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High Grade Squamous Intraepithelial NeoplasiaPhase 2NCT00503581

Megestrol in Treating Patients With Endometrial Neoplasia or Endometrial Hyperplasia

A Phase 2 study testing Biopsy, Laboratory Biomarker Analysis and Megestrol Acetate in people with High Grade Squamous Intraepithelial Neoplasia and Stage 0 Uterine Corpus Cancer, run by Gynecologic Oncology Group at 50 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This randomized phase II trial is studying how well megestrol works in treating patients with endometrial neoplasia or endometrial hyperplasia. Estrogen can cause the growth of endometrial cancer cells. Hormone therapy using megestrol may fight endometrial cancer by blocking the use of estrogen by the abnormal cells. Read more about this study

Who is it for?
Ages 18 years and older, women only
What is being tested?
Biopsy, Laboratory Biomarker Analysis and Megestrol Acetate
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
50 sitesUnited States
Study dates
Jul 2007to Jul 2012
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

  • Patients must have a diagnosis of atypical endometrial hyperplasia (AEH) or endometrial intraepithelial neoplasia (EIN) diagnosed by dilatation and curettage (D\&C), Novak curettage, Vabra aspirate, or Pipelle endometrial biopsy at the enrolling institution within 12 weeks of enrollment
  • Patients must desire uterine retention for duration of study (18 months or after 3rd biopsy) if they remain EIN negative (-); patients are allowed to attempt pregnancy after their initial post-treatment biopsy without it being a major protocol violation
  • Patients must have a GOG performance status of 0, 1, or 2
  • White blood cell (WBC) >= 3000
  • Platelets >= 100,000
  • Granulocytes >= 1,500
Show all 14Show fewer
  • Creatinine =< 2
  • Bilirubin =< 1.5 x institutional upper limit normal
  • Serum glutamic oxaloacetic transaminase (SGOT) =< 3 x institutional upper limit normal
  • Alkaline phosphatase =< 3 x institutional upper limit normal
  • Patients of child-bearing potential must have a negative serum pregnancy test prior to starting study drug and prior to each biopsy if capable of becoming pregnant (and at the discretion of the referring physician)
  • Patients of childbearing potential must use appropriate non-hormonal contraception while on study medication
  • Patients who have met the pre-entry requirements
  • Patients must have signed an approved informed consent and authorization permitting release of personal health information

Who cannot join

  • Patients with a GOG performance status of 3 or 4
  • Patients with recognized endometrial carcinoma
  • Patients with current or prior history of breast cancer
  • Patients with invasive malignancies, with the exception of nonmelanoma skin cancer who had (or have) any evidence of the other cancer present within the past 5 years or whose previous cancer treatment contraindicates this protocol therapy
  • Patients who have received prior radiotherapy to any portion of the abdominal cavity or pelvis are excluded
  • Patients who have received prior chemotherapy for any abdominal or pelvic tumor are excluded
Show all 9Show fewer
  • Patients who are pregnant or lactating
  • Patients with a history of thrombophlebitis, thromboembolic phenomena, or cerebrovascular disorders within the past 5 years
  • Patients under 18 years of age

Trial locations and contacts

50 sites in 1 country.

50 sites in 1 country

United States50 sites
  • Saint Anthony's Health

    Alton, Illinois, 62002

  • Rush - Copley Medical Center

    Aurora, Illinois, 60504

  • State University of New York Downstate Medical Center

    Brooklyn, New York, 11203

  • Southeast Missouri Hospital

    Cape Girardeau, Missouri, 63701

  • Saint Francis Medical Center

    Cape Girardeau, Missouri, 63703

  • Northwestern University

    Chicago, Illinois, 60611

  • Mount Carmel Health Center West

    Columbus, Ohio, 43222

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Elkhart Clinic

    Elkhart, Indiana, 46514-2098

  • Michiana Hematology Oncology PC-Elkhart

    Elkhart, Indiana, 46514

  • Elkhart General Hospital

    Elkhart, Indiana, 46515

  • Gynecologic Oncology of West Michigan PLLC

    Grand Rapids, Michigan, 49546

  • Hartford Hospital

    Hartford, Connecticut, 06102

  • Saint Francis Hospital and Medical Center

    Hartford, Connecticut, 06105

  • Indiana University/Melvin and Bren Simon Cancer Center

    Indianapolis, Indiana, 46202

  • Joliet Oncology-Hematology Associates Limited

    Joliet, Illinois, 60435

  • Community Howard Regional Health

    Kokomo, Indiana, 46904

  • IU Health La Porte Hospital

    La Porte, Indiana, 46350

  • Women's Cancer Center of Nevada

    Las Vegas, Nevada, 89169

  • Kaiser Permanente Los Angeles Medical Center

    Los Angeles, California, 90027

  • Columbia Saint Mary's Hospital - Ozaukee

    Mequon, Wisconsin, 53097

  • Franciscan Saint Anthony Health-Michigan City

    Michigan City, Indiana, 46360

  • Columbia Saint Mary's Water Tower Medical Commons

    Milwaukee, Wisconsin, 53211

  • Michiana Hematology Oncology PC-Mishawaka

    Mishawaka, Indiana, 46545

  • Saint Joseph Regional Medical Center-Mishawaka

    Mishawaka, Indiana, 46545

  • Good Samaritan Regional Health Center

    Mount Vernon, Illinois, 62864

  • The Hospital of Central Connecticut

    New Britain, Connecticut, 06050

  • Michiana Hematology Oncology PC-Niles

    Niles, Michigan, 49120

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • FirstHealth of the Carolinas-Moore Regional Hosiptal

    Pinehurst, North Carolina, 28374

  • Michiana Hematology Oncology PC-Plymouth

    Plymouth, Indiana, 46563

  • Virginia Commonwealth University/Massey Cancer Center

    Richmond, Virginia, 23298

  • Lakeland Hospital

    Saint Joseph, Michigan, 49085

  • Marie Yeager Cancer Center

    Saint Joseph, Michigan, 49085

  • Memorial University Medical Center

    Savannah, Georgia, 31404

  • Memorial Hospital of South Bend

    South Bend, Indiana, 46601

  • Michiana Hematology Oncology PC-South Bend

    South Bend, Indiana, 46601

  • South Bend Clinic

    South Bend, Indiana, 46617

  • Northern Indiana Cancer Research Consortium CCOP

    South Bend, Indiana, 46628

  • Mercy Hospital Springfield

    Springfield, Missouri, 65804

  • Ozark Health Ventures LLC-Cancer Research for The Ozarks Springfield

    Springfield, Missouri, 65804

  • Saint Louis Cancer and Breast Institute-South City

    St Louis, Missouri, 63109

  • Saint John's Mercy Medical Center

    St Louis, Missouri, 63141

  • Saint Louis-Cape Girardeau CCOP

    St Louis, Missouri, 63141

  • Olive View-University of California Los Angeles Medical Center

    Sylmar, California, 91342

  • Montefiore Medical Center-Einstein Campus

    The Bronx, New York, 10461

  • Cancer Care Associates-Midtown

    Tulsa, Oklahoma, 74104

  • Tulsa Cancer Institute

    Tulsa, Oklahoma, 74146

  • Carle Clinic-Urbana Main

    Urbana, Illinois, 61801

  • Michiana Hematology Oncology-PC Westville

    Westville, Indiana, 46391

Other trials in United States

About this study

Official title: A Randomized Phase II Evaluation of Continuous Progestin Therapy vs. Sequential Progestin Therapy in the Treatment of Endometrial Intraepithelial Neoplasia (EIN) From a Referred Cohort of Atypical Endometrial Hyperplasia (AEH) or EIN Patients That Desire Uterine Preservation

This randomized phase II trial is studying how well megestrol works in treating patients with endometrial neoplasia or endometrial hyperplasia. Estrogen can cause the growth of endometrial cancer cells. Hormone therapy using megestrol may fight endometrial cancer by blocking the use of estrogen by the abnormal cells.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the frequency of complete remission by a central pathology review panel in diagnosed endometrial intraepithelial neoplasia (EIN) patients treated for 24 weeks with oral continuous versus interrupted progestin therapy.

