Clinical trial NCT03343067: A Study to Evaluate Safety and Efficacy of Elagolix in Participants With Endometriosis With Associated Moderate to Severe Pain
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Endometriosis
Keywords: Endometriosis associated pain
Elagolix
Non-menstrual pelvic pain (NMPP)
Dysmenorrhea (DYS)
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2017-11-17
Enrollment: 11
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 49
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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:

  1. At least 2 days of "moderate" or "severe" DYS, AND either,
  2. At least 2 days of "moderate" or "severe" NMPP and an average NMPP score of at least 1.0, OR
  3. At least 4 days of "moderate" or "severe" NMPP and an average NMPP score of at least 0.5.

Exclusion Criteria:

* 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).
* Participant has either

  1. 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
  2. a clinically significant medical condition that is anticipated to required intervention during the course of study participation (e.g., anticipated major elective surgery) OR
  3. 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

Primary outcomes:
measure: Proportion of Responders at Month 6 Based on DYS Pain Scale
timeFrame: Month 6
measure: Proportion of Responders at Month 6 Based on NMPP Pain Scale
timeFrame: Month 6
Secondary outcomes:
measure: Change From Baseline in DYS at Month 6
timeFrame: Month 0 (baseline), Month 6
measure: Change From Baseline in NMPP at Month 6
timeFrame: Month 0 (baseline), Month 6
measure: Change From Baseline in Dyspareunia at Month 6
timeFrame: Month 0 (baseline), Month 6
measure: Change From Baseline in Daily Diary Endometriosis-Associated Pain Score Via Numeric Rating Scale (NRS) at Month 6
timeFrame: Month 0 (baseline), Month 6
measure: Change From Baseline in Rescue Analgesic Use Across Both Classes of Rescue Analgesics (NSAIDs/Opioids) at Month 6
timeFrame: Month 0 (baseline), Month 6
measure: 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
timeFrame: Month 6
measure: Proportion of Responders Over Time
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Proportion of Responders Over Time (Not Taking Into Consideration of Analgesic Use)
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Change From Baseline Over Time in Monthly Average DYS Pain Score
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Change From Baseline Over Time in Monthly Average NMPP Pain Score
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Change From Baseline Over Time in Monthly Average Dyspareunia Pain Score
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Change From Baseline Over Time in Monthly Average Daily Diary Endometriosis-Associated Pain Score Via NRS
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Percent Change From Baseline to Each Month During the Treatment Period for DYS
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Percent Change From Baseline to Each Month During the Treatment Period for NMPP
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Percent Change From Baseline to Each Month During the Treatment Period for Dyspareunia
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Percent Change From Baseline to Each Month During the Treatment Period for Daily Diary Endometriosis-Associated Pain Score Via NRS
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Change From Baseline Over Time in Daily Rescue Analgesic Use Across Both Classes of Rescue Analgesics
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Number of Analgesic Use Responders and Non-Responders Over Time
timeFrame: Months 1, 2, 3, 4, 5, 6
measure: Patient Global Impression of Change (PGIC) Scores Over Time
timeFrame: Months 1, 2, 3, 4, 5, 6
measure: Overall Endometriosis-Associated Pain Via NRS (7-Day Recall) Scores Over Time
timeFrame: Month 0 (baseline), Months 1, 2, 3, 4, 5, 6
measure: Endometriosis Health Profile-30 (EHP-30) Scores Over Time: Pain
timeFrame: Month 0 (baseline), Months 3, 6
measure: EHP-30 Scores Over Time: Control and Powerlessness
timeFrame: Month 0 (baseline), Months 3, 6
measure: EHP-30 Scores Over Time: Emotional Well-Being
timeFrame: Month 0 (baseline), Months 3, 6
measure: EHP-30 Scores Over Time: Social Support
timeFrame: Month 0 (baseline), Months 3, 6
measure: EHP-30 Scores Over Time: Self-Image
timeFrame: Month 0 (baseline), Months 3, 6
measure: EHP-30 Scores Over Time: Sexual Intercourse
timeFrame: Month 0 (baseline), Months 3, 6
measure: EuroQol-5D 5 Level (EQ-5D-5L) Scores Over Time: Mobility
timeFrame: Month 0 (baseline), Months 3, 6
measure: EQ-5D-5L Scores Over Time: Self-Care
timeFrame: Month 0 (baseline), Months 3, 6
measure: EQ-5D-5L Scores Over Time: Usual Activities
timeFrame: Month 0 (baseline), Months 3, 6
measure: EQ-5D-5L Scores Over Time: Pain/Discomfort
