An Endometrial Cancer Chemoprevention Study of Metformin
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ClinicalTrials.gov Identifier: NCT01697566 |
Recruitment Status :
Active, not recruiting
First Posted : October 2, 2012
Last Update Posted : September 16, 2022
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The goal of this clinical research study is to learn about the effects of metformin and/or a program called "lifestyle intervention" on the endometrium (inner lining of the uterus) in post-menopausal women who are also obese (both are risk factors for endometrial cancer). Researchers also want to learn how insulin levels will be affected by metformin, diet and/or exercise.
Metformin is designed to treat both diabetes and insulin resistance. Insulin resistance is a condition in which the body makes insulin but does not use it properly. It is often referred to as "pre-diabetes". Many people with insulin resistance have high levels of both sugar and insulin in their blood at the same time, which have been reported in patients with endometrial cancer.
In this study, metformin will be compared to a placebo. A placebo is not a drug. It looks like the study drug but is not designed to treat any disease or illness. It is designed to be compared with a study drug to learn if the study drug has any real effect.
Lifestyle intervention is made up of a series of in-person sessions where you meet with a coach to discuss strategies for losing weight and ways to increase physical activity. It also consists of materials designed to help you lose weight and will offer opportunities for supervised exercise.
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Gynecology | Drug: Metformin Other: Placebo Procedure: Endometrial Biopsy Behavioral: Lifestyle Intervention Behavioral: Questionnaires | Phase 3 |

Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 100 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | An Endometrial Cancer Chemoprevention Study of Metformin Versus No Treatment in Women With a Body Mass Index (BMI) >/= 30 kg.m2 and Hyperinsulinemia |
Actual Study Start Date : | May 2013 |
Estimated Primary Completion Date : | April 30, 2023 |
Estimated Study Completion Date : | April 30, 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Metformin
Participants gradually increase the dose of metformin by mouth as listed below: Week 1: One capsule each day Week 2: One capsule 2 times each day Week 3: One capsule 3 times each day Week 4: Two capsules 2 times each day After week 4, participant continues to take 2 capsules of metformin 2 times each day. Each capsule is 425 mg. |
Drug: Metformin
Participants gradually increase the dose of metformin by mouth as listed below: Week 1: One capsule each day Week 2: One capsule 2 times each day Week 3: One capsule 3 times each day Week 4: Two capsules 2 times each day After week 4, participant continues to take 2 capsules of metformin 2 times each day. Each capsule is 425 mg. Procedure: Endometrial Biopsy Endometrial biopsies obtained at baseline and after 4 months. Behavioral: Questionnaires Completion of questionnaires about diet and exercise at baseline, during treatment and at end of study. Questionnaires take about 30-40 minutes to complete.
Other Name: surveys |
Experimental: Placebo + Lifestyle Intervention
Placebo taken by mouth twice daily for 4, 30 day cycles. Lifestyle intervention consists of 16 in-person sessions offered over a 4 month period.
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Other: Placebo
Placebo taken by mouth twice daily for 4, 30 day cycles.
Other Name: sugar pill Procedure: Endometrial Biopsy Endometrial biopsies obtained at baseline and after 4 months. Behavioral: Lifestyle Intervention Lifestyle intervention consists of 16 in-person sessions offered over a 4 month period. Behavioral: Questionnaires Completion of questionnaires about diet and exercise at baseline, during treatment and at end of study. Questionnaires take about 30-40 minutes to complete.
Other Name: surveys |
Experimental: Metformin + Lifestyle Intervention
Participants gradually increase the dose of metformin by mouth as listed below: Week 1: One capsule each day Week 2: One capsule 2 times each day Week 3: One capsule 3 times each day Week 4: Two capsules 2 times each day After week 4, participant continues to take 2 capsules of metformin 2 times each day. Each capsule is 425 mg. Lifestyle intervention consists of 16 in-person sessions offered over a 4 month period. |
Drug: Metformin
Participants gradually increase the dose of metformin by mouth as listed below: Week 1: One capsule each day Week 2: One capsule 2 times each day Week 3: One capsule 3 times each day Week 4: Two capsules 2 times each day After week 4, participant continues to take 2 capsules of metformin 2 times each day. Each capsule is 425 mg. Procedure: Endometrial Biopsy Endometrial biopsies obtained at baseline and after 4 months. Behavioral: Lifestyle Intervention Lifestyle intervention consists of 16 in-person sessions offered over a 4 month period. Behavioral: Questionnaires Completion of questionnaires about diet and exercise at baseline, during treatment and at end of study. Questionnaires take about 30-40 minutes to complete.
Other Name: surveys |
Placebo Comparator: Placebo
Placebo taken by mouth twice daily for 4, 30 day cycles.
|
Other: Placebo
Placebo taken by mouth twice daily for 4, 30 day cycles.
Other Name: sugar pill Procedure: Endometrial Biopsy Endometrial biopsies obtained at baseline and after 4 months. Behavioral: Questionnaires Completion of questionnaires about diet and exercise at baseline, during treatment and at end of study. Questionnaires take about 30-40 minutes to complete.
Other Name: surveys |
- Effect of Metformin and/or Lifestyle Intervention on Biomarkers. [ Time Frame: 4 months ]Differences in Ki-67 and other biomarkers analyzed using a 2x2 ANOVA. Other biomarkers examined include a) a panel of genes relevant to estrogen dependent endometrial proliferation, hyperplasia and cancer using Q-PCR, b) biomarkers specific to the effect of metformin treatment (phospho-AMPKa, phospho-ACC, phospho-mTOR and phospho-S6 ribosomal protein and c) potential biomarkers of modulation of insulin sensitivity on the human endometrial proteome using reverse phase protein arrays (RPPA).

