NCT00619866

An Efficacy and Safety Study of Elagolix (NBI-56418) in Women With Endometriosis

Completed Phase 2 Results posted Last updated 7 September 2018

What this trial tests

Phase 2 trial testing Elagolix in Endometriosis, Pain in 155 participants. Completed in 28 August 2009.

Timeline

19 February 2008

Primary endpoint
20 April 2009

28 August 2009

Quick facts

Lead sponsor	AbbVie
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	155
Start date	19 February 2008
Primary completion	20 April 2009
Estimated completion	28 August 2009

Drugs / interventions tested

• Elagolix (ELAGOLIX) — full drug profile →
• placebo

Conditions studied

• Endometriosis, Pain — all drugs for Endometriosis, Pain →

Sponsor

AbbVie — full company profile →

Who can join

Adults 18 to 49, female only, with Endometriosis, Pain. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in the Monthly Mean Numerical Rating Score (NRS) for Endometriosis Pain at Week 12 Primary · Baseline and week 12

The NRS is an 11-point scale used to measure endometriosis pain and was completed at approximately the same time each day using an electronic diary (e-Diary). Participants were instructed to select a single number between 0 (No pain) and 10 (Worst pain ever) that best described their endometriosis pain at its worst over the past day. The monthly mean NRS is the average of the daily values reported during the 4 weeks prior to each visit.

Group	Value	95% CI
Placebo	-0.88	± 0.18
Elagolix 150 mg	-1.19	± 0.18
Elagolix 250 mg	-1.25	± 0.18

Change From Baseline in the Monthly Mean Numerical Rating Score (NRS) for Endometriosis Pain Secondary · Baseline and weeks 4 and 8

The NRS is an 11-point scale used to measure endometriosis pain and was completed at approximately the same time each day using an electronic diary (e-Diary). Participants were instructed to select a single number between 0 (No pain) and 10 (Worst pain ever) that best described their endometriosis pain at its worst over the past day. The monthly mean NRS is the average of the daily values reported during the 4 weeks prior to each visit.

Week 4

Group	Value	95% CI
Placebo	-0.60	± 0.17
Elagolix 150 mg	-0.90	± 0.17
Elagolix 250 mg	-0.83	± 0.17

Week 8

Group	Value	95% CI
Placebo	-0.71	± 0.18
Elagolix 150 mg	-1.23	± 0.17
Elagolix 250 mg	-0.94	± 0.17

Change From Baseline in the Monthly Peak Numerical Rating Score (NRS) for Endometriosis Pain Secondary · Baseline and Weeks 4, 8, and 12

The NRS is an 11-point scale used to measure endometriosis pain and was completed at approximately the same time each day using an electronic diary (e-Diary). Participants were instructed to select a single number between 0 (No pain) and 10 (Worst pain ever) that best described their endometriosis pain at its worst over the past day. The monthly peak NRS is the maximum of the daily values reported during the 4 weeks prior to each visit.

Week 4

Group	Value	95% CI
Placebo	-1.27	± 0.32
Elagolix 150 mg	-1.39	± 0.32
Elagolix 250 mg	-1.58	± 0.32

Week 8

Group	Value	95% CI
Placebo	-1.25	± 0.34
Elagolix 150 mg	-1.94	± 0.33
Elagolix 250 mg	-2.41	± 0.33

Week 12

Group	Value	95% CI
Placebo	-1.30	± 0.36
Elagolix 150 mg	-2.45	± 0.34
Elagolix 250 mg	-2.74	± 0.34

Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score Secondary · Baseline and weeks 4, 8, and 12

Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time every day in an e-Diary according to the following response options: * 0 = No pelvic pain * 1 = Mild pelvic pain; subject could not do some of the things she usually does * 2 = Moderate pelvic pain; subject could not do many of the things she usually does * 3 = Severe pelvic pain; subject could not do most or all of the things she usually does. The monthly mean non-menstrual pelvic pain score is the average of the daily values reported during the 4 weeks prior

Week 4

Group	Value	95% CI
Placebo	-0.14	± 0.05
Elagolix 150 mg	-0.18	± 0.05
Elagolix 250 mg	-0.16	± 0.05

