EndometriosisPhase 3NCT03343067
A Study to Evaluate Safety and Efficacy of Elagolix in Participants With Endometriosis With Associated Moderate to Severe Pain
A Phase 3 study testing elagolix and estradiol/norethindrone acetate (E2/NETA) in people with Endometriosis, run by AbbVie at 37 sites in United States.
Trial summary
This is a Phase 3, dose-escalation study designed to evaluate the safety and efficacy of both elagolix alone and elagolix plus estradiol/norethindrone acetate (E2/NETA) over 24 months in the management of endometriosis with associated moderate to severe pain in premenopausal women. Read more about this study
- Who is it for?
- Ages 18 to 49 years, women only
- Conditions the study is for
- Endometriosis
- What is being tested?
- elagolix and estradiol/norethindrone acetate (E2/NETA)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 37 sitesUnited States
- Study dates
- 27 Dec 2017to 23 Oct 2018
- Phase
- Phase 3
- 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 to 49 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Participant is a premenopausal female 18 to 49 years of age (inclusive) at the time of Screening.
- Participant has a documented surgical diagnosis (e.g., laparoscopy or laparotomy) of endometriosis established by visualization or histology within 10 years prior to entry into Washout or Screening,
- Participant must agree to use only those rescue analgesics permitted by the protocol during the Screening and Treatment Periods for her endometriosis-associated pain.
- Participant must have the following documented in the e-Diary during the last 35 days prior to Study Day 1:
- At least 2 days of "moderate" or "severe" DYS, AND either,
- At least 2 days of "moderate" or "severe" NMPP and an average NMPP score of at least 1.0, OR
- At least 4 days of "moderate" or "severe" NMPP and an average NMPP score of at least 0.5.
Who cannot join
- Participant has chronic pelvic pain that is not caused by endometriosis that requires chronic analgesic therapy, which would interfere with the assessment of endometriosis-associated pain.
- Participant is using any systemic corticosteroids for over 14 days within 3 months prior to Screening or is likely to require treatment with systemic corticosteroids during the course of the study. Over-the-counter and prescription topical, inhaled or intranasal corticosteroids are allowed.
- Participant has a history of major depression or post-traumatic stress disorder (PTSD) within 2 years prior to screening or other major psychiatric disorder at any time
- Participant has a history of suicide attempts or answered "yes" to questions 4 or 5 on the suicidal ideation portion of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening or prior to study drug dosing on Day 1.
- Participant has any condition that would interfere with obtaining adequate dual energy x-ray absorptiometry (DXA) measurements
- Screening DXA results of the lumbar spine (L1 - L4), femoral neck or total hip bone mineral density (BMD) corresponding to less than 2 or more standard deviations below normal (Z-score < -2.0 for participants < 40 years of age, T-score for participants >= 40 years of age).
Show all 11Show fewer
- Participant has either
- a newly diagnosed, clinically significant medical condition that requires therapeutic intervention (e.g., new onset hypertension) that has not been stabilized 30 days prior to enrollment on Day 1 OR
- a clinically significant medical condition that is anticipated to required intervention during the course of study participation (e.g., anticipated major elective surgery) OR
- an unstable medical condition that makes the participant an unsuitable candidate for the study in the opinion of the Investigator
- Participant has any conditions contraindicated with use of E2/NETA
Trial locations and contacts
37 sites in 1 country.