SECONDARY OBJECTIVES:

  1. To evaluate whether quality of life is superior in patients who take continuous megestrol versus sequential megestrol by evaluating mood, concerns about weight changes and bleeding.

TERTIARY:

  1. To assess the expression levels of PTEN using immunohistochemistry and to explore the association of PTEN expression levels with patient response to treatment.

II. To assess the expression levels of the hormone receptors ER and PR using immunohistochemistry and to explore the association of ER/PR expression levels with patient response to treatment.

III. To assess histomorphometry and karyometry characteristics of the pre-treatment biopsy in this patient population.

IV. To identify patterns of protein and glycoprotein expression associated with invasive cancer in serum specimens obtained from patients with a diagnosis of atypical endometrial hyperplasia (AEH) or EIN.

  1. To assess differences in plasma concentrations of megestrol acetate HPLC in this patient population.

VI. To assess patient compliance to their treatment regimen using HPLC.

OUTLINE: Patients are stratified according to the collection method of the initial/intake biopsy (dilatation and curettage vs all other methods). Patients are randomized to 1 of the following treatment regimens:

REGIMEN 1: Patients receive oral megestrol twice daily every day for 24 weeks. Approximately twelve weeks after treatment starts, clinical blood tests are obtained and research serum and plasma collected. Twenty-four weeks constitutes one course of treatment and a pill count is performed during the 12-week f/u visit and at the completion of the treatment course to determine compliance. After progestin therapy the patient has an induced-withdrawal bleed. Patients in this arm undergo a re-evaluation biopsy and hysterectomy a minimum of two weeks and a maximum of eight weeks after completing the megestrol treatment.

REGIMEN 2: Patients receive oral megestrol twice daily for two weeks continuously followed by no treatment for two weeks. This course is repeated for a total of 24 weeks. Approximately twelve weeks after treatment starts, clinical blood tests are obtained and research serum and plasma collected. Twenty-four weeks constitutes one course of treatment and a pill count is performed during the 12-week f/u visit and at the completion of the treatment course to determine compliance. After progestin therapy the patient has an induced-withdrawal bleed. Patients in this arm undergo a re-evaluation biopsy and hysterectomy a minimum of two weeks and a maximum of eight weeks after the megestrol treatment.

REGIMEN 3: (Closed as of 6/3/2010) Patients do not receive megestrol. At the discretion of the managing physician, patients undergo the re-evaluation biopsy and hysterectomy anytime between 2-20 weeks after enrollment and randomization. Patients undergo biopsy and blood sample collection periodically for immunological and pharmacodynamic studies. Samples are analyzed for presence or absence of myoinvasion or deep myoinvasion in hysterectomy specimens, hormone receptivity status, and to compare PTEN status against treatment via karyometry or morphometry, expression of VEGF and tenascin-C (TN-C) via ELISA, presence of TN-C fragmentation via western immunoblots, additional biomarkers via proteomic analysis, protein and glycoprotein expression patterns via electrophoresis and image analysis, and plasma megestrol concentrations via high-performance liquid chromatography (HPLC). Patients complete the Hospital Anxiety and Depression Scale (HADS) and two items on bleeding and weight gain at baseline and periodically during study. A Treatment Decision Assessment is completed at baseline, and for patients withdrawing from the study, a Study Withdraw Assessment is also completed. There will be no additional follow-up on this study after the patient's hysterectomy.

