Efficacy and Safety Study of Elagolix Versus Placebo or Leuprorelin Acetate in Endometriosis

AbbVie

Status and phase: Completed; Phase 2

Conditions: Endometriosis

Treatments: Drug: Placebo to Leuprorelin Acetate; Drug: Leuprorelin Acetate Depot; Drug: Elagolix; Drug: Placebo to Elagolix

Study type: Interventional

Funder types: Industry

Identifiers

NCT00797225

2007-006474-28 (EudraCT Number)

NBI-56418-0703

Details and patient eligibility

About

This study is designed to evaluate the safety and beneficial effects of elagolix (NBI-56418) compared to placebo and leuprorelin (an approved endometriosis therapy) over a three month period followed by an additional three months of treatment on elagolix.

Full description

The study followed a parallel-group design in which participants were randomized (1:1:1:1) to one of the following treatment groups for the first 12 weeks of dosing: 150 mg elagolix once daily (q.d.); 250 mg elagolix q.d.; placebo; or leuprorelin acetate depot injection 3.75 mg (monthly). Blinding was achieved using a double-dummy design. Following 12 weeks of dosing, participants continued in the study for an additional 12 weeks; participants randomized to elagolix continued to receive their assigned dose and participants randomized to placebo or leuprorelin acetate were re-randomized to receive one of the two doses of elagolix (150 mg q.d. or 250 mg q.d.) for 12 weeks in a double-blind fashion. Six weeks after the last dose of the study drug at the end of Week 24, a follow-up visit was performed (end of Week 30).

There was no pre-specified primary efficacy end point as there was no single key efficacy outcome measure in this exploratory Phase 2 study. For purposes of results reported here, Change From Baseline in the Monthly Mean Numerical Rating Score (NRS) for Endometriosis Pain is designated as the primary outcome measure.

Enrollment: 174 patients

Sex: Female

Ages: 18 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

• Female, aged 18 to 45 years, inclusive
• Have moderate to severe pelvic pain due to endometriosis
• Have been surgically (laparoscopy) diagnosed with endometriosis within the last 5 years and have recurrent or persistent endometriosis symptoms
• Have regular menstrual cycle (23-33 day)
• Agree to use two forms of non-hormonal contraception during the study

Exclusion Criteria:

• Received a Gonadotropin-releasing hormone (GnRH) agonist, GnRH antagonist, danazol, or have received any of these agents within 6 months of the start of screening.
• Received subcutaneous medroxyprogesterone acetate (DMPA-SC) or intramuscular (i.m.) medroxyprogesterone acetate (DMPA-IM), or have received either 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
• Are using systemic steroids on a chronic or regular basis within 3 months
• Have uterine fibroids or other pelvic lesions ≥ 3 cm in diameter
• Have had a hysterectomy or oophorectomy
• Have pelvic pain that is not caused by endometriosis
• Have unstable medical condition or chronic disease
• Have been pregnant within the last 6 months and is currently breast feeding

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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174 participants in 4 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Participants received placebo tablets once a day and placebo intramuscular injection once a month for 12 weeks. At the end of 12 weeks participants were re-randomized to receive one of the two doses of elagolix (150 mg or 250 mg) for 12 weeks.

Treatment:

Drug: Placebo to Elagolix

Drug: Placebo to Leuprorelin Acetate

Elagolix 150 mg

Experimental group

Description:

Participants received elagolix 150 mg tablets once a day and placebo intramuscular injection once a month for 12 weeks. At the end of 12 weeks participants continued to receive elagolix 150 mg for an additional 12 weeks.

Treatment:

Drug: Elagolix

Drug: Placebo to Leuprorelin Acetate

Elagolix 250 mg

Experimental group

Description:

Participants received elagolix 250 mg tablets once a day and placebo intramuscular injection once a month for 12 weeks. At the end of 12 weeks participants continued to receive elagolix 250 mg for an additional 12 weeks.

Treatment:

Drug: Elagolix

Drug: Placebo to Leuprorelin Acetate

Leuprorelin

Other group

Description:

Participants received placebo tablets once a day and leuprorelin acetate 1-month depot 3.75 mg intramuscular injection once a month for 12 weeks. At the end of 12 weeks participants were re-randomized to receive one of the two doses of elagolix (150 mg or 250 mg) for 12 weeks.

Treatment:

Drug: Placebo to Elagolix

Drug: Leuprorelin Acetate Depot