Endometriosis, PainPhase 2NCT00973973

Efficacy and Safety Study of Elagolix in Women With Endometriosis

A Phase 2 study testing Elagolix in people with Endometriosis, Pain, run by AbbVie.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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. Read more about this study

Who is it for?: Ages 18 to 49 years, women only
Conditions the study is for: Endometriosis, Pain
What is being tested?: Elagolix
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 137 participants
Where: No sites listed yet
Study dates: 12 Oct 2009to 22 Sep 2010
Phase: Phase 2
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

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.

Who cannot join

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.

Show all 11Show fewer

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.

About this study

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

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.

Read the 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.

Conditions: Endometriosis, Pain
Being tested: Placebo, Elagolix
Keywords: Pelvic Pain,NBI-56418,Endometriosis
Medical subject headings: Endometriosis, Pain

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PlaceboDrug
Matching placebo tablets taken orally once a day
ElagolixDrug
Also called NBI-56418, Orilissa™
Immediate release (IR) tablets taken orally once a day

Study groups in NCT00973973
Study group	Role	What they receive
Elagolix 150 mg	Experimental	Participants received 150 mg elagolix orally once a day for 8 weeks during the double-blind treatment period and continued to receive 150 mg elagolix for 16 additional weeks during the open-label treatment period. Receives: Drug: Elagolix
Placebo	Placebo comparator	Participants received placebo orally once a day for 8 weeks during the double-blind treatment period and switched to receive 150 mg elagolix for 16 weeks during the open-label treatment period. Receives: Drug: Placebo; Drug: Elagolix

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Double-blind Treatment Phase
Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day of their period in an electronic diary (e-Diary) according to the following response options:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean dysmenorrhea score is the average of the daily values reported during the 4 weeks prior to each visit.
Measured: Baseline and Weeks 4 and 8
Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Open-label and Posttreatment Phases
Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day of their period in an electronic diary (e-Diary) according to the following response options:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean dysmenorrhea score is the average of the daily values reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Double-Blind Treatment Phase
Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time each day they were not having their period in an e-Diary according to the following response options:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior to each visit.
Measured: Baseline and weeks 4 and 8
Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Open-label and Posttreatment Phases
Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time each day they were not having their period in an e-Diary according to the following response options:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Monthly Mean Cumulative Pain Score During the Double-Blind Treatment Phase
Participants assessed dysmenorrhea or non-menstrual pelvic pain at approximately the same time each day in an e-Diary according to the following:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean cumulative pain score is the average of the daily values for all days (menstrual and non-menstrual) reported during the 4 weeks prior to each visit.
Measured: Baseline and weeks 4 and 8
Change From Baseline in the Monthly Mean Cumulative Pain Score During the Open-label and Posttreatment Phases
Participants assessed dysmenorrhea or non-menstrual pelvic pain at approximately the same time each day in an e-Diary according to the following:
- 0: No discomfort
- 1: Mild discomfort, I was easily able to do the things I usually do
- 2: Moderate discomfort or pain making it difficult to do some of the things I usually do
- 3: Severe pain making it difficult to do the things I usually do
The monthly mean cumulative pain score is the average of the daily values for all days (menstrual and non-menstrual) reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Monthly Mean Dyspareunia Score During the Double-Blind Treatment Phase
Participants assessed their dyspareunia (pain during sexual intercourse) at approximately the same time every day in an e-Diary according to the following response options:
- 0 = Absent; No discomfort during sexual intercourse
- 1 = Mild; I was able to tolerate the discomfort during sexual intercourse
- 2 = Moderate; Intercourse was interrupted due to pain
- 3 = Severe; I avoided intercourse because of pain
- Does not apply; I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse
The monthly mean dyspareunia score is the average of the daily values reported during the 4 weeks prior to each visit. Responses of "does not apply" were not included in the calculations.
Measured: Baseline and weeks 4 and 8
Change From Baseline in the Monthly Mean Dyspareunia Score During the Open-label and Posttreatment Phases
Participants assessed their dyspareunia (pain during sexual intercourse) at approximately the same time every day in an e-Diary according to the following response options:
- 0: Absent; No discomfort during sexual intercourse
- 1: Mild; I was able to tolerate the discomfort during sexual intercourse
- 2: Moderate; Intercourse was interrupted due to pain
- 3: Severe; I avoided intercourse because of pain
- Does not apply; I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse
The monthly mean dyspareunia score is the average of the daily values reported during the 4 weeks prior to each visit, except for week 30 which is based on 6 weeks of data. Responses of "does not apply" were not included in the calculations.
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)