Week 8

Group	Value	95% CI
Placebo	-0.12	± 0.05
Elagolix 150 mg	-0.30	± 0.05
Elagolix 250 mg	-0.17	± 0.05

Week 12

Group	Value	95% CI
Placebo	-0.22	± 0.06
Elagolix 150 mg	-0.27	± 0.05
Elagolix 250 mg	-0.25	± 0.05

Change From Baseline in the Monthly Mean Dysmenorrhea Score Secondary · Baseline and Weeks 4, 8, and 12

Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day in an e-Diary according to the following response options: * Subject is not having her period * 0 = No pain related to period * 1 = Mild pain related to period; subject could not do some of the things she usually does * 2 = Moderate pain related to period; subject could not do many of the things she usually does * 3 = Severe pain related to period; subject could not do most of or all of the things she usually does. The monthly mean dysmenorrhea score

Week 4

Group	Value	95% CI
Placebo	-0.20	± 0.09
Elagolix 150 mg	-0.49	± 0.09
Elagolix 250 mg	-0.40	± 0.09

Week 8

Group	Value	95% CI
Placebo	-0.29	± 0.10
Elagolix 150 mg	-0.71	± 0.09
Elagolix 250 mg	-0.79	± 0.09

Week 12

Group	Value	95% CI
Placebo	-0.24	± 0.10
Elagolix 150 mg	-0.68	± 0.10
Elagolix 250 mg	-0.76	± 0.10

Change From Baseline in the Monthly Mean Total of Dysmenorrhea and Non-menstrual Pelvic Pain Scores Secondary · Baseline and Weeks 4, 8, and 12

Participants assessed dysmenorrhea and pelvic pain not related to menses and their impact on daily activities at approximately the same time every day on a 4-point scale (0 = none, 1 = mild, 2 = moderate, and 3 = severe) in an e-Diary. The sum of the dysmenorrhea and non-menstrual pelvic pain scores on each day were calculated to create a daily total score. On days the participant was not having her period, the dysmenorrhea score was not defined; hence, the total score was equal to the non-menstrual pelvic pain score (range 0 to 3). On days where the participant recorded menstruation the tota

Week 4

Group	Value	95% CI
Placebo	-0.18	± 0.06
Elagolix 150 mg	-0.34	± 0.06
Elagolix 250 mg	-0.29	± 0.06

Week 8

Group	Value	95% CI
Placebo	-0.18	± 0.06
Elagolix 150 mg	-0.48	± 0.06
Elagolix 250 mg	-0.33	± 0.06

Week 12

Group	Value	95% CI
Placebo	-0.27	± 0.07
Elagolix 150 mg	-0.44	± 0.06
Elagolix 250 mg	-0.41	± 0.06

Percentage of Days With No Pain Based on NRS Secondary · Baseline and weeks 4, 8 and 12

The NRS is an 11-point scale used to measure endometriosis pain and was completed at approximately the same time each day using an electronic diary (e-Diary). Participants were instructed to select a single number between 0 (No pain) and 10 (Worst pain ever) that best described their endometriosis pain at its worst over the past day. The percentage of days a participant reported a value of zero (or "no pain") for the NRS was calculated for the 4 weeks prior to each visit.

Baseline

Group	Value	95% CI
Placebo	27.600	± 3.588
Elagolix 150 mg	25.380	± 3.564
Elagolix 250 mg	25.515	± 3.493

Week 4

Group	Value	95% CI
Placebo	35.252	± 4.118
Elagolix 150 mg	36.401	± 4.606
Elagolix 250 mg	32.355	± 4.604

Week 8

Group	Value	95% CI
Placebo	40.861	± 4.671
Elagolix 150 mg	43.780	± 5.114
Elagolix 250 mg	36.737	± 5.467

Week 12

Group	Value	95% CI
Placebo	47.431	± 4.955
Elagolix 150 mg	44.841	± 5.857
Elagolix 250 mg	41.760	± 5.796

Percentage of Days With No Pain Based Based on Non-menstrual Pelvic Pain Daily Assessment Secondary · Baseline and weeks 4, 8 and 12

