A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease

What Was Tested

In this phase 2 trial, researchers set out to test Leuprolide.

It had two arms (groups): Direct start (active comparator) and Delayed start (other).

36 months of treatment with study medication.

First 18 months standard care, hereafter 18 months treatment with study medication.

The registry classes the interventions as drug.

Who Took Part

The registry lists 36 planned participants were the target.

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: Diagnosis of polycystic liver disease defined as the presence of more than 10 liver cysts. Age between 18 to 45 (inclusive) years;. 18-30 yr; height adjusted TLV \> 2.0 L/m. 30-35 yr; height adjusted TLV \> 2.2 L/m. 35-40 yr; height adjusted TLV \> 2.5 L/m.

The full record lists 14 inclusion and 10 exclusion criteria; among the exclusions: anti mullerian hormone (amh) measurement at screening visit \<0.03 ng/ml; active desire to have children, pregnancy or breast-feeding; liver transplantation or liver surgery expected within 1.5 years, to the discretion of the study doctor.

How the Study Was Run

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

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

The Leuprolide group and the comparison group ran side by side rather than switching.

Only one side, usually the participants, was kept unaware of whether Leuprolide was given.

Those kept unaware of assignments: outcomes assessor.

It started in 2022-06-01 with completion listed for 2027-06-01.

It ran at 2 sites in Netherlands (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Liver growth (time frame: 36 months).

It also tracked 16 secondary outcomes, for example Polycystic liver disease related complaints, Menopause related complaints, Patient reported mental health, Patient reported physical health, Kidney growth and Sex hormone levels.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Leuprolide in 36 people.

Its size is small, so on its own it can only give an early look.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

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