Clinical trial NCT00973973: Efficacy and Safety Study of Elagolix in Women With Endometriosis
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of NBI-56418 Sodium in Subjects With Endometriosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Endometriosis, Pain
Keywords: Pelvic Pain,NBI-56418,Endometriosis
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2009-09-09
Enrollment: 137
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: The purpose of this study is to evaluate elagolix (NBI-56418) compared to placebo for its effects on endometriosis related pelvic pain and its safety.

Detailed description: Participants were randomized (1:1) to 150 mg elagolix once daily or placebo once daily for the first 8 weeks of the study. Following 8 weeks of dosing, participants continued in the study for an additional 16 weeks in an open-label phase where all participants still enrolled in the study received 150 mg elagolix once daily.

There was no pre-specified primary efficacy end point for this study as there is no single key efficacy outcome measure in this exploratory Phase 2 study. However, the efficacy measures of primary interest included the daily assessment of dysmenorrhea, non-menstrual pelvic pain and dyspareunia on a 4-point scale (0 = none, 1 = mild, 2 = moderate, 3 = severe) using an e-Diary.

Eligibility criteria: Inclusion Criteria:

* Be female, aged 18 to 49 years, inclusive.
* Have moderate to severe pelvic pain due to endometriosis.
* Have a history of regular menstrual cycles.
* Have been surgically (laparoscopy or laparotomy) diagnosed with endometriosis within 8 years of the start of screening.
* Have a Body Mass Index (BMI) of 18 to 36 kg/m², inclusive.
* Agree to use two forms of non-hormonal contraception during the study.

Exclusion Criteria:

* Are currently receiving gonadotropin-releasing hormone (GnRH) agonist, a GnRH antagonist other than NBI-56418, or danazol or have received any of these agents within 6 months of the start of screening.
* Are currently receiving subcutaneous medroxyprogesterone acetate (DMPA-SC) or intramuscular medroxyprogesterone acetate (DMPA-IM) or have received any of these agents within 3 months of the start of screening.
* Are currently using hormonal contraception or other forms of hormonal therapy or received such treatment within the last month.
* Have had surgery for endometriosis within the last month.
* Have had a hysterectomy or bilateral oophorectomy.
* Are using systemic steroids on a chronic or regular basis within 3 months.
* Have uterine fibroids ≥ 3 cm in diameter.
* Have pelvic pain that is not caused by endometriosis.
* Have unstable medical condition or chronic disease.
* Have been pregnant within the last six months.
* Currently breast feeding.

Primary outcomes:
measure: Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Double-blind Treatment Phase
timeFrame: Baseline and Weeks 4 and 8
measure: Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Open-label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Double-Blind Treatment Phase
timeFrame: Baseline and weeks 4 and 8
measure: Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Open-label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline in the Monthly Mean Cumulative Pain Score During the Double-Blind Treatment Phase
timeFrame: Baseline and weeks 4 and 8
measure: Change From Baseline in the Monthly Mean Cumulative Pain Score During the Open-label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline in the Monthly Mean Dyspareunia Score During the Double-Blind Treatment Phase
timeFrame: Baseline and weeks 4 and 8
measure: Change From Baseline in the Monthly Mean Dyspareunia Score During the Open-label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Secondary outcomes:
measure: Percentage of Participants With a Response in Monthly Mean Dysmenorrhea Score at Week 8
timeFrame: Baseline and Week 8
measure: Percentage of Participants With a Response in Monthly Mean Non-menstrual Pelvic Pain Score at Week 8
timeFrame: Baseline and Week 8
measure: Percentage of Participants With a Response in Monthly Mean Cumulative Pain Score at Week 8
timeFrame: Baseline and Week 8
measure: Percentage of Participants With a Response in Monthly Mean Dyspareunia Score at Week 8
timeFrame: Baseline and Week 8
measure: Change From Baseline in the Percentage of Days of Any Analgesic Use During the Double-Blind Treatment Phase
timeFrame: Baseline and Weeks 4 and 8
measure: Change From Baseline in the Percentage of Days of Any Analgesic Use During the Open-Label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Double-Blind Treatment Phase
timeFrame: Baseline and Weeks 4 and 8
measure: Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Open-Label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Double-Blind Treatment Phase
timeFrame: Baseline and Weeks 4 and 8
measure: Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Open-Label and Posttreatment Phases
timeFrame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline to the End of the Double-blind Treatment Phase in Composite Pelvic Signs and Symptoms Score (CPSSS) Total Score and Component Scores
timeFrame: Baseline and Week 8
measure: Change From Baseline to the End of the Open-label Treatment Phase in Composite Pelvic Signs and Symptoms Score (CPSSS) Total Score and Component Scores
timeFrame: Baseline and week 24
measure: Patient Global Impression of Change During the Double-blind Treatment Phase
timeFrame: Weeks 4 and 8
measure: Patient Global Impression of Change During the Open-Label and Posttreatment Phases
timeFrame: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Double-blind Treatment Phase
timeFrame: Weeks 4 and 8
measure: Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Open-label Treatment Phase
timeFrame: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
measure: Change From Baseline to the End of the Double-blind Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
timeFrame: Baseline and week 8
measure: Change From Baseline to the End of the Open-label Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
timeFrame: Baseline and week 24
measure: Percentage of Days With Uterine Bleeding During the Double- Blind Treatment Phase
timeFrame: Screening (8 weeks prior to day 1) and the double-blind treatment phase (Weeks 1-8)
measure: Number of Days to First Posttreatment Menses
timeFrame: From last day of study drug up to 6 weeks after the last dose.
Other outcomes:
Locations:
Study officials:
affiliation: AbbVie
name: AbbVie Inc.
role: STUDY_DIRECTOR
References: