Protocol for a randomized controlled multicenter trial assessing the efficacy of leuprorelin for severe polycystic liver disease: the AGAINST-PLD study

What Was Tested

This clinical study looks at Leuprolide.

In patients with severe polycystic liver disease (PLD), there is a need for new treatments.

Estrogens and possibly other female sex hormones stimulate growth in PLD.

Who Took Part

PubMed tags the participants as women only.

This design enables comparison between patients on study treatment and standard of care (first 18 months) and within patients before and during treatment (whole trial).

Main outcome is annualized liver growth rate compared between standard of care and study treatment.

How the Study Was Run

The design is described as randomized, open-label and multicenter.

Because people in this Leuprolide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Being open-label, the Leuprolide study did not mask who received what.

Running at several centres spread the Leuprolide study over different clinics and patient groups.

The AGAINST-PLD study is an investigator-driven, multicenter, randomized controlled trial.

Institutional review board (IRB) approval was received at the University Medical Center of Groningen and will be collected in other sites before opening these sites.

The trial duration is 36 months.

MRI scans to measure liver volume will be performed at screening, 6 months, 18 months, 24 months and 36 months.

In addition, blood will be drawn, DEXA-scans will be performed and questionnaires will be collected.

What Was Measured

Secondary outcomes are PLD disease severity, change in liver growth within individuals and (serious) adverse events.

The study is designed as a prospective open-label study with blinded endpoint assessment (PROBE).

Topics PubMed links to this paper include Cysts, Leuprolide, Liver Diseases and Randomized Controlled Trials as Topic.

Author keywords: Estrogen, GnRHa and Polycystic liver disease.

What This Means in Plain Language

The paper is a randomized, open-label and multicenter study of Leuprolide.

Its findings take up roughly 3 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2022 (BMC gastroenterology), a journal based in England.

More context: the Leuprolide compound page, all human trials.