Levonorgestrel-Releasing Intrauterine System (LNG-IUS) in the Management of Atypical Endometrial Hyperplasia
Primary Purpose
Endometrial Hyperplasia
Status
Withdrawn
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Megestrol Acetate
Levonorgestrel Drug Implant
Sponsored by
About this trial
This is an interventional treatment trial for Endometrial Hyperplasia
Eligibility Criteria
Inclusion Criteria:
- Dilatation and curettage proven complex atypical endometrial hyperplasia only. Confirmed by pathology report.
- Normal renal function and liver function tests.
- Age 18 or older.
- The effects of megestrol acetate on the developing human fetus at the recommended therapeutic dose are unknown. For this reason and because megestrol acetate is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
Exclusion Criteria:
- Prior complex atypical endometrial hyperplasia or carcinoma.
- Prior hormone sensitive malignancy.]
- Exogenous estrogen or progestin use presently or within the past 12 months.
- Standard contraindications to progestin therapy.
- Standard contraindications to intrauterine device use.
- Simple hyperplasia, complex hyperplasia without atypia (may be present in addition to atypical endometrial hyperplasia).
- Endometrial carcinoma (worrisome or possible carcinoma not exclusionary but requires dilatation and curettage if based only on office biopsy).
- Pregnant women are excluded from this study because megestrol acetate has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with megestrol acetate, breastfeeding should be discontinued if the mother is treated with megestrol acetate.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
Megestrol Acetate Arm - Control Arm
Levonorgestrel IUD - Comparison Arm
Arm Description
Megestrol Acetate 160 mg by mouth daily
Levonorgestrel intrauterine device with 52 mg progestin (Releases 20mcg/daily)
Outcomes
Primary Outcome Measures
Histologic Response Scoring of Biopsy Specimens
The primary outcome is a response score (0: no response, 1: incomplete response, 2: complete response): Score -1 will be assigned to specimens with progression of disease (ie. Endometrial carcinoma); Score 0 will be assigned to specimens with atypical hyperplasia; Score 1 will be assigned to specimens with benign hyperplasia and will be indicative partial response to therapy; Score 2 will be assigned to specimens without hyperplasia and will be indicative of complete response to therapy. All study cases will be reviewed by a pathologist and all cases will be scored following the standard 2014 WHO classification of endometrial hyperplasia. Investigators will compare the estimated 90% confidence interval (CI) of the levonorgestrel-releasing Intrauterine system versus the standard therapy from the noninferiority trial to a predefined margin.
Secondary Outcome Measures
Number of Toxicities for Each Treatment
Toxicities for each treatment modality based on standard toxicity scoring using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Investigators will compare the number of toxicities between the two treatment groups using chi-squared tests.
Pathological Response to Treatment
The pathological response variable will be treated as a binary variable, the Wilcoxon rank sum test will be used to explore the association between measurement of the endometrial stripe and the response outcome at each visit. Score -1 will be assigned to specimens with progression of disease (ie. Endometrial carcinoma); Score 0 will be assigned to specimens with atypical hyperplasia; Score 1 will be assigned to specimens with benign hyperplasia and will be indicative partial response to therapy; Score 2 will be assigned to specimens without hyperplasia and will be indicative of complete response to therapy.
Full Information
NCT ID
NCT04897217
First Posted
May 17, 2021
Last Updated
July 3, 2023
Sponsor
Wake Forest University Health Sciences
Collaborators
National Cancer Institute (NCI)
1. Study Identification
Unique Protocol Identification Number
NCT04897217
Brief Title
Levonorgestrel-Releasing Intrauterine System (LNG-IUS) in the Management of Atypical Endometrial Hyperplasia
Official Title
Levonorgestrel-Releasing Intrauterine System (LNG-IUS) in the Management of Atypical Endometrial Hyperplasia: A Non-Inferiority Study
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Withdrawn
Why Stopped
PI decision
Study Start Date
June 30, 2023 (Anticipated)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
November 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wake Forest University Health Sciences
Collaborators
National Cancer Institute (NCI)
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this research study is to compare the uterus tissue of women who receive an intrauterine system to treat their endometrial hyperplasia with the uterine tissue of women who receive megestrol acetate to treat their hyperplasia. While both methods are commonly used in practice, investigators would like to see what effects each treatment has on uterine tissue.