Participants assessed their pelvic pain not related to menses and its impact on their daily activities at approximately the same time every day in an e-Diary according to the following response options: * 0 = No pelvic pain * 1 = Mild pelvic pain; subject could not do some of the things she usually does * 2 = Moderate pelvic pain; subject could not do many of the things she usually does * 3 = Severe pelvic pain; subject could not do most or all of the things she usually does. The percentage of days a participant reported a value of zero ("no pain") for non-menstrual pelvic pain was calculate

Baseline

Group	Value	95% CI
Placebo	34.939	± 3.831
Elagolix 150 mg	36.821	± 4.041
Elagolix 250 mg	38.530	± 4.065

Week 4

Group	Value	95% CI
Placebo	42.986	± 4.229
Elagolix 150 mg	45.469	± 4.968
Elagolix 250 mg	44.443	± 4.905

Week 8

Group	Value	95% CI
Placebo	46.228	± 4.777
Elagolix 150 mg	54.967	± 5.291
Elagolix 250 mg	47.524	± 5.367

Week 12

Group	Value	95% CI
Placebo	54.270	± 4.768
Elagolix 150 mg	53.836	± 6.077
Elagolix 250 mg	50.061	± 5.496

Percentage of Days With No Pain Based Based on Dysmenorrhea Daily Assessment Secondary · Baseline and weeks 4, 8 and 12

Participants assessed dysmenorrhea (pain during menstruation) and its impact on their daily activities at approximately the same time each day in an e-Diary according to the following response options: * Subject is not having her period * 0 = No pain related to period * 1 = Mild pain related to period; subject could not do some of the things she usually does * 2 = Moderate pain related to period; subject could not do many of the things she usually does * = Severe pain related to period; subject could not do most of or all of the things she usually does. The percentage of days a participant r

Baseline

Group	Value	95% CI
Placebo	26.350	± 3.710
Elagolix 150 mg	14.340	± 2.154
Elagolix 250 mg	21.086	± 3.210

Week 4

Group	Value	95% CI
Placebo	30.513	± 4.435
Elagolix 150 mg	43.411	± 5.775
Elagolix 250 mg	39.475	± 5.509

Week 8

Group	Value	95% CI
Placebo	35.057	± 5.135
Elagolix 150 mg	56.952	± 6.016
Elagolix 250 mg	67.411	± 6.190

Week 12

Group	Value	95% CI
Placebo	34.439	± 5.000
Elagolix 150 mg	61.658	± 6.717
Elagolix 250 mg	66.186	± 6.336

Percentage of Days With No Pain Based Based on Total Score of Non-menstrual Pelvic Pain and Dysmenorrhea Daily Assessment Secondary · Baseline and weeks 4, 8 and 12

Participants assessed dysmenorrhea and pelvic pain not related to menses and their impact on daily activities at approximately the same time every day in an e-Diary on a 4-point scale (0 = none, 1 = mild, 2 = moderate, and 3 = severe). The sum of the dysmenorrhea and non-menstrual pelvic pain scores on each day were calculated to create a daily total score. On days the participant was not having her period, the dysmenorrhea score was not defined; hence, the total score was equal to the non-menstrual pelvic pain score (range 0 to 3). On days where the participant recorded menstruation the total

Baseline

Group	Value	95% CI
Placebo	33.009	± 3.690
Elagolix 150 mg	32.940	± 3.649
Elagolix 250 mg	35.967	± 3.815

Week 4

Group	Value	95% CI
Placebo	39.586	± 4.058
Elagolix 150 mg	44.020	± 4.825
Elagolix 250 mg	43.230	± 4.844

Week 8

Group	Value	95% CI
Placebo	43.961	± 4.607
Elagolix 150 mg	53.526	± 5.200
Elagolix 250 mg	47.108	± 5.380

Week 12

Group	Value	95% CI
Placebo	50.340	± 4.552
Elagolix 150 mg	52.584	± 5.999
Elagolix 250 mg	49.409	± 5.494

Change From Baseline in the Percentage of Days of Any Analgesic Use Secondary · Baseline and Weeks 4, 8, and 12

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").

