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

What Was Tested

The trial (phase 2) is built around Leuprolide and Elagolix (non-peptide) for endometriosis.

It had four arms (groups): Placebo (placebo comparator), one (experimental), one (experimental) and Leuprorelin (other).

The registry classes the interventions as drug.

More on the Plan

Blinding was achieved using a double-dummy design.

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.

Who Took Part

174 actual participants took part.

It was open to women only aged from 18 years up to 45 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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.

The full record lists 5 inclusion and 10 exclusion criteria; among the exclusions: 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; 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.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

Random assignment meant chance, not the researchers, decided who got Leuprolide and who got the comparison, which limits bias.

Groups ran in parallel: each of the endometriosis participants stayed in one arm throughout.

Neither the endometriosis participants nor the staff measuring them knew who was on Leuprolide, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2008-11-26 with completion listed for 2010-02-24.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Change From Baseline in the Monthly Mean Numerical Rating Score (NRS) for Endometriosis Pain (time frame: Baseline and Weeks 4, 8, and 12).

It also tracked 23 secondary outcomes, for example Change From Baseline in the Monthly Peak Numerical Rating Score (NRS) for Endometriosis Pain, Change From Baseline in the Monthly Mean Non-menstrual Pelvic Pain Score, Change From Baseline in the Monthly Mean Dysmenorrhea Score, Change From Baseline in the Monthly Mean Sum of Dysmenorrhea and Non-menstrual Pelvic Pain Scores, Change From Baseline in the Percentage of Days of Any Analgesic Use and Change From Baseline in the Percentage of Days of Prescription Analgesic Use.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Leuprolide and Elagolix (non-peptide) in 174 people with endometriosis.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Leuprolide compound page and the Elagolix (non-peptide) compound page, Endometriosis research.