37 sites in 1 country
United States37 sites
Radiant Research, Inc /ID# 200045
Akron, Ohio, 44311
Pinnacle Research Group /ID# 202016
Anniston, Alabama, 36207
Montana Health Research Inst /ID# 170624
Billings, Montana, 59102
Bingham Memorial Hospital /ID# 170110
Blackfoot, Idaho, 83221
Nova Clinical Research, LLC /ID# 202227
Bradenton, Florida, 34209-4616
Quad Clinical Research, LLC /ID# 168294
Chicago, Illinois, 60605
Quad Clinical Research, LLC /ID# 170629
Chicago, Illinois, 60605
Central Ohio Clinical Research /ID# 170750
Columbus, Ohio, 43213-3399
Optimed Research /ID# 167642
Columbus, Ohio, 43235
Midland Florida Clinical Research Center /ID# 201327
DeLand, Florida, 32720-0920
Southeastern Integrated Med /ID# 203109
Gainesville, Florida, 32607
SC Clinical Research /ID# 165049
Garden Grove, California, 92844
VitaLink Research /ID# 168401
Greenville, South Carolina, 29601
VitaLink Research /ID# 170625
Greenville, South Carolina, 29601
Centex Studies, Inc /ID# 169897
Houston, Texas, 77058-2705
America's Adv. Wellness Center /ID# 170005
Houston, Texas, 77080
Marvel Clinical Research /ID# 169633
Huntington Beach, California, 92647
Leavitt Womens Healthcare /ID# 169495
Idaho Falls, Idaho, 83404-8322
Care Partners Clinical Researc /ID# 168395
Jacksonville, Florida, 32277
Excel Clinical Research /ID# 170620
Las Vegas, Nevada, 89109
Lynn Institute of the Ozarks /ID# 165052
Little Rock, Arkansas, 72205
Northwell health system /ID# 200162
Manhasset, New York, 11030-3816
LCC Medical Research Institute /ID# 168888
Miami, Florida, 33126
Delricht Research /ID# 200219
New Orleans, Louisiana, 70115
Women's Health Care, PC /ID# 165033
Newburgh, Indiana, 47630-8940
Eastern Virginia Med School /ID# 165016
Norfolk, Virginia, 23507-1627
A Premier Clinical Research of Florida, LLC /ID# 201887
Orange City, Florida, 32763-2833
ClinRx Research, LLC /ID# 201189
Plano, Texas, 75024-5280
Progressive Clinical Research /ID# 205565
San Antonio, Texas, 78229
Health care Affiliates Medical Group /ID# 165048
Santa Ana, California, 92704
Seattle Reproductive Medicine /ID# 171079
Seattle, Washington, 98109
Fusion Clinical Research of Spartanburg /ID# 200010
Spartanburg, South Carolina, 29301-5642
Quad Clinical Research, LLC /ID# 200180
St Louis, Missouri, 63108-3204
GCP Clinical Research, LLC /ID# 169774
Tampa, Florida, 33609-4044
Noble Clinical Research /ID# 170628
Tucson, Arizona, 85704
Triple O Research Institute /ID# 201128
West Palm Beach, Florida, 33407-3100
Western States Clinical Res /ID# 169809
Wheat Ridge, Colorado, 80033-2896
About this study
Official title: A Study to Evaluate Safety and Efficacy of Elagolix Alone or Elagolix With Hormonal Add-Back in Subjects With Endometriosis With Associated Moderate to Severe Pain
This is a Phase 3, dose-escalation study designed to evaluate the safety and efficacy of both elagolix alone and elagolix plus estradiol/norethindrone acetate (E2/NETA) over 24 months in the management of endometriosis with associated moderate to severe pain in premenopausal women.
- Conditions
- Endometriosis
- Being tested
- elagolix, estradiol/norethindrone acetate (E2/NETA)
- Keywords
- Endometriosis associated pain, Elagolix, Non-menstrual pelvic pain (NMPP), Dysmenorrhea (DYS)
- Medical subject headings
- Endometriosis, Dysmenorrhea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
elagolixDrug
Tablets
estradiol/norethindrone acetate (E2/NETA)Drug
Capsules
| Study group | Role | What they receive |
|---|---|---|
| Arm A | Experimental | Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD
Month 4 Through Month 24 (Double-Blind): Efficacy responders to elagolix 150 mg QD at Month 3 and continue treatment through Month 24 Receives: Drug: elagolix |
| Arm C | Experimental | Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD
Month 4 Through Month 24 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 200 mg BID plus E2/NETA 1/0.5 mg QD treatment group and continue treatment through Month 24. Receives: Drug: elagolix; Drug: estradiol/norethindrone acetate (E2/NETA) |
| Arm D | Experimental | Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD
Month 4 Through Month 6 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 150 mg QD treatment group
Month 7 Through Month 24 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 6 and assigned to elagolix 200 mg BID plus E2/NETA 1/0.5 mg QD treatment group and continue treatment through Month 24. Receives: Drug: elagolix; Drug: estradiol/norethindrone acetate (E2/NETA) |
| Arm B | Experimental | Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD
Month 4 Through Month 6 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 150 mg QD treatment group
Month 7 Through Month 24 (Double-Blind): Efficacy responders to elagolix 150 mg QD at Month 6 and continue treatment through Month 24. Receives: Drug: elagolix |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Responders at Month 6 Based on DYS Pain Scale
Proportion of responders at Month 6 based upon the scale for DYS pain scale (ranging from 0 [none] to 3 [severe]) using the daily e-Diary, as well as no increased analgesic rescue use for endometriosis-associated pain.