Being tested
Biopsy, Laboratory Biomarker Analysis, Megestrol Acetate, Pharmacological Study, Quality-of-Life Assessment
Medical subject headings
Squamous Intraepithelial Lesions

What participants receive

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

  • BiopsyProcedure or surgery

    Also called Bx

    Undergo biopsy

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • 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 orally

  • Pharmacological StudyOther

    Correlative studies

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

  • Therapeutic Conventional SurgeryProcedure or surgery

    undergo hysterectomy

Study groups in NCT00503581
Study groupRoleWhat they receive
Regimen 1 (megestrol acetate, surgery)ExperimentalPatients receive oral megestrol twice daily every day for 24 weeks. Approximately twelve weeks after treatment starts, clinical blood tests are obtained and research serum and plasma collected. Twenty-four weeks constitutes one course of treatment and a pill count is performed during the 12-week f/u visit and at the completion of the treatment course to determine compliance. After progestin therapy the patient has an induced-withdrawal bleed. Patients in this arm undergo a re-evaluation biopsy and hysterectomy a minimum of two weeks and a maximum of eight weeks after completing the megestrol treatment.
Receives: Procedure: Biopsy; Other: Laboratory Biomarker Analysis; Drug: Megestrol Acetate; Other: Pharmacological Study; Other: Quality-of-Life Assessment; Procedure: Therapeutic Conventional Surgery
Regimen 2 (megestrol acetate, surgery)ExperimentalPatients receive oral megestrol twice daily for two weeks continuously followed by no treatment for two weeks. This course is repeated for a total of 24 weeks. Approximately twelve weeks after treatment starts, clinical blood tests are obtained and research serum and plasma collected. Twenty-four weeks constitutes one course of treatment and a pill count is performed during the 12-week f/u visit and at the completion of the treatment course to determine compliance. After progestin therapy the patient has an induced-withdrawal bleed. Patients in this arm undergo a re-evaluation biopsy and hysterectomy a minimum of two weeks and a maximum of eight weeks after the megestrol treatment.
Receives: Procedure: Biopsy; Other: Laboratory Biomarker Analysis; Drug: Megestrol Acetate; Other: Pharmacological Study; Other: Quality-of-Life Assessment; Procedure: Therapeutic Conventional Surgery
Regimen 3 (surgery/biopsy)Active comparator(Closed as of 6/3/2010) Patients do not receive megestrol. At the discretion of the managing physician, patients undergo the re-evaluation biopsy and hysterectomy anytime between 2-20 weeks after enrollment and randomization.
Receives: Procedure: Biopsy; Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Other: Quality-of-Life Assessment; Procedure: Therapeutic Conventional Surgery

What is being measured

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

  1. Number of Patients Who Experience a Response as Determined by a Central Blinded Review of the Three Post Treatment Endometrial

    Treated and eligible participants. This study had 0 participants that completed treatment, therefore no analysis was done. Study closed prior to completion. Central blinded review was not performed for any participants in the study.

    Measured: Up to 12 months after completion of treatment

Secondary outcomes (1)
  1. Change in Quality of Life (QOL) Evaluated Using the Hospital Anxiety and Depression Scale (HADS) and the Two Items on Bleeding and Weight Gain

    Eligible and Treated patients. This study had 0 participants that completed study.

    Measured: Baseline to up to 12 months

Timeline

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

  1. First posted19 Jul 2007 (estimated)
  2. Study startJul 2007
  3. Primary completionJul 2012

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
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00503581
Sponsor's protocol ID
GOG-0224
Other IDs
NCI-2009-00594, GOG-0224, CDR0000555427, GOG-0224, GOG-0224, GOG-0224, U10CA101165
Status last verified
Oct 2020
First submitted
17 Jul 2007
Last updated
3 Nov 2020

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Biopsy, Laboratory Biomarker Analysis and Megestrol Acetate in people with High Grade Squamous Intraepithelial Neoplasia and Stage 0 Uterine Corpus Cancer, run by Gynecologic Oncology Group at 50 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 14 inclusion and 9 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: stopped early, as of the sponsor's last update on 3 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 50 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 14 Oct 2020). 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?

Number of Patients Who Experience a Response as Determined by a Central Blinded Review of the Three Post Treatment Endometrial (time frame: Up to 12 months after completion of treatment). 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 Nov 2020.

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