A Clinical Study to Evaluate the Safety and Efficacy of Elagolix in Participants With Moderate to Severe Endometriosis-Associated Pain

What Was Tested

The trial (phase 3) is built around Elagolix (non-peptide) for endometriosis.

It had three arms (groups): Placebo (placebo comparator), Elagolix / Elagolix + E2/NETA (experimental) and Elagolix + E2/NETA (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 681 actual participants took part.

It was open to women only aged from 18 years up to 49 years.

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

Examples of who could join: Participant is a premenopausal female 18 to 49 years of age (inclusive) at the time of Screening. Participant has a documented surgical diagnosis (e.g., laparoscopy or laparotomy) of endometriosis established by visualization within 10 years prior to entry into Washout or Screening. Participant must agree to use only protocol specified rescue analgesics during the Screening and Treatment Periods for endometriosis-associated pain. Participant must have the following documented in the e-Diary during the last 35 days prior to Study Day 1:. At least 2 days of "moderate" or "severe" dysmenorrhea (DYS) AND either.

The full record lists 7 inclusion and 11 exclusion criteria; among the exclusions: participant has chronic pelvic pain that is not caused by endometriosis, that requires chronic analgesic therapy, which would interfere with the assessment of endometriosis-related pain; participant has either:; participant has any conditions contraindicated with use of e2/neta.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

Random assignment meant chance, not the researchers, decided who got Elagolix (non-peptide) 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 Elagolix (non-peptide), so expectations could not easily colour the readings.

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

It started in 2017-07-07 with completion listed for 2020-03-27.

It ran at 197 sites in United States (188), Canada (5) and Puerto Rico (4).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Co-Primary Endpoint: Percentage of Participants With a Response for Dysmenorrhea (DYS) at Months 6 and 12 Based on Daily Assessment (time frame: Month 6, Month 12). Co-Primary Endpoint: Percentage of Participants With a Response for Non-menstrual Pelvic Pain (NMPP) at Months 6 and 12 Based on Daily Assessment (time frame: Month 6, Month 12).

It also tracked 14 secondary outcomes, for example Change From Baseline in DYS at Month 12 Based on Daily Assessment, Change From Baseline in DYS at Month 6 Based on Daily Assessment, Change From Baseline in DYS at Month 3 Based on Daily Assessment, Change From Baseline in Non-menstrual Pelvic Pain (NMPP) at Month 12 Based on Daily Assessment, Change From Baseline in NMPP at Month 6 Based on Daily Assessment and Change From Baseline in NMPP at Month 3 Based on Daily Assessment.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Elagolix (non-peptide) in 681 people with endometriosis.

With 681 participants it is a large trial for this field.

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

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