Detailed Description
Primary Objective: To determine if Levonorgestrel-releasing intrauterine system is of equal efficacy to the standard systemic progestin therapy (megestrol acetate) based on endometrial sampling at 6 months after randomization. Non-inferiority analysis.
Secondary Objective(s):
To determine the safety of each treatment modality.
Determine the feasibility of transvaginal ultrasound to predict treatment response.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Endometrial Hyperplasia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Megestrol Acetate Arm - Control Arm
Arm Type
Active Comparator
Arm Description
Megestrol Acetate 160 mg by mouth daily
Arm Title
Levonorgestrel IUD - Comparison Arm
Arm Type
Experimental
Arm Description
Levonorgestrel intrauterine device with 52 mg progestin (Releases 20mcg/daily)
Intervention Type
Drug
Intervention Name(s)
Megestrol Acetate
Intervention Description
The control arm will consist of oral progesterone therapy (megestrol acetate 160mg po daily). The intervention will be administered on an outpatient basis.
Intervention Type
Drug
Intervention Name(s)
Levonorgestrel Drug Implant
Intervention Description
Participants in the comparison arm will have a levonorgestrel intrauterine device placed in the office or in the operating room if the office procedure not tolerated or if they have not had a prior dilation and curettage.
Primary Outcome Measure Information:
Title
Histologic Response Scoring of Biopsy Specimens
Description
The primary outcome is a response score (0: no response, 1: incomplete response, 2: complete response): Score -1 will be assigned to specimens with progression of disease (ie. Endometrial carcinoma); Score 0 will be assigned to specimens with atypical hyperplasia; Score 1 will be assigned to specimens with benign hyperplasia and will be indicative partial response to therapy; Score 2 will be assigned to specimens without hyperplasia and will be indicative of complete response to therapy. All study cases will be reviewed by a pathologist and all cases will be scored following the standard 2014 WHO classification of endometrial hyperplasia. Investigators will compare the estimated 90% confidence interval (CI) of the levonorgestrel-releasing Intrauterine system versus the standard therapy from the noninferiority trial to a predefined margin.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Number of Toxicities for Each Treatment
Description
Toxicities for each treatment modality based on standard toxicity scoring using NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Investigators will compare the number of toxicities between the two treatment groups using chi-squared tests.
Time Frame
Up to 8 months
Title
Pathological Response to Treatment
Description
The pathological response variable will be treated as a binary variable, the Wilcoxon rank sum test will be used to explore the association between measurement of the endometrial stripe and the response outcome at each visit. Score -1 will be assigned to specimens with progression of disease (ie. Endometrial carcinoma); Score 0 will be assigned to specimens with atypical hyperplasia; Score 1 will be assigned to specimens with benign hyperplasia and will be indicative partial response to therapy; Score 2 will be assigned to specimens without hyperplasia and will be indicative of complete response to therapy.
Time Frame
Up to 8 months
10. Eligibility
Sex
Female
Gender Based
Yes
Gender Eligibility Description
Women of all races and ethnicity who meet the eligibility criteria are eligible for this trial. Men are excluded from participation due to the site-specific nature of the disease being studied (endometrial hyperplasia).
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Complex atypical endometrial hyperplasia only. Confirmed by pathology report.
Normal renal function and liver function tests.
Age 18 or older.
The effects of megestrol acetate on the developing human fetus at the recommended therapeutic dose are unknown. For this reason and because megestrol acetate is known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
Exclusion Criteria:
Prior complex atypical endometrial hyperplasia or carcinoma.
Prior hormone sensitive malignancy.]
Exogenous estrogen or progestin use presently or within the past 12 months.
Standard contraindications to progestin therapy.
Standard contraindications to intrauterine device use.
Simple hyperplasia, complex hyperplasia without atypia (may be present in addition to atypical endometrial hyperplasia).
Endometrial carcinoma (worrisome or possible carcinoma not exclusionary but requires dilatation and curettage if based only on office biopsy).
Pregnant women are excluded from this study because megestrol acetate has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with megestrol acetate, breastfeeding should be discontinued if the mother is treated with megestrol acetate.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Janelle Darby, MD
Organizational Affiliation
Wake Forest Baptist Health Sciences
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Levonorgestrel-Releasing Intrauterine System (LNG-IUS) in the Management of Atypical Endometrial Hyperplasia
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