Secondary outcomes (20)

Percentage of Participants With a Response in Monthly Mean Dysmenorrhea Score at Week 8
Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day of their period in an e-Diary according to the following response options:
- 0: No discomfort
- 1: Mild discomfort but I was easily able to do the things I usually do
- 2: Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3: Severe pain that made it difficult to do the things I usually do.
The monthly mean dysmenorrhea score is the average of the daily values reported during the 4 weeks prior to each time point.
Response was defined as the percentage of participants with a percent decrease from baseline in the week 8 monthly mean score that was greater than or equal to each specified threshold value (10% through 90% in steps of 10%).
Measured: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Non-menstrual Pelvic Pain Score at Week 8
Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time each day they were not having their period in an e-Diary according to the following response options:
- 0 = No discomfort
- 1 = Mild discomfort but I was easily able to do the things I usually do
- 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do
- 3 = Severe pain that made it difficult to do the things I usually do.
The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior to each time point.
Response is defined as the percentage of participants with a percent decrease from baseline in the week 8 monthly mean score that was greater than or equal to each specified threshold value (10% through 90% in steps of 10%).
Measured: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Cumulative Pain Score at Week 8
Participants assessed dysmenorrhea or non-menstrual pelvic pain at approximately the same time every day in an e-Diary according to the following:
- 0 = No discomfort
- 1 = Mild discomfort
- 2 = Moderate discomfort or pain
- 3 = Severe pain
The monthly mean cumulative pain score is the average of the daily values for all days (menstrual and non-menstrual) in the 4 weeks prior to each time point.
Response is the percentage of participants with a percent decrease from baseline in the week 8 monthly mean score that was greater than or equal to each specified threshold value (10% through 90% in steps of 10%).
Measured: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Dyspareunia Score at Week 8
Participants assessed their dyspareunia (pain during sexual intercourse) at approximately the same time every day in an e-Diary according to the following response options:
- 0 = Absent; No discomfort during sexual intercourse
- 1 = Mild; I was able to tolerate the discomfort during sexual intercourse
- 2 = Moderate; Intercourse was interrupted due to pain
- 3 = Severe; I avoided intercourse because of pain
- Does not apply; I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse
The monthly mean dyspareunia score is the average of the daily values reported during the 4 weeks prior to each time point.
Response is defined as the percentage of participants with a percent decrease from baseline in the week 8 monthly mean score that was greater than or equal to each specified threshold value (10% through 90% in steps of 10%).
Measured: Baseline and Week 8
Change From Baseline in the Percentage of Days of Any Analgesic Use During the Double-Blind Treatment Phase
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of any analgesic use is defined as the number of days in the 4 weeks prior to each study visit that the participant reported the use of an analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Any Analgesic Use During the Open-Label and Posttreatment Phases
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of any analgesic use is defined as the number of days in the 4 weeks prior to each study visit (except for week 30 which is based on 6 weeks of data) that the participant reported the use of an analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Double-Blind Treatment Phase
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of prescription analgesic use is defined as the number of days in the 4 weeks prior to each study visit that the participant reported the use of a prescription analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Open-Label and Posttreatment Phases
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of prescription analgesic use is defined as the number of days in the 4 weeks prior to each study visit (except for week 30 which is based on 6 weeks of data) that the participant reported the use of a prescription analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Double-Blind Treatment Phase
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of narcotic analgesic use is defined as the number of days in the 4 weeks prior to each study visit that the participant reported the use of a narcotic analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Open-Label and Posttreatment Phases
The daily use of endometriosis analgesics was reported by participants daily using the e-Diary. Participants reported whether the medication was over-the-counter (OTC) or prescription, and, if prescription, whether the medication was a narcotic.
The percentage of days of narcotic analgesic use is defined as the number of days in the 4 weeks prior to each study visit (except for week 30 which is based on 6 weeks of data) that the participant reported the use of a narcotic analgesic, divided by the number of study days in the interval that the participant provided an e-Diary report regarding the use of endometriosis analgesics (including a response of "none").
Measured: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
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
The CPSSS consists of 5 components that address dysmenorrhea, dyspareunia, non-menstrual pelvic pain, pelvic tenderness, and pelvic induration. Each component was scored on a scale of 0 to 3 (0 = absent, 1 = mild, 2 = moderate, and 3 = severe).
Dysmenorrhea, dyspareunia, and non-menstrual pelvic pain scores are based on the participant's assessment of symptoms during the past 28 days; pelvic tenderness and induration were assessed by the investigator based on findings associated with a pelvic examination.
The total CPSSS has a maximum possible value of 15 (total score range: 0 to 15, where a lower score indicates less signs and symptoms of endometriosis or better functioning). Individual component scores range from 0 (absent) to 3 (severe).
Measured: Baseline and Week 8
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
The CPSSS consists of 5 components that address dysmenorrhea, dyspareunia, non-menstrual pelvic pain, pelvic tenderness, and pelvic induration. Each component was scored on a scale of 0 to 3 (0 = absent, 1 = mild, 2 = moderate, and 3 = severe).
Dysmenorrhea, dyspareunia, and non-menstrual pelvic pain scores are based on the participant's assessment of symptoms during the past 28 days; pelvic tenderness and induration were assessed by the investigator based on findings associated with a pelvic examination.
The total CPSSS has a maximum possible value of 15 (total score range: 0 to 15, where a lower score indicates less signs and symptoms of endometriosis or better functioning). Individual component scores range from 0 (absent) to 3 (severe).
Measured: Baseline and week 24
Patient Global Impression of Change During the Double-blind Treatment Phase
The Patient Global Impression of Change (PGIC) is a questionnaire-based assessment of the change in endometriosis pain since the initiation of study drug. The participant was asked to select from one of seven response categories:
1. Very Much Improved
2. Much Improved
3. Minimally Improved
4. Not Changed
5. Minimally Worse
6. Much Worse
7. Very Much Worse
Measured: Weeks 4 and 8
Patient Global Impression of Change During the Open-Label and Posttreatment Phases
The Patient Global Impression of Change (PGIC) is a questionnaire-based assessment of the change in endometriosis pain since the initiation of study drug. The participant was asked to select from one of seven response categories:
1. Very Much Improved
2. Much Improved
3. Minimally Improved
4. Not Changed
5. Minimally Worse
6. Much Worse
7. Very Much Worse
Measured: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Double-blind Treatment Phase
The Patient Global Impression of Change (PGIC) is a questionnaire-based assessment of the change in endometriosis pain since the initiation of study drug. The participant was asked to select from one of seven response categories:
1. Very Much Improved
2. Much Improved
3. Minimally Improved
4. Not Changed
5. Minimally Worse
6. Much Worse
7. Very Much Worse
Measured: Weeks 4 and 8
Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Open-label Treatment Phase
The Patient Global Impression of Change (PGIC) is a questionnaire-based assessment of the change in endometriosis pain since the initiation of study drug. The participant was asked to select from one of seven response categories:
1. Very Much Improved
2. Much Improved
3. Minimally Improved
4. Not Changed
5. Minimally Worse
6. Much Worse
7. Very Much Worse
Measured: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline to the End of the Double-blind Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
The EHP-5 is an instrument to measure health-related quality of life in women with endometriosis. The EHP-5 consists of two parts:
- A core questionnaire consisting of five questions that measure the impact of endometriosis in areas of pain, control and powerlessness, emotional well-being, social support, and self-image with five response categories for each item (Never, Rarely, Sometimes, Often, Always)
- A supplemental questionnaire consisting of six additional questions which assess the impact of endometriosis on the areas of work, relationship with children, sexual intercourse, feelings about the medical profession, treatment, and infertility with the same five response categories plus an additional response category of Not Relevant which was not scored.
The scores associated with each possible outcome category are as follows: never (0), rarely (25), sometimes (50), often (75), and always (100). A negative change from baseline score indicates improvement in quality of life.
Measured: Baseline and week 8
Change From Baseline to the End of the Open-label Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
The EHP-5 is an instrument to measure health-related quality of life in women with endometriosis. The EHP-5 consists of two parts:
- A core questionnaire consisting of five questions that measure the impact of endometriosis on areas of pain, control and powerlessness, emotional well-being, social support, and self-image with five response categories for each item (Never, Rarely, Sometimes, Often, Always)
- A supplemental questionnaire consisting of six additional questions which assess the impact of endometriosis on areas of work, relationship with children, sexual intercourse, feelings about the medical profession, treatment, and infertility with the same five response categories plus an additional response category of Not Relevant which was not scored.
The scores associated with each possible outcome category are as follows: never (0), rarely (25), sometimes (50), often (75), and always (100). A negative change from baseline score indicates improvement in quality of life.
Measured: Baseline and week 24
Percentage of Days With Uterine Bleeding During the Double- Blind Treatment Phase
Uterine bleeding was reported daily by participants during the study using the e-Diary.
The percentage of days a participant reported any bleeding was calculated as the total number of days the participant reported any bleeding ( light, moderate, or heavy) divided by the total number of days the participant had a non-missing e-Diary report of vaginal bleeding in the phase.
Measured: Screening (8 weeks prior to day 1) and the double-blind treatment phase (Weeks 1-8)
Number of Days to First Posttreatment Menses
Defined as the number of days from the last dose of study drug until the start date of the first post-treatment menses.
Measured: From last day of study drug up to 6 weeks after the last dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

12 Oct 200922 Sep 2010

First posted9 Sep 2009 (estimated)
Study start12 Oct 2009
Primary completion22 Sep 2010
Study completion22 Sep 2010

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 Endometriosis, PainStudies 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 137 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00973973
Sponsor's protocol ID: NBI-56418-0901
Status last verified: Apr 2018
First submitted: 7 Sep 2009
Last updated: 26 Sep 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

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Endometriosis
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Endometriosis Pelvic, Endometriosis Ovaries
Recruiting
Evaluating the Feasibility of elleFA App Implementation
Endometriosis
Recruiting
Impact of Food Supplementation on Quality of Life in Patients With Endometriosis and Pelvic Pain.
Endometriosis (Diagnosis)
RecruitingObservational study
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All Endometriosis, Pain trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00973973
Acronym / sponsor ID: NBI-56418-0901
Conditions: Endometriosis, Pain
Keywords: Pelvic Pain,NBI-56418,Endometriosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 137 participants (actual)
Lead sponsor: AbbVieIndustry (pharma, biotech, devices)
Study start: 12 Oct 2009 (actual)
Primary completion: 22 Sep 2010 (actual)
Study completion: 22 Sep 2010 (actual)
First posted: 9 Sep 2009 (estimated)
Results: Posted 26 Sep 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Elagolix 150 mg	Experimental	Drug: ElagolixParticipants received 150 mg elagolix orally once a day for 8 weeks during the double-blind treatment period and continued to receive 150 mg elagolix for 16 additional weeks during the open-label treatment period.
Placebo	Placebo comparator	Drug: Placebo, Drug: ElagolixParticipants received placebo orally once a day for 8 weeks during the double-blind treatment period and switched to receive 150 mg elagolix for 16 weeks during the open-label treatment period.

Who may qualify

Ages: 18 to 49 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (6 listed)

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.

Key exclusion criteria (11 listed)

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.

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
PlaceboMatching placebo tablets taken orally once a day	Drug	Placebo
ElagolixAlso: NBI-56418, Orilissa™Immediate release (IR) tablets taken orally once a day	Drug	Elagolix 150 mg, Placebo

Primary outcome

Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Double-blind Treatment Phase
Time frame: Baseline and Weeks 4 and 8
Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day of their period in an electronic diary (e-Diary) according to the following response options: * 0 = No discomfort * 1 = Mild discomfort but I was easily able to do the things I usually do * 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do * 3 = Severe pain that made it difficult to do the things I usually do. The monthly mean dysmenorrhea score is the average of the daily values reported during the 4 weeks prior to each visit.
Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Open-label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day of their period in an electronic diary (e-Diary) according to the following response options: * 0 = No discomfort * 1 = Mild discomfort but I was easily able to do the things I usually do * 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do * 3 = Severe pain that made it difficult to do the things I usually do. The monthly mean dysmenorrhea score is the average of the daily values reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Double-Blind Treatment Phase
Time frame: Baseline and weeks 4 and 8
Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time each day they were not having their period in an e-Diary according to the following response options: * 0 = No discomfort * 1 = Mild discomfort but I was easily able to do the things I usually do * 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do * 3 = Severe pain that made it difficult to do the things I usually do. The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior to each visit.
Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Open-label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time each day they were not having their period in an e-Diary according to the following response options: * 0 = No discomfort * 1 = Mild discomfort but I was easily able to do the things I usually do * 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do * 3 = Severe pain that made it difficult to do the things I usually do. The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Change From Baseline in the Monthly Mean Cumulative Pain Score During the Double-Blind Treatment Phase
Time frame: Baseline and weeks 4 and 8
Participants assessed dysmenorrhea or non-menstrual pelvic pain at approximately the same time each day in an e-Diary according to the following: * 0 = No discomfort * 1 = Mild discomfort but I was easily able to do the things I usually do * 2 = Moderate discomfort or pain that made it difficult to do some of the things I usually do * 3 = Severe pain that made it difficult to do the things I usually do. The monthly mean cumulative pain score is the average of the daily values for all days (menstrual and non-menstrual) reported during the 4 weeks prior to each visit.
Change From Baseline in the Monthly Mean Cumulative Pain Score During the Open-label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Participants assessed dysmenorrhea or non-menstrual pelvic pain at approximately the same time each day in an e-Diary according to the following: * 0: No discomfort * 1: Mild discomfort, I was easily able to do the things I usually do * 2: Moderate discomfort or pain making it difficult to do some of the things I usually do * 3: Severe pain making it difficult to do the things I usually do The monthly mean cumulative pain score is the average of the daily values for all days (menstrual and non-menstrual) reported during the 4 weeks prior to each visit, except for the week 30 value which is based on 6 weeks of data.
Change From Baseline in the Monthly Mean Dyspareunia Score During the Double-Blind Treatment Phase
Time frame: Baseline and weeks 4 and 8
Participants assessed their dyspareunia (pain during sexual intercourse) at approximately the same time every day in an e-Diary according to the following response options: * 0 = Absent; No discomfort during sexual intercourse * 1 = Mild; I was able to tolerate the discomfort during sexual intercourse * 2 = Moderate; Intercourse was interrupted due to pain * 3 = Severe; I avoided intercourse because of pain * Does not apply; I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse The monthly mean dyspareunia score is the average of the daily values reported during the 4 weeks prior to each visit. Responses of "does not apply" were not included in the calculations.
Change From Baseline in the Monthly Mean Dyspareunia Score During the Open-label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Participants assessed their dyspareunia (pain during sexual intercourse) at approximately the same time every day in an e-Diary according to the following response options: * 0: Absent; No discomfort during sexual intercourse * 1: Mild; I was able to tolerate the discomfort during sexual intercourse * 2: Moderate; Intercourse was interrupted due to pain * 3: Severe; I avoided intercourse because of pain * Does not apply; I was not sexually active for reasons other than my endometriosis or did not have sexual intercourse The monthly mean dyspareunia score is the average of the daily values reported during the 4 weeks prior to each visit, except for week 30 which is based on 6 weeks of data. Responses of "does not apply" were not included in the calculations.

20 secondary outcomes

Percentage of Participants With a Response in Monthly Mean Dysmenorrhea Score at Week 8
Time frame: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Non-menstrual Pelvic Pain Score at Week 8
Time frame: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Cumulative Pain Score at Week 8
Time frame: Baseline and Week 8
Percentage of Participants With a Response in Monthly Mean Dyspareunia Score at Week 8
Time frame: Baseline and Week 8
Change From Baseline in the Percentage of Days of Any Analgesic Use During the Double-Blind Treatment Phase
Time frame: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Any Analgesic Use During the Open-Label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Double-Blind Treatment Phase
Time frame: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Prescription Analgesic Use During the Open-Label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Double-Blind Treatment Phase
Time frame: Baseline and Weeks 4 and 8
Change From Baseline in the Percentage of Days of Narcotic Analgesic Use During the Open-Label and Posttreatment Phases
Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
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
Time frame: Baseline and Week 8
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
Time frame: Baseline and week 24
Patient Global Impression of Change During the Double-blind Treatment Phase
Time frame: Weeks 4 and 8
Patient Global Impression of Change During the Open-Label and Posttreatment Phases
Time frame: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Double-blind Treatment Phase
Time frame: Weeks 4 and 8
Percentage of Participants With a PGIC Response of Much Improved or Very Much Improved During the Open-label Treatment Phase
Time frame: Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment)
Change From Baseline to the End of the Double-blind Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
Time frame: Baseline and week 8
Change From Baseline to the End of the Open-label Treatment Phase in Endometriosis Health Profile-5 (EHP-5)
Time frame: Baseline and week 24
Percentage of Days With Uterine Bleeding During the Double- Blind Treatment Phase
Time frame: Screening (8 weeks prior to day 1) and the double-blind treatment phase (Weeks 1-8)
Number of Days to First Posttreatment Menses
Time frame: From last day of study drug up to 6 weeks after the last dose.

Results and safety

Results posted to the registry on 26 Sep 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	69	63
Elagolix 150 mg	68	60

Primary outcome: Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Double-blind Treatment Phase

Time frame: Baseline and Weeks 4 and 8. Least squares mean (Standard Error). Unit: units on a scale

Measure	Placebo n = 66	Elagolix 150 mg n = 66
Week 4	-0.24 (0.076)	-0.49 (0.076)
Week 8	-0.37 (0.107)	-1.13 (0.107)

Placebo vs Elagolix 150 mg: LS Mean Difference: -0.24 (95% CI -0.45 to -0.03), p = 0.0262, ANCOVA
Placebo vs Elagolix 150 mg: LS Mean Difference: -0.76 (95% CI -1.06 to -0.46), p < 0.0001, ANCOVA

Population analysed: All randomized participants who received at least 1 dose of study drug and had at least 10 evaluable e-Diary reports during the double-blind period (intent-to-treat population). The analysis includes participants with non-missing data at each time point.

Primary outcome: Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Open-label and Posttreatment Phases

Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment). Mean (Standard Error). Unit: units on a scale

Measure	Placebo n = 61	Elagolix 150 mg n = 60
Week 12	-0.768 (0.118)	-1.060 (0.130)
Week 16	-1.325 (0.112)	-1.022 (0.137)
Week 20	-1.026 (0.119)	-0.991 (0.121)
Week 24	-1.282 (0.118)	-1.372 (0.132)
Week 30	-0.547 (0.104)	-0.534 (0.102)

Population analysed: All randomized participants who received at least 1 dose of study drug and had at least 10 evaluable e-Diary reports during the double-blind period (intent-to-treat population) with data on or after Week 12. The analysis includes participants with non-missing data at each time point.

Primary outcome: Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Double-Blind Treatment Phase

Time frame: Baseline and weeks 4 and 8. Least squares mean (Standard Error). Unit: units on a scale

Measure	Placebo n = 66	Elagolix 150 mg n = 66
Week 4	-0.05 (0.061)	-0.26 (0.061)
Week 8	-0.19 (0.071)	-0.47 (0.071)

Placebo vs Elagolix 150 mg: LS Mean Difference: -0.21 (95% CI -0.38 to -0.04), p = 0.0163, ANCOVA
Placebo vs Elagolix 150 mg: LS Mean Difference: -0.28 (95% CI -0.48 to -0.08), p = 0.0066, ANCOVA

Primary outcome: Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score During the Open-label and Posttreatment Phases

Time frame: Baseline and Weeks 12, 16, 20, 24, and 30 (6 weeks posttreatment). Mean (Standard Error). Unit: units on a scale

Measure	Placebo n = 61	Elagolix 150 mg n = 60
Week 12	-0.355 (0.087)	-0.727 (0.072)
Week 16	-0.422 (0.078)	-0.763 (0.079)
Week 20	-0.524 (0.090)	-0.759 (0.082)
Week 24	-0.543 (0.092)	-0.801 (0.082)
Week 30	-0.326 (0.104)	-0.689 (0.085)

Population analysed: All randomized participants who received at least 1 dose of study drug and had at least 10 evaluable e-Diary reports during the double-blind period (intent-to-treat population) with data on or after Week 12. The analysis includes participants with non-missing data at each time point.

20 secondary outcomes are reported in the full results.

Adverse events

Time frame: From the first dose of any study drug through week 24. The Placebo treatment group includes data for the 8-week double-blind treatment phase. The Elagolix treatment group included data for the total 24-week treatment period for participants initially randomized to elagolix, and 16-week open-label treatment period for participants initially randomized to placebo.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	3 of 69 (4.3%)	13 of 69 (18.8%)	0 of 69
Elagolix	1 of 131 (0.8%)	49 of 131 (37.4%)	0 of 131

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Undecided

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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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Elagolix in people with Endometriosis, Pain, run by AbbVie.

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 6 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: finished, as of the sponsor's last update on 26 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 26 Sep 2018). 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?

Change From Baseline in the Monthly Mean Dysmenorrhea Score During the Double-blind Treatment Phase (time frame: Baseline and Weeks 4 and 8). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00973973.

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 26 Sep 2018.