Measured: Month 6
Proportion of Responders at Month 6 Based on NMPP Pain Scale
Proportion of responders at Month 6 based upon the scale for NMPP pain scale (ranging from 0 [none] to 3 [severe]) using the daily e-Diary, as well as no increased analgesic rescue use for endometriosis-associated pain.
Measured: Month 6
Secondary outcomes (38)
Change From Baseline in DYS at Month 6
The DYS pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Month 6
Change From Baseline in NMPP at Month 6
The NMPP pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Month 6
Change From Baseline in Dyspareunia at Month 6
The dyspareunia pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Month 6
Change From Baseline in Daily Diary Endometriosis-Associated Pain Score Via Numeric Rating Scale (NRS) at Month 6
The NRS for overall endometriosis-associated pain ranges from 0 (none) to 10 (worst pain ever), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Month 6
Change From Baseline in Rescue Analgesic Use Across Both Classes of Rescue Analgesics (NSAIDs/Opioids) at Month 6
Based on average pill counts and assessed using the daily e-Diary. Permitted non-steroidal anti-inflammatory drugs (NSAIDs) included naproxen, ibuprofen, diclofenac, and celecoxib. Permitted opioids included hydrocodone + acetaminophen and codeine phosphate + acetaminophen.
Measured: Month 0 (baseline), Month 6
Percentage of Participants With 30% or More Reduction From Baseline Based on the 35 Day Mean of the Daily Diary Endometriosis-Associated Pain Score Via NRS at Month 6
The NRS for overall endometriosis-associated pain ranges from 0 (none) to 10 (worst pain ever), recorded in a daily eDiary and averaged monthly based on a 35-day window.
Measured: Month 6
Proportion of Responders Over Time
As assessed by change and percent change from baseline in average pain score and rescue analgesic use monthly for DYS, NMPP, dyspareunia, and Daily Diary endometriosis-associated pain score via NRS, respectively.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Proportion of Responders Over Time (Not Taking Into Consideration of Analgesic Use)
As assessed by change and percent change from Baseline in average pain score and rescue analgesic use monthly for DYS, NMPP, dyspareunia, and Daily Diary endometriosis-associated pain score via NRS, respectively.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average DYS Pain Score
The DYS pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
The analysis was based on a 28-day window. If a participant prematurely discontinued during the open-label period, based on the analysis window, some data might fall into Month 4 analysis.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average NMPP Pain Score
The NMPP pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average Dyspareunia Pain Score
The dyspareunia pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average Daily Diary Endometriosis-Associated Pain Score Via NRS
The NRS for overall endometriosis-associated pain ranges from 0 (none) to 10 (worst pain ever), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for DYS
The DYS pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for NMPP
The NMPP pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for Dyspareunia
The dyspareunia pain scale score ranged from 0 to 3 (0=none, 1=mild, 2=moderate, 3=severe), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for Daily Diary Endometriosis-Associated Pain Score Via NRS
The NRS for overall endometriosis-associated pain ranges from 0 (none) to 10 (worst pain ever), recorded in a daily eDiary and averaged monthly based on a 35-day window. A negative change from baseline indicates improvement.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Daily Rescue Analgesic Use Across Both Classes of Rescue Analgesics
Based on average pill counts and assessed using a daily e-Diary. Permitted non-steroidal anti-inflammatory drugs (NSAIDs) included naproxen, ibuprofen, diclofenac, and celecoxib. Permitted opioids included hydrocodone + acetaminophen and codeine phosphate + acetaminophen.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Number of Analgesic Use Responders and Non-Responders Over Time
Based only on reduction of rescue analgesics used. Responders were defined as:
- participants with no analgesic use at screening and no analgesic use added
