Metformin for the Treatment of Endometrial Hyperplasia

The purpose of this study is to see if metformin will be effective in making endometrial hyperplasia without atypia better by returning the tissue to a normal state.

Study Overview

• Status: Completed
• Conditions: Endometrial Hyperplasia; Endometrial Hyperplasia Without Atypia
• Intervention / Treatment: Drug: Metformin

Detailed Description

This is a multi-institutional pilot clinical trial designed to estimate the response rate and safety of metformin for the treatment of simple and complex endometrial hyperplasia (EH) without atypia. Enrollment of patients will occur at UNC-Chapel Hill and Southern Pines Women's Health Center. Fifteen patients will be enrolled over the course of 1 year. Metformin will be initiated at 850 mg orally once daily, and titrated up to twice daily over a minimum of 1 months time. Treatment will last 12 weeks and then patients will undergo repeat endometrial biopsy to assess for regression or persistence of EH.

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • The University of North Carolina at Chapel Hill
    • Southern Pines, North Carolina, United States, 28388
      • Southern Pines Women's Health Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • University of Pennsylvania

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Be between the ages of 18-75 years old
• Have a confirmed diagnosis of endometrial hyperplasia without atypia based upon endometrial biopsy
• Have no contraindications to short-term metformin therapy
• Have a creatinine clearance of ≥ 90 ml/min, as calculated by the Cockroft-Gault formula
• Have normal serum transaminase values (AST and ALT)
• Need to be able to undergo metformin treatment for a duration of 12 weeks prior to repeat endometrial biopsy

Exclusion Criteria:

• Are currently taking metformin or have taken metformin in the past 6 months or have a history of an allergic reaction or intolerance at any time to metformin
• Have a history of liver or renal dysfunction.
• Have a random glucose of ≤ 65 or ≥ 200
• Have a recent history of alcoholism. Former alcoholics who have abstained from alcohol for 5 years or more may be enrolled in this study.
• Have a history of vitamin B12 deficiency
• Are pregnant
• Are currently taking insulin
• Are taking a drug that may significantly interact or influence the metabolism of metformin
• In the opinion of the investigator, the patient is felt not to be appropriate for the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Metformin; Metformin once daily for 4 weeks (weeks 1-4) and then twice daily for 8 weeks (weeks 5-12).	Drug: Metformin; 850 mg of metformin taken once daily by mouth for 4 weeks (weeks 1-4) and then twice daily by mouth for 8 weeks (weeks 5-12).; Other Names: Glucophage; Glumetza; Fortamet; Riomet

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response Rate	Evaluation of hyperplasia resolution by comparison of pre- and post-treatment endometrial biopsies.	12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Toxicity evaluation	Number of subjects who experience side effects	12 weeks
Patient Compliance	Percentage of patients successfully completing metformin therapy.	12 weeks
Potential molecular markers in response to treatment with Metformin	Compare changes in potential biomarkers, including metabolic factors and molecular markers of downstream targets of the metformin/mTOR signaling pathway, before and after metformin treatment.	12 weeks

Collaborators and Investigators

• Sponsor: UNC Lineberger Comprehensive Cancer Center
• Investigators: Principal Investigator: Victoria Bae-Jump, MD, PhD, University of North Carolina, Chapel Hill

Publications and helpful links

Helpful Links

• UNC Lineberger Comprehensive Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2013
• Primary Completion (Actual): December 1, 2015
• Study Completion (Actual): December 1, 2015

Study Registration Dates

• First Submitted: July 26, 2012
• First Submitted That Met QC Criteria: September 13, 2012
• First Posted (Estimated): September 14, 2012

Study Record Updates

• Last Update Posted (Actual): April 27, 2025
• Last Update Submitted That Met QC Criteria: April 23, 2025
• Last Verified: January 1, 2019

More Information

Terms related to this study

• Keywords: Metformin; Uterus; Pilot; Endometrial hyperplasia
• Additional Relevant MeSH Terms: Urogenital Diseases; Genital Diseases; Pathologic Processes; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Uterine Diseases; Genital Diseases, Female; Hyperplasia; Endometrial Hyperplasia; Physiological Effects of Drugs; Hypoglycemic Agents; Metformin
• Other Study ID Numbers: LCCC 1205