timeFrame: Month 0 (baseline), Months 3, 6
measure: EQ-5D-5L Scores Over Time: Anxiety/Depression
timeFrame: Month 0 (baseline), Months 3, 6
measure: EQ-5D-5L VAS Scores Over Time: Health Today
timeFrame: Month 0 (baseline), Months 3, 6
measure: Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) Scores Over Time: Percent Work Missed Due to Problem
timeFrame: Month 0 (baseline), Month 6
measure: WPAI:SHP Scores Over Time: Percent Impairment While Working Due to Problem
timeFrame: Month 0 (baseline), Month 6
measure: WPAI:SHP Scores Over Time: Percent Overall Work Impairment Due to Problem
timeFrame: Month 0 (baseline), Month 6
measure: WPAI:SHP Scores Over Time: Percent Activity Impairment Due to Problem
timeFrame: Month 0 (baseline), Month 6
measure: Health Endometriosis Treatment Satisfaction Questionnaire (ETSQ) Scores Over Time
timeFrame: Month 0 (baseline), Months 3, 6
measure: PROMIS Fatigue Short Form 6a Scores Over Time
timeFrame: Month 0 (baseline), Month 6
Other outcomes:
Locations:
city: Anniston
country: United States
facility: Pinnacle Research Group /ID# 202016
state: Alabama
city: Tucson
country: United States
facility: Noble Clinical Research /ID# 170628
state: Arizona
city: Little Rock
country: United States
facility: Lynn Institute of the Ozarks /ID# 165052
state: Arkansas
city: Garden Grove
country: United States
facility: SC Clinical Research /ID# 165049
state: California
city: Huntington Beach
country: United States
facility: Marvel Clinical Research /ID# 169633
state: California
city: Santa Ana
country: United States
facility: Health care Affiliates Medical Group /ID# 165048
state: California
city: Wheat Ridge
country: United States
facility: Western States Clinical Res /ID# 169809
state: Colorado
city: Bradenton
country: United States
facility: Nova Clinical Research, LLC /ID# 202227
state: Florida
city: DeLand
country: United States
facility: Midland Florida Clinical Research Center /ID# 201327
state: Florida
city: Gainesville
country: United States
facility: Southeastern Integrated Med /ID# 203109
state: Florida
city: Jacksonville
country: United States
facility: Care Partners Clinical Researc /ID# 168395
state: Florida
city: Miami
country: United States
facility: LCC Medical Research Institute /ID# 168888
state: Florida
city: Orange City
country: United States
facility: A Premier Clinical Research of Florida, LLC /ID# 201887
state: Florida
city: Tampa
country: United States
facility: GCP Clinical Research, LLC /ID# 169774
state: Florida
city: West Palm Beach
country: United States
facility: Triple O Research Institute /ID# 201128
state: Florida
city: Blackfoot
country: United States
facility: Bingham Memorial Hospital /ID# 170110
state: Idaho
city: Idaho Falls
country: United States
facility: Leavitt Womens Healthcare /ID# 169495
state: Idaho
city: Chicago
country: United States
facility: Quad Clinical Research, LLC /ID# 168294
state: Illinois
city: Chicago
country: United States
facility: Quad Clinical Research, LLC /ID# 170629
state: Illinois
city: Newburgh
country: United States
facility: Women's Health Care, PC /ID# 165033
state: Indiana
city: New Orleans
country: United States
facility: Delricht Research /ID# 200219
state: Louisiana
city: Saint Louis
country: United States
facility: Quad Clinical Research, LLC /ID# 200180
state: Missouri
city: Billings
country: United States
facility: Montana Health Research Inst /ID# 170624
state: Montana
city: Las Vegas
country: United States
facility: Excel Clinical Research /ID# 170620
state: Nevada
city: Manhasset
country: United States
facility: Northwell health system /ID# 200162
state: New York
city: Akron
country: United States
facility: Radiant Research, Inc /ID# 200045
state: Ohio
city: Columbus
country: United States
facility: Central Ohio Clinical Research /ID# 170750
state: Ohio
city: Columbus
country: United States
facility: Optimed Research /ID# 167642
state: Ohio
city: Greenville
country: United States
facility: VitaLink Research /ID# 168401
state: South Carolina
city: Greenville
country: United States
facility: VitaLink Research /ID# 170625
state: South Carolina
city: Spartanburg
country: United States
facility: Fusion Clinical Research of Spartanburg /ID# 200010
state: South Carolina
city: Houston
country: United States
facility: Centex Studies, Inc /ID# 169897
state: Texas
city: Houston
country: United States
facility: America's Adv. Wellness Center /ID# 170005
state: Texas
city: Plano
country: United States
facility: ClinRx Research, LLC /ID# 201189
state: Texas
city: San Antonio
country: United States
facility: Progressive Clinical Research /ID# 205565
state: Texas
city: Norfolk
country: United States
facility: Eastern Virginia Med School /ID# 165016
state: Virginia
city: Seattle
country: United States
facility: Seattle Reproductive Medicine /ID# 171079
state: Washington
Study officials:
affiliation: AbbVie
name: AbbVie Inc.
role: STUDY_DIRECTOR
References: