Trial Outcomes & Findings for An Endometrial Cancer Chemoprevention Study of Metformin (NCT NCT01697566)
NCT ID: NCT01697566
Last Updated: 2025-06-11
Results Overview
This study will use a 2 x 2 randomized design with all 100 post- menopausal subjects being randomized to metformin, placebo, placebo and intensive lifestyle intervention, or metformin and intensive lifestyle intervention. Treatments will last 4 months. Participants will be assessed at baseline and end of treatment with endometrial sampling to see if there are changes in the biomarkers.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
29 participants
Primary outcome timeframe
Visit 2 (1 week), 4 months, and 1 year
Results posted on
2025-06-11
Participant Flow
Recruitment was from May 2013 until May 2016 and all recruitments were done in a medical clinic setting.
Participant milestones
| Measure |
Metformin + Lifestyle
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
7
|
7
|
8
|
|
Overall Study
COMPLETED
|
7
|
7
|
5
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
2
|
1
|
Reasons for withdrawal
| Measure |
Metformin + Lifestyle
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
0
|
1
|
1
|
|
Overall Study
Final biopsy not obtained
|
0
|
0
|
1
|
0
|
Baseline Characteristics
An Endometrial Cancer Chemoprevention Study of Metformin
Baseline characteristics by cohort
| Measure |
Metformin + Lifestyle
n=7 Participants
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 Participants
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=7 Participants
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=8 Participants
Placebo given daily with no lifestyle intervention program
|
Total
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
29 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Continuous
|
56.3 years
n=5 Participants
|
56 years
n=7 Participants
|
57 years
n=5 Participants
|
59.8 years
n=4 Participants
|
57.4 years
n=21 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
29 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
13 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
7 participants
n=7 Participants
|
7 participants
n=5 Participants
|
8 participants
n=4 Participants
|
29 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Visit 2 (1 week), 4 months, and 1 yearThis study will use a 2 x 2 randomized design with all 100 post- menopausal subjects being randomized to metformin, placebo, placebo and intensive lifestyle intervention, or metformin and intensive lifestyle intervention. Treatments will last 4 months. Participants will be assessed at baseline and end of treatment with endometrial sampling to see if there are changes in the biomarkers.
Outcome measures
| Measure |
Metformin + Lifestyle
n=7 Participants
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 Participants
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=5 Participants
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=7 Participants
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
To Evaluate the Participant Body Weight in Pounds (Lbs)
Weight Month 4
|
101.19 lbs
Standard Deviation 22.47
|
96.39 lbs
Standard Deviation 15.36
|
97.98 lbs
Standard Deviation 16.46
|
94.37 lbs
Standard Deviation 13.46
|
|
To Evaluate the Participant Body Weight in Pounds (Lbs)
Weight Year 1
|
102.08 lbs
Standard Deviation 35.32
|
95.93 lbs
Standard Deviation 12.63
|
93.25 lbs
Standard Deviation 8.15
|
94.96 lbs
Standard Deviation 13.68
|
|
To Evaluate the Participant Body Weight in Pounds (Lbs)
Weight Visit 2
|
108.57 lbs
Standard Deviation 22.51
|
99.3 lbs
Standard Deviation 15.36
|
101.64 lbs
Standard Deviation 13.44
|
94.1 lbs
Standard Deviation 12.89
|
PRIMARY outcome
Timeframe: Visit 2 (1 week), 4 months, and 1 yearThe Q-PCR analyses will be performed in the Quantitative Genomics Core Facility at the University of Texas Medical School at Houston. Over the last 3 years we have used several techniques including differential display, microchip array screening, and real-time quantitative PCR to find such genes. The tools we have developed are directly applicable to the investigation of surrogate endpoint biomarkers (SEBs). Identification of appropriate biomarkers is essential for this study.