- participants with NSAID only use at screening and NSAID dose stopped, decreased, or stable (<15% increase) and no opioid use added
- participants with opioid only use at screening and opioid dose stopped, decreased, or stable (<15% increase), opioid dose stopped and NSAID substituted (any dose), opioid dose decreased and NSAID added (any dose)
- participants with NSAID + opioid use at screening and any of the following: NSAID dose stopped + opioid analgesic use stopped, decreased, or stable (<15% increase); NSAID dose decreased + opioid analgesic use stopped, decreased, or stable (<15% increase); NSAID dose stable (< 15% increase) + opioid analgesic use stopped, decreased, or stable (<15% increase); NSAID dose increased by >15% + opioid analgesic use stopped; NSAID dose increased by >15% + opioid analgesic dose decreases.
Measured: Months 1, 2, 3, 4, 5, 6
Patient Global Impression of Change (PGIC) Scores Over Time
The PGIC is a 7-point response scale where participants rate their endometriosis related pain as: very much improved (1), much improved (2), minimally improved (3), not changed (4), minimally worse (5), much worse (6), very much worse (7).
Measured: Months 1, 2, 3, 4, 5, 6
Overall Endometriosis-Associated Pain Via NRS (7-Day Recall) Scores Over Time
The endometriosis-associated pain questionnaire is an 11-point NRS assessing overall endometriosis-associated pain over a 7-day recall period. Participants assessed their endometriosis-associated pain on a scale of 0 to 10, with 0 = no pain and 10 = worst pain ever.
Measured: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Endometriosis Health Profile-30 (EHP-30) Scores Over Time: Pain
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Control and Powerlessness
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Emotional Well-Being
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Social Support
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Self-Image
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Sexual Intercourse
The EHP-30 is a disease-specific self-administered questionnaire used to measure health-related quality of life in women with endometriosis. Domains used in the study included Pain, Control and Powerlessness, Well-Being, Social Support, Self-Image, and Sexual Intercourse. Each domain is calculated on a scale from 0 = best possible health status to 100 = worst possible health status.
Measured: Month 0 (baseline), Months 3, 6
EuroQol-5D 5 Level (EQ-5D-5L) Scores Over Time: Mobility
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate visual analog scale (VAS) indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Self-Care
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate visual analog scale (VAS) indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Usual Activities
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate visual analog scale (VAS) indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Pain/Discomfort
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate visual analog scale (VAS) indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Anxiety/Depression
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate visual analog scale (VAS) indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
EQ-5D-5L VAS Scores Over Time: Health Today
The EQ-5D-5L is a health state utility instrument with 5 items that comprise 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each of which is rated on 5 levels of severity (1=no problem, 2=slight problems, 3=moderate problems, 4=severe problems, 5=extreme problems) and a separate VAS indicating a participant's rating of their current health status (health today) on a scale of from 0 (worst health imaginable) to 100 (best health imaginable).
Measured: Month 0 (baseline), Months 3, 6
Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) Scores Over Time: Percent Work Missed Due to Problem
Absenteeism is presented as the mean percentage of work time missed due to health problem (as reported on the WPAI:SHP), and is calculated as: 100*number of hours of work missed due to health problem / (number of hours of work missed due to health problem + number of hours worked). WPAI is a questionnaire used to evaluate lost productivity; scores are presented as percentages (multiplying the scores by 100), with 0% representing no impact on productivity and 100% representing complete impact on productivity.
