug: Megestrol Acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tumor Ki-67 Proliferation Index
Ki-67 represents a specific nuclear marker for cell proliferation that is measured by staining with specific antibodies by immunohistochemical (IHC) staining. The Ki-67 proliferation index is defined as percent tumor cells staining positive, and measured on a continuous scale of 0-100%, with higher values indicating higher proliferation. Ki-67 hotspot values were assessed at two time-points during this study, prior to treatment with megace +/- pterostilbene (pre-treatment), and following completion of treatment (post-treatment). Descriptive statistics were used to compare treatment-associated percent change in Ki-67 proliferation index between the 2 study arms, using a 1-sided test with significance at p < 0.05.
Measured: Pre- and post-treatment up to 6 weeks
Secondary outcomes (6)
Histologic Response of Gland Cellularity
These histologic changes represent measures of growth and apoptosis, and will be separately scored in the pretreatment endometrial sample and the section of tumor from the hysterectomy (post-treatment sample). Gland cellularity will be assessed by counting the number of cells in one quarter of a high-power field (HPF) (average of 3 fields). The number of patients with an improvement in gland cellularity were compared between study arms.
Measured: Up to 6 weeks
Histologic Response of Mitotic Index
These histologic changes represent measures of growth and apoptosis, and will be separately scored in the pretreatment endometrial sample and the section of tumor from the hysterectomy (post-treatment sample). The mitotic index will be calculated as the number of mitoses per HPF (average of 3 fields). The number of patients with an improvement in mitotic index were compared between study arms.
Measured: Up to 6 weeks
Histologic Response of Metaplasia
These histologic changes represent measures of growth and apoptosis, and will be separately scored in the pretreatment endometrial sample and the section of tumor from the hysterectomy (post-treatment sample). The percentages of tumor that display squamous or mucinous metaplasia will be estimated as percentages, with < 10% considered negative and >= 10% as positive. The number of patients with an improvement in metaplasia were compared between study arms.
Measured: Up to 6 weeks
Histologic Response of Eosinophilic Metaplasia
These histologic changes represent measures of growth and apoptosis, and will be separately scored in the pretreatment endometrial sample and the section of tumor from the hysterectomy (post-treatment sample). The number of patients with an improvement in eosinophilic metaplasia were compared between study arms.
Measured: Up to 6 weeks
Immunohistochemical Expression of Bcl-2 to Assess Tumor Growth and Apoptosis
Immunohistochemistry stains with antibodies directed against Bcl-2 will be performed on pre- and post-treatment endometrial samples. Samples will be scored on a continuous scale (0-100%) using the product of the intensity of cytoplasmic staining, and the proportion of cells staining based on the distribution of staining. Descriptive statistics were used to compare the percent change in scores from pre-treatment to post-treatment between the 2 study arms.
Measured: Pre- and post-treatment up to 6 weeks
Immunohistochemical Expression of Casp3 to Assess Tumor Growth and Apoptosis
Immunohistochemistry stains with antibodies directed against Casp3 to assess apoptosis will be performed on pre- and post-treatment endometrial samples. Samples will be scored by counting the number of positive staining nuclei per HPF. Descriptive statistics were used to compare the percent change in scores from pre-treatment to post-treatment between the 2 study arms.
Measured: Pre- and post-treatment up to 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2018
- Study start21 Jan 2019
- Primary completion12 Dec 2022
- Study completion28 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Atypical Endometrial HyperplasiaStudies of the same condition, open and under way.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Thanh H Dellinger, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03671811
- Sponsor's protocol ID
- 17327
- Other IDs
- NCI-2018-01555, 17327, P30CA033572
- Status last verified
- May 2026
- First submitted
- 27 Jul 2018
- Last updated
- 3 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Randomized Phase II Trial of Megestrol Acetate With or Without Pterostilbene in Patients With Endometrial Cancer Scheduled for Hysterectomy
- ClinicalTrials.gov ID
- NCT03671811
- Acronym / sponsor ID
- 17327
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 44 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 21 Jan 2019 (actual)
- Primary completion
- 12 Dec 2022 (actual)
- Study completion
- 28 Feb 2027 (estimated)
- First posted
- 14 Sep 2018 (actual)
- Results
- Posted 6 Jan 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well megestrol acetate with or without pterostilbene works in treating patients with endometrial cancer undergoing hysterectomy. Drugs used in chemotherapy, such as megestrol acetate, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Pterostilbene is an antioxidant found in blueberries or grapes, and it has been shown to be effective in killing tumor cells and reducing cancer burden. It is not yet known whether giving megestrol acetate with or without pterostilbene may work better in treating patients with endometrial cancer.
