A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Elagolix in Subjects With Moderate to Severe Endometriosis-Associated Pain
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 815
- Primary Endpoint
- Percentage of Responders at Month 3 Based on Daily Assessment of Dysmenorrhea (DYS)
Study Overview
Brief Summary
A randomized study evaluating the safety and efficacy of elagolix in the management of moderate to severe endometriosis-associated pain in adult premenopausal female subjects.
Detailed Description
The study consists of 4 periods: 1) Washout Period (if applicable); 2) a Screening Period of up to 100 days prior to first dose; 3) a 6 month Treatment Period; and 4) a Post treatment Follow-up Period of up to 12 months (if applicable). An electronic diary will be dispensed and training provided to record endometriosis-associated pain, uterine bleeding, and analgesic medication use for endometriosis-associated pain on a daily basis. Pregnancy testing will be performed monthly throughout the study. Subjects will be required to use nonhormonal dual contraception during the study, and will be counseled on appropriate and effective forms of birth control to promote pregnancy prevention.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 49 Years (Adult)
- Sex
- Female
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Premenopausal female, between 18 and 49 years of age, inclusive, at the time of signing consent.
- •Clinical diagnosis of endometriosis (laparoscopy or laparotomy) performed within 10 years of entry into the Washout Period.
- •Agrees to use required birth control methods during the entire length of participation in the study.
- •Subject has a Composite Pelvic Signs and Symptoms Score total score of 6 or greater at Screening with a score of at least 2 for dysmenorrhea AND at least 2 for non-menstrual pelvic pain.
- •Subjects must have at least two regular menstrual cycles with an interval of 24-38 days within the Screening Period, prior to Day 1.
Exclusion Criteria
- •Subject is pregnant or breast feeding or is planning a pregnancy within the next 24 months or is less than 6 months postpartum, post-abortion, or post-pregnancy at the time of entry into the Screening Period.
- •Subject has a history of previous non-response to gonadotropin-releasing hormone (GnRH) agonists, GnRH antagonists, Depot medroxyprogesterone acetate, or aromatase inhibitors as assessed by subject report of no improvement in dysmenorrhea or non-menstrual pelvic pain.
- •Subject has chronic pelvic pain that is not caused by endometriosis that requires chronic analgesic or other chronic therapy, or that would interfere with the assessment of endometriosis related pain.
- •Clinically significant gynecologic condition identified on Screening transvaginal ultrasound or endometrial biopsy.
- •Subject has a history of osteoporosis or other metabolic bone disease.
- •Subject has a current history of undiagnosed abnormal uterine bleeding.
Arms & Interventions
Placebo
Placebo twice daily (BID) for the 6-month Treatment Period
Intervention: placebo (Other)
Elagolix 150 mg QD
Elagolix 150 mg once daily (QD) for the 6-month Treatment Period
Intervention: Elagolix (Drug)
Elagolix 200 mg BID
Elagolix 200 mg BID for the 6-month Treatment Period
Intervention: Elagolix (Drug)
Outcomes
Primary Outcomes
Percentage of Responders at Month 3 Based on Daily Assessment of Dysmenorrhea (DYS)
Time Frame: At Month 3 of the Treatment Period
The DYS pain scale ranges from 0 (none) to 3 (severe) as recorded in a daily electronic diary. The criteria for defining a participant as a responder included a reduction of -0.85 or greater from Baseline in DYS pain as well as no increased rescue analgesic use for endometriosis-associated pain.
Percentage of Responders at Month 3 Based on Daily Assessment of Non-Menstrual Pelvic Pain (NMPP)
Time Frame: At Month 3 of Treatment Period
The NMPP pain scale ranges from 0 (none) to 3 (severe) as recorded in a daily electronic diary. The criteria for defining a participant as a responder included a reduction of -0.43 or greater from Baseline in NMPP as well as no increased rescue analgesic use for endometriosis-associated pain.
Secondary Outcomes
- Change From Baseline to Month 6 in DYS(Baseline, Month 6 of Treatment Period)
- Change From Baseline to Month 6 in NMPP(Baseline, Month 6 of Treatment Period)
- Percent Change From Baseline to Each Month in Mean Pain Score for DYS(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Percent Change From Baseline to Each Month in the Mean Pain Score for NMPP(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Number of Participants With Emergency Room/Outpatient Procedures During the Treatment Period, by Type(Up to Month 6 of Treatment Period)
- Change From Baseline to Each Month, Except Month 3, in the Mean Pain Score of DYSP(Baseline, Months 1, 2, 4, 5, 6 of Treatment Period)
- Change From Baseline to Month 3 in Numeric Rating Scale (NRS) Scores(Baseline, Month 3 of the Treatment Period)
- Change From Baseline to Month 3 in Analgesic Use Across Both Classes of Rescue Analgesics(Baseline, Month 3 of Treatment Period)
- Change From Baseline to Month 6 in Analgesic Use Across Both Classes of Rescue Analgesics(Baseline, Month 6 of Treatment Period)
- Change From Baseline to Month 3 in Dyspareunia (DYSP)(Baseline, Month 3 of Treatment Period)
- Change From Baseline to Month 3 in Use of Narcotic Class of Medication (Opioids)(Baseline, Month 3 of Treatment Period)
- Percentage of Responders for Each Month, Except Month 3, in DYS(Months 1, 2, 4, 5, 6 of the Treatment Period)
- Percentage of Responders for Each Month, Except Month 3, in NMPP(Months 1, 2, 4, 5, 6 of the Treatment Period)
- Percentage of Responders at Each Month for DYSP(Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month in Health Related Productivity Questionnaire (HRPQ): Number of Hours of Work Lost From Workplace Due to Absenteeism(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month in HRPQ: Number of Hours of Work Lost From Household Due to Absenteeism(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month, Except Month 6, in Mean Pain Score for DYS(Baseline (Prior to administering study drug), Months 1, 2, 3, 4, 5 of Treatment Period)
- Change From Baseline to Each Month, Except Month 6, in Mean Pain Score for NMPP(Baseline, Months 1, 2, 3, 4, 5 of Treatment Period)
- Change From Baseline to Each Month, Except Months 3 and 6, in Analgesic Use Across Both Classes of Rescue Analgesics(Baseline, Months 1, 2, 4, 5)
- Patient Global Impression of Change (PGIC) Questionnaire(Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month, Except Month 3, in NRS Scores(Baseline, Months 1, 2, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Scheduled Assessment in the Pain Domain of Endometriosis Health Profile-30 (EHP-30) Questionnaire Scores(Baseline, Months 1, 3, 6 of Treatment Period)
- Change From Baseline to Each Scheduled Assessment in the Sexual Intercourse Domain of EHP-30 Questionnaire Scores(Baseline, Months 1, 3, 6 of Treatment Period)
- Change From Baseline to Each Month in HRPQ: Number of Hours of Work Lost From Workplace Due to Presenteeism(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month in HRPQ: Number of Hours of Work Lost From Household Due to Presenteeism(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month in HRPQ: Total (Absenteeism and Presenteeism) Number of Hours of Work Lost From Workplace(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Change From Baseline to Each Month in HRPQ: Total (Absenteeism and Presenteeism) Number of Hours of Work Lost From Household(Baseline, Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Number of Participants With Endometriosis-Related Non-Study Health Visits During the Treatment Period(Months 1, 2, 3, 4, 5, 6 of Treatment Period)
- Number of Days of Hospitalization(Up to Month 6 of Treatment Period)