Week 4

Group	Value	95% CI
Placebo	-4.8	± 2.3
Elagolix 150 mg	-8.7	± 2.3
Elagolix 250 mg	-6.8	± 2.3

Week 8

Group	Value	95% CI
Placebo	-5.8	± 2.4
Elagolix 150 mg	-12.6	± 2.4
Elagolix 250 mg	-10.4	± 2.4

Week 12

Group	Value	95% CI
Placebo	-6.1	± 2.5
Elagolix 150 mg	-10.5	± 2.4
Elagolix 250 mg	-13.9	± 2.4

Change From Baseline in the Percentage of Days of Prescription Analgesic Use Secondary · Baseline and Weeks 4, 8, and 12

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

Week 4

Group	Value	95% CI
Placebo	-1.0	± 1.2
Elagolix 150 mg	-4.0	± 1.2
Elagolix 250 mg	-2.3	± 1.2

Week 8

Group	Value	95% CI
Placebo	-0.8	± 1.4
Elagolix 150 mg	-3.2	± 1.3
Elagolix 250 mg	-3.2	± 1.4

Week 12

Group	Value	95% CI
Placebo	-2.1	± 1.6
Elagolix 150 mg	-2.6	± 1.6
Elagolix 250 mg	-3.3	± 1.6

Adverse events — posted to ClinicalTrials.gov

Time frame: From the first dose of any study drug through week 24. The Placebo treatment group includes data for the initial 12-week treatment phase. The Elagolix treatment groups include data for the total 24-week treatment period for participants initially randomized to elagolix, and 12-week treatment period for participants initially randomized to placebo and re-randomized at week 12.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Placebo

Serious: 0/52 (0%)

Deaths: 0/52

Elagolix 150 mg

Serious: 0/69 (0%)

Deaths: 0/69

Elagolix 250 mg

Serious: 1/72 (1%)

Deaths: 0/72

Serious adverse events (1 terms)

Reaction	System	Placebo	Elagolix 150 mg	Elagolix 250 mg
ABORTION SPONTANEOUS	Pregnancy, puerperium and perinatal conditions	—	—	—

Other adverse events (12 terms — click to expand)

Reaction	System	Placebo	Elagolix 150 mg	Elagolix 250 mg
NAUSEA	Gastrointestinal disorders	—	—	—
SINUSITIS	Infections and infestations	—	—	—
UPPER RESPIRATORY TRACT INFECTION	Infections and infestations	—	—	—
VAGINAL MYCOSIS	Infections and infestations	—	—	—
HEADACHE	Nervous system disorders	—	—	—
ACNE	Skin and subcutaneous tissue disorders	—	—	—
URINARY TRACT INFECTION	Infections and infestations	—	—	—
ARTHRALGIA	Musculoskeletal and connective tissue disorders	—	—	—
BLOOD CHOLESTEROL INCREASED	Investigations	—	—	—
BACK PAIN	Musculoskeletal and connective tissue disorders	—	—	—
MIGRAINE	Nervous system disorders	—	—	—
NASOPHARYNGITIS	Infections and infestations	—	—	—

Most-reported serious reactions: ABORTION SPONTANEOUS.

Data from ClinicalTrials.gov NCT00619866 adverse events section.

Sponsor's own description

This study is designed to see how elagolix works compared to placebo in women with endometriosis and to see the effect, if any, on bone mineral density.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. Endometriosis: current challenges in modeling a multifactorial disease of unknown etiology.
   Malvezzi H, Marengo EB, Podgaec S, Piccinato CA. · · 2020 · cited 57× · PMID 32787880 · DOI 10.1186/s12967-020-02471-0
2. Elagolix: First Global Approval.
   Lamb YN. · · 2018 · cited 53× · PMID 30194661 · DOI 10.1007/s40265-018-0977-4
3. A call for more transparency of registered clinical trials on endometriosis.
   Guo SW, Hummelshoj L, Olive DL, Bulun SE, et al · · 2009 · cited 32× · PMID 19264712 · DOI 10.1093/humrep/dep045
4. Endometriosis and pain in the adolescent- striking early to limit suffering: A narrative review.
   Sieberg CB, Lunde CE, Borsook D. · · 2020 · cited 29× · PMID 31862211 · DOI 10.1016/j.neubiorev.2019.12.004