Outcome measures
| Measure |
Metformin + Lifestyle
n=7 Participants
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 Participants
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=5 Participants
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=7 Participants
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
To Evaluate the Percentage of Ki-67 Positive Cells
Visit 2
|
8.78 percentage of cells
Standard Deviation 8.29
|
7.95 percentage of cells
Standard Deviation 5.13
|
9.23 percentage of cells
Standard Deviation 3.96
|
4.73 percentage of cells
Standard Deviation 1.39
|
|
To Evaluate the Percentage of Ki-67 Positive Cells
Month 4
|
6.73 percentage of cells
Standard Deviation 4.17
|
4.98 percentage of cells
Standard Deviation 3.94
|
4.17 percentage of cells
Standard Deviation 2.37
|
9.1 percentage of cells
Standard Deviation 5.96
|
SECONDARY outcome
Timeframe: Visit 2 (1 week), 4 months, and 1 yearOutcome measures
| Measure |
Metformin + Lifestyle
n=7 Participants
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 Participants
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=5 Participants
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=7 Participants
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
BMI
BMI Month 4
|
39.83 weight (kg) / height (m)2
Standard Deviation 7.82
|
37.34 weight (kg) / height (m)2
Standard Deviation 4.78
|
37.83 weight (kg) / height (m)2
Standard Deviation 5.53
|
35.31 weight (kg) / height (m)2
Standard Deviation 4.16
|
|
BMI
BMI Year 1
|
41.13 weight (kg) / height (m)2
Standard Deviation 13.45
|
37.18 weight (kg) / height (m)2
Standard Deviation 4.41
|
36 weight (kg) / height (m)2
Standard Deviation 2.3
|
35.54 weight (kg) / height (m)2
Standard Deviation 4.23
|
|
BMI
BMI Visit 2
|
45.25 weight (kg) / height (m)2
Standard Deviation 9.36
|
38.17 weight (kg) / height (m)2
Standard Deviation 5.18
|
39.49 weight (kg) / height (m)2
Standard Deviation 4.71
|
35.29 weight (kg) / height (m)2
Standard Deviation 4
|
SECONDARY outcome
Timeframe: Visit 2 (1 week), 4 months, and 1 yearOutcome measures
| Measure |
Metformin + Lifestyle
n=7 Participants
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 Participants
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=5 Participants
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=7 Participants
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
Weight Loss
Weight Loss (Month 4 - Visit 2)/Visit 2
|
-7.10 percentage of weight loss
Standard Deviation 5.22
|
-2.95 percentage of weight loss
Standard Deviation 2.34
|
-3.88 percentage of weight loss
Standard Deviation 5.11
|
0.25 percentage of weight loss
Standard Deviation 1.61
|
|
Weight Loss
Weight Loss (Year 1 - Visit 2)/Visit 2
|
-5.49 percentage of weight loss
Standard Deviation 13.51
|
-3.09 percentage of weight loss
Standard Deviation 3.45
|
-3.37 percentage of weight loss
Standard Deviation 4.92
|
0.83 percentage of weight loss
Standard Deviation 0.95
|
Adverse Events
Metformin + Lifestyle
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Metformin + No Lifestyle
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo + Lifestyle
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Placebo + No Lifestyle
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Metformin + Lifestyle
n=7 participants at risk
Metformin 1700 mg daily along with lifestyle intervention program
|
Metformin + No Lifestyle
n=7 participants at risk
Metformin 1700 mg daily with no lifestyle intervention program
|
Placebo + Lifestyle
n=7 participants at risk
Placebo given daily along with lifestyle intervention program
|
Placebo + No Lifestyle
n=8 participants at risk
Placebo given daily with no lifestyle intervention program
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Flatulence
|
28.6%
2/7 • Number of events 7 • An average of 36 months
NCI CTCAE version 4.03
|
57.1%
4/7 • Number of events 12 • An average of 36 months
NCI CTCAE version 4.03
|
28.6%
2/7 • Number of events 6 • An average of 36 months
NCI CTCAE version 4.03
|
50.0%
4/8 • Number of events 7 • An average of 36 months
NCI CTCAE version 4.03
|
|
Gastrointestinal disorders
Diarrhea
|
14.3%
1/7 • Number of events 2 • An average of 36 months
NCI CTCAE version 4.03
|
71.4%
5/7 • Number of events 11 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
|
Nervous system disorders
Headache
|
28.6%
2/7 • Number of events 4 • An average of 36 months
NCI CTCAE version 4.03
|
57.1%
4/7 • Number of events 8 • An average of 36 months
NCI CTCAE version 4.03
|
42.9%
3/7 • Number of events 4 • An average of 36 months
NCI CTCAE version 4.03
|
25.0%
2/8 • Number of events 3 • An average of 36 months
NCI CTCAE version 4.03
|
|
Gastrointestinal disorders
Nausea
|
14.3%
1/7 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
42.9%
3/7 • Number of events 5 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 2 • An average of 36 months
NCI CTCAE version 4.03
|
|
Gastrointestinal disorders
Constipation
|
14.3%
1/7 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
14.3%
1/7 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
|
Eye disorders
Blurred vision
|
14.3%
1/7 • Number of events 2 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/8 • An average of 36 months
NCI CTCAE version 4.03
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
42.9%
3/7 • Number of events 8 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
37.5%
3/8 • Number of events 4 • An average of 36 months
NCI CTCAE version 4.03
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
14.3%
1/7 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/8 • An average of 36 months
NCI CTCAE version 4.03
|
|
General disorders
Fatigue
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
14.3%
1/7 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 2 • An average of 36 months
NCI CTCAE version 4.03
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 3 • An average of 36 months
NCI CTCAE version 4.03
|
|
General disorders
Flu like symptoms
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 3 • An average of 36 months
NCI CTCAE version 4.03
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness right-sided
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness trunk
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
0.00%
0/7 • An average of 36 months
NCI CTCAE version 4.03
|
12.5%
1/8 • Number of events 1 • An average of 36 months
NCI CTCAE version 4.03
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place