Measured: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Impairment While Working Due to Problem
Presenteeism (the extent to which health problem decreased productivity) is presented as the mean percentage of impairment while working due to health problem, and is calculated as: 100*scale value of question 5 on the WPAI (between 0 and 10) / 10. WPAI:SHP is a questionnaire used to evaluate lost productivity; scores are presented as percentages (multiplying the scores by 100), with 0% representing no impact on productivity and 100% representing complete impact on productivity.
Measured: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Overall Work Impairment Due to Problem
The mean percentage of overall work impairment due to health problem (based on the WPAI questionnaire) is presented, and is calculated as: Absenteeism (%) + extent to which health problem decreased productivity (%)* [number of hours worked / (number of hours of work missed due to health problem + number of hours worked)]. WPAI is a questionnaire used to evaluate lost productivity; scores are presented as percentages (multiplying the scores by 100), with 0% representing no impact on productivity and 100% representing complete impact on productivity.
Measured: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Activity Impairment Due to Problem
Activity impairment due to health problem (the extent to which health problem affected the ability to perform usual daily activities) is presented as the mean percentage of activity impairment, and is calculated as 100*scale value of WPAI question 6 (between 0 and 10) / 10. WPAI is a questionnaire used to evaluate lost productivity; scores are presented as percentages (multiplying the scores by 100), with 0% representing no impact on productivity and 100% representing complete impact on productivity.
Measured: Month 0 (baseline), Month 6
Health Endometriosis Treatment Satisfaction Questionnaire (ETSQ) Scores Over Time
The 6-item ETSQ was developed to assess patient-reported satisfaction with effects on endometriosis pain, dysmenorrhea, dyspareunia, amount of bleeding tolerability and overall treatment satisfaction. The ETSQ has a 7 point response scale. The range for this scale is 0 to 36, with lower ETSQ scores reflecting lower levels of satisfaction with endometriosis treatment.
Measured: Month 0 (baseline), Months 3, 6
PROMIS Fatigue Short Form 6a Scores Over Time
The PROMIS Fatigue Short Form 6a is self-administered and composed of 6 questions to evaluate fatigue. Possible scores range from 6 to 30, 6 = not at all (no fatigue), and 30 = very much (most fatigue).
Measured: Month 0 (baseline), Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start27 Dec 2017
- Primary completion23 Oct 2018
- Study completion23 Oct 2018
Why it stopped: The study was terminated early for business reasons, not for safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for EndometriosisStudies of the same condition, open and under way.
- More trials run by AbbVieThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- AbbVie
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- AbbVie Inc., Study director, AbbVie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03343067
- Sponsor's protocol ID
- M16-383
- Status last verified
- Jul 2019
- First submitted
- 13 Nov 2017
- Last updated
- 27 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Evaluate Safety and Efficacy of Elagolix Alone or Elagolix With Hormonal Add-Back in Subjects With Endometriosis With Associated Moderate to Severe Pain
- ClinicalTrials.gov ID
- NCT03343067
- Acronym / sponsor ID
- M16-383
- Conditions
- Endometriosis
- Keywords
- Endometriosis associated pain, Elagolix, Non-menstrual pelvic pain (NMPP), Dysmenorrhea (DYS)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated early for business reasons, not for safety concerns.