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Ages Eligible for Study: | 50 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | Female |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
- For this study, only women will be enrolled.
- Body Mass Index (BMI) > or = 30 kg/m2
- Not frankly diabetic, as measured by a fasting blood glucose </=126 mg/dL.
- Demonstrate hyperinsulinemia with a QUICK I value </= 0.357.
- Age >/= 50 and </= 65
- Zubrod Performance Scale 0-1
- Hemoglobin >/= 10 g/dl
- TSH 0.27 - 4.20 µlU/mL
- Menopause as defined as no menses for 1 year and/or FSH >/= 25.8 mIU/ml
- Must be able to read, write, and speak English.
- Must have a Primary Care Provider (PCP).
Exclusion Criteria:
- Prior hysterectomy or endometrial ablation
- ALT >/r = 2.0x Upper Limit of Normal (ULN)
- Serum creatinine > /= 1.4 mg/dl
- Triglycerides (fasting) >/ = 400
- Known inability to participate in the ongoing appointments for the four months of the study and scheduled follow-up tests.
- Significant medical or psychiatric history which would make the participant a poor protocol candidate, in the opinion of the principal investigator, for any aspect of study participation including metformin, unsupervised exercise program or dietary behavior change.
- Participant reported history of congestive heart failure
- Prior treatment with Metformin
- Currently being treated for diabetes or meeting criteria for new diagnosis of diabetes.
- Known allergy to Metformin or other biguanide (Proguanil).
- Use of Aromatase Inhibitors, GNRH-agonists i.e.Lupron, Zoladex within the last 6 months
- Use of SERMS (selective estrogen receptor modulators) in the past 6 months, including Tamoxifen and Raloxifene
- Hormone replacement therapy within the last 6 months
- Women who have been treated with chemotherapy for prior malignant disease or currently have an untreated malignancy other than non-melanoma skin cancer
- Patients who have had prior radiation to the pelvis

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01697566
United States, Texas | |
University of Texas MD Anderson Cancer Center | |
Houston, Texas, United States, 77030 |
Principal Investigator: | Karen H. Lu, MD | M.D. Anderson Cancer Center |
Responsible Party: | M.D. Anderson Cancer Center |
ClinicalTrials.gov Identifier: | NCT01697566 |
Other Study ID Numbers: |
2011-0739 5P50CA098258 ( U.S. NIH Grant/Contract ) NCI-2013-00834 ( Registry Identifier: NCI CTRP ) |
First Posted: | October 2, 2012 Key Record Dates |
Last Update Posted: | September 16, 2022 |
Last Verified: | September 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Gynecology Endometrial Cancer Chemoprevention Obese Body Mass Index (BMI) >/= 35 kg.m2 Post-menopausal Hyperinsulinemia |
Metformin Placebo Sugar pill Lifestyle intervention Diet Exercise |
Endometrial Neoplasms Uterine Neoplasms Genital Neoplasms, Female Urogenital Neoplasms Neoplasms by Site |
Neoplasms Uterine Diseases Metformin Hypoglycemic Agents Physiological Effects of Drugs |