Detailed description
PRIMARY OBJECTIVE:
- Determine the effect of megestrol acetate (MA) plus pterostilbene (PTE) versus MA alone on tumor proliferation (Ki-67) during the preoperative window in patients with endometrial cancer (EC) who are scheduled for hysterectomy.
EXPLORATORY OBJECTIVES:
- Determine the effect of MA plus PTE versus MA alone on histologic response during the preoperative window in patients with EC or endometrial complex atypical hyperplasia who are scheduled for hysterectomy.
II. Explore biological characteristics of tumors to determine potential biomarkers which could select for treatment eligibility in future studies.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive pterostilbene orally (PO) twice daily (BID) and megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity.
ARM II: Patients receive megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity.
After completion of study treatment, patients are followed up at 6 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (pterostilbene, megestrol acetate) | Experimental | Drug: Megestrol Acetate, Biological: PterostilbenePatients receive 100mg pterostilbene BID and 80mg megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity. |
| Arm II (megestrol acetate) | Experimental | Drug: Megestrol AcetatePatients receive megestrol acetate PO BID for 3 weeks in the absence of disease progression or unaccepted toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Documented informed consent of the participant and/or legally authorized representative
- Willing to undergo an intraoperative biopsy/or standard of care tissue collection during surgery, following completion of treatment with MA +/- PTE
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Histologically confirmed EC or complex atypical hyperplasia of the endometrium
- Candidate for a total hysterectomy with or without bilateral salpingo-oophorectomy
Key exclusion criteria (16 listed)
- Pterostilbene supplements within 30 days prior to day 1 of protocol therapy
- Any of the following phytochemical-based supplements within 30 days prior to day 1 of protocol therapy: resveratrol, genistein, and quercetin
- Chemotherapy for EC
- Allergic reaction/hypersensitivity to similar agents, excipients
- Unstable cardiac disease as defined by one of the following:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Megestrol AcetateAlso: 17-Hydroxy-6-methylpregna-4,6-diene-3,20-dione acetate, 17.alpha.-Acetoxy-6-methylpregna-4,6-diene-3,20-dione, 6-Dehydro-6-methyl-17.alpha.-acetoxyprogesterone, 6-Methyl-6-dehydro-17.alpha.-acetoxyprogesteroneGiven PO | Drug | Arm I (pterostilbene, megestrol acetate), Arm II (megestrol acetate) |
| PterostilbeneAlso: 3'',5''-Dimethoxy-4-stilbenol, 3,5-Dimethoxy-4''-hydroxystilbene, Trans-3,5-dimethoxy-4-hydroxystilbeneGiven PO | Biological | Arm I (pterostilbene, megestrol acetate) |
Primary outcome
Tumor Ki-67 Proliferation Index
Time frame: Pre- and post-treatment up to 6 weeks
Ki-67 represents a specific nuclear marker for cell proliferation that is measured by staining with specific antibodies by immunohistochemical (IHC) staining. The Ki-67 proliferation index is defined as percent tumor cells staining positive, and measured on a continuous scale of 0-100%, with higher values indicating higher proliferation. Ki-67 hotspot values were assessed at two time-points during this study, prior to treatment with megace +/- pterostilbene (pre-treatment), and following completion of treatment (post-treatment). Descriptive statistics were used to compare treatment-associated percent change in Ki-67 proliferation index between the 2 study arms, using a 1-sided test with significance at p < 0.05.
6 secondary outcomes
Histologic Response of Gland Cellularity
Time frame: Up to 6 weeks
Histologic Response of Mitotic Index
Time frame: Up to 6 weeks
Histologic Response of Metaplasia
Time frame: Up to 6 weeks
Histologic Response of Eosinophilic Metaplasia
Time frame: Up to 6 weeks
Immunohistochemical Expression of Bcl-2 to Assess Tumor Growth and Apoptosis
Time frame: Pre- and post-treatment up to 6 weeks
Immunohistochemical Expression of Casp3 to Assess Tumor Growth and Apoptosis
Time frame: Pre- and post-treatment up to 6 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thanh H Dellinger principal investigator
City of Hope Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Megestrol Acetate and Pterostilbene in people with Atypical Endometrial Hyperplasia and Endometrial Carcinoma, run by City of Hope Medical Center at 1 site 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 13 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 6 Jan 2026). 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?
Tumor Ki-67 Proliferation Index (time frame: Pre- and post-treatment up to 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jun 2026.