- Enrolment
- 11 participants (actual)
- Lead sponsor
- AbbVieIndustry (pharma, biotech, devices)
- Study start
- 27 Dec 2017 (actual)
- Primary completion
- 23 Oct 2018 (actual)
- Study completion
- 23 Oct 2018 (actual)
- First posted
- 17 Nov 2017 (actual)
- Results
- Posted 27 Nov 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 3, dose-escalation study designed to evaluate the safety and efficacy of both elagolix alone and elagolix plus estradiol/norethindrone acetate (E2/NETA) over 24 months in the management of endometriosis with associated moderate to severe pain in premenopausal women.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm A | Experimental | Drug: elagolixDay 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD Month 4 Through Month 24 (Double-Blind): Efficacy responders to elagolix 150 mg QD at Month 3 and continue treatment through Month 24 |
| Arm C | Experimental | Drug: elagolix, Drug: estradiol/norethindrone acetate (E2/NETA)Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD Month 4 Through Month 24 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 200 mg BID plus E2/NETA 1/0.5 mg QD treatment group and continue treatment through Month 24. |
| Arm D | Experimental | Drug: elagolix, Drug: estradiol/norethindrone acetate (E2/NETA)Day 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD Month 4 Through Month 6 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 150 mg QD treatment group Month 7 Through Month 24 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 6 and assigned to elagolix 200 mg BID plus E2/NETA 1/0.5 mg QD treatment group and continue treatment through Month 24. |
| Arm B | Experimental | Drug: elagolixDay 1 Through Month 3 (Open-Label): Open-Label elagolix 150 mg QD Month 4 Through Month 6 (Double-Blind): Incomplete efficacy responders to elagolix 150 mg QD at Month 3 and randomized to elagolix 150 mg QD treatment group Month 7 Through Month 24 (Double-Blind): Efficacy responders to elagolix 150 mg QD at Month 6 and continue treatment through Month 24. |
Who may qualify
- Ages
- 18 to 49 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Participant is a premenopausal female 18 to 49 years of age (inclusive) at the time of Screening.
- Participant has a documented surgical diagnosis (e.g., laparoscopy or laparotomy) of endometriosis established by visualization or histology within 10 years prior to entry into Washout or Screening,
- Participant must agree to use only those rescue analgesics permitted by the protocol during the Screening and Treatment Periods for her endometriosis-associated pain.
- Participant must have the following documented in the e-Diary during the last 35 days prior to Study Day 1:
Key exclusion criteria (11 listed)
- Participant has chronic pelvic pain that is not caused by endometriosis that requires chronic analgesic therapy, which would interfere with the assessment of endometriosis-associated pain.
- Participant is using any systemic corticosteroids for over 14 days within 3 months prior to Screening or is likely to require treatment with systemic corticosteroids during the course of the study. Over-the-counter and prescription topical, inhaled or intranasal corticosteroids are allowed.
- Participant has a history of major depression or post-traumatic stress disorder (PTSD) within 2 years prior to screening or other major psychiatric disorder at any time
- Participant has a history of suicide attempts or answered "yes" to questions 4 or 5 on the suicidal ideation portion of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening or prior to study drug dosing on Day 1.
- Participant has any condition that would interfere with obtaining adequate dual energy x-ray absorptiometry (DXA) measurements
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| elagolixTablets | Drug | Arm A, Arm B, Arm C, Arm D |
| estradiol/norethindrone acetate (E2/NETA)Capsules | Drug | Arm C, Arm D |
Primary outcome
Proportion of Responders at Month 6 Based on DYS Pain Scale
Time frame: Month 6
Proportion of responders at Month 6 based upon the scale for DYS pain scale (ranging from 0 [none] to 3 [severe]) using the daily e-Diary, as well as no increased analgesic rescue use for endometriosis-associated pain.
Proportion of Responders at Month 6 Based on NMPP Pain Scale
Time frame: Month 6
Proportion of responders at Month 6 based upon the scale for NMPP pain scale (ranging from 0 [none] to 3 [severe]) using the daily e-Diary, as well as no increased analgesic rescue use for endometriosis-associated pain.
38 secondary outcomes
Change From Baseline in DYS at Month 6
Time frame: Month 0 (baseline), Month 6
Change From Baseline in NMPP at Month 6
Time frame: Month 0 (baseline), Month 6
Change From Baseline in Dyspareunia at Month 6
Time frame: Month 0 (baseline), Month 6
Change From Baseline in Daily Diary Endometriosis-Associated Pain Score Via Numeric Rating Scale (NRS) at Month 6
Time frame: Month 0 (baseline), Month 6
Change From Baseline in Rescue Analgesic Use Across Both Classes of Rescue Analgesics (NSAIDs/Opioids) at Month 6
Time frame: Month 0 (baseline), Month 6
Percentage of Participants With 30% or More Reduction From Baseline Based on the 35 Day Mean of the Daily Diary Endometriosis-Associated Pain Score Via NRS at Month 6
Time frame: Month 6
Proportion of Responders Over Time
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Proportion of Responders Over Time (Not Taking Into Consideration of Analgesic Use)
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average DYS Pain Score
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average NMPP Pain Score
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average Dyspareunia Pain Score
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Monthly Average Daily Diary Endometriosis-Associated Pain Score Via NRS
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for DYS
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for NMPP
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for Dyspareunia
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Percent Change From Baseline to Each Month During the Treatment Period for Daily Diary Endometriosis-Associated Pain Score Via NRS
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Change From Baseline Over Time in Daily Rescue Analgesic Use Across Both Classes of Rescue Analgesics
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Number of Analgesic Use Responders and Non-Responders Over Time
Time frame: Months 1, 2, 3, 4, 5, 6
Patient Global Impression of Change (PGIC) Scores Over Time
Time frame: Months 1, 2, 3, 4, 5, 6
Overall Endometriosis-Associated Pain Via NRS (7-Day Recall) Scores Over Time
Time frame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
Endometriosis Health Profile-30 (EHP-30) Scores Over Time: Pain
Time frame: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Control and Powerlessness
Time frame: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Emotional Well-Being
Time frame: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Social Support
Time frame: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Self-Image
Time frame: Month 0 (baseline), Months 3, 6
EHP-30 Scores Over Time: Sexual Intercourse
Time frame: Month 0 (baseline), Months 3, 6
EuroQol-5D 5 Level (EQ-5D-5L) Scores Over Time: Mobility
Time frame: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Self-Care
Time frame: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Usual Activities
Time frame: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Pain/Discomfort
Time frame: Month 0 (baseline), Months 3, 6
EQ-5D-5L Scores Over Time: Anxiety/Depression
Time frame: Month 0 (baseline), Months 3, 6
EQ-5D-5L VAS Scores Over Time: Health Today
Time frame: Month 0 (baseline), Months 3, 6
Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) Scores Over Time: Percent Work Missed Due to Problem
Time frame: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Impairment While Working Due to Problem
Time frame: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Overall Work Impairment Due to Problem
Time frame: Month 0 (baseline), Month 6
WPAI:SHP Scores Over Time: Percent Activity Impairment Due to Problem
Time frame: Month 0 (baseline), Month 6
Health Endometriosis Treatment Satisfaction Questionnaire (ETSQ) Scores Over Time
Time frame: Month 0 (baseline), Months 3, 6
PROMIS Fatigue Short Form 6a Scores Over Time
Time frame: Month 0 (baseline), Month 6
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.
Related trials for Endometriosis
Not yet recruiting
Prevalence of Small Intestinal Bacterial Overgrowth (SIBO) in Women With Endometriosis
SIBO, Endometriosis
Recruiting
Endometriosis Transcriptomic Cell Atlas
Endometriosis, Infertility
Not yet recruiting
Psychotherapy in Patients With Endometriosis: Cognitive Behavioral Therapy Versus Acceptance and Commitment Therapy
Endometriosis, Adenomyosis
Not yet recruitingObservational study
Impact of a Diagnosis or Lack of a Diagnosis of Endometriosis Via a Saliva Test on Pain Catastrophizing, Quality of Life, Symptoms of Anxiety and Depression, and Treatment Adherence
Endometriosis
Not yet recruitingPhase 2
PRECISION-E2: Teverelix in Women With Endometriosis - Pharmacodynamics, Safety and Precision Medicine Correlates
Endometriosis Pelvic, Endometriosis Ovaries
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
AbbVie Inc. study director
AbbVie
37 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing elagolix and estradiol/norethindrone acetate (E2/NETA) in people with Endometriosis, run by AbbVie at 37 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 7 inclusion and 11 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 27 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 27 Nov 2019). 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?
Proportion of Responders at Month 6 Based on DYS Pain Scale (time frame: Month 6). The study lists 2 primary outcomes in all. 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 27 Nov 2019.