In short
There are currently 2 clinical trials recruiting patients with polycystic liver disease. These studies are testing different hormonal treatments aimed at slowing liver growth and reducing symptoms in patients affected by this condition. Trials are taking place in the Netherlands, Belgium, and Germany.
Clinical trial locations
- Belgium
- Germany
- Netherlands
Study on Leuprorelin Acetate for Slowing Disease Progression in Premenopausal Women with Severe Polycystic Liver Disease
This trial is investigating whether leuprorelin acetate can slow down liver growth in premenopausal women with severe polycystic liver disease. The study will last 18 months and use MRI scans to monitor changes in liver size.
Who can participate:
- Female patients between 18 and 45 years old
- Must have a very large liver for their age group, with specific volume measurements based on age ranges
- Must have polycystic liver disease with more than 10 liver cysts
- Either currently using somatostatin analogues without success, or have a valid reason not to use these medications
- Must be able to provide written consent and complete study questionnaires
Who cannot participate:
- Male patients
- Women who have already gone through menopause
- Those without severe polycystic liver disease
What the study involves: Participants will be randomly assigned to either start treatment immediately or after a delay. Those receiving treatment will get leuprorelin acetate as an injection under the skin. The medication works by lowering estrogen and progesterone levels, which researchers believe may slow the growth of liver cysts. Throughout the study, participants will have regular check-ups, MRI scans to measure liver growth, and will complete questionnaires about their symptoms and any side effects.
Investigational drug: Leuprorelin acetate belongs to a class of medications called GnRH agonists. It works by reducing hormone levels in the body, specifically estrogen and progesterone, which are thought to contribute to cyst growth in premenopausal women.
Study on Octreotide for Patients with Symptomatic Polycystic Liver Disease
This trial is evaluating CAM2029, also known as octreotide subcutaneous depot, for patients experiencing symptoms related to polycystic liver disease. The study aims to determine whether this treatment can reduce liver volume and improve quality of life.
Who can participate:
- Male or female patients at least 18 years old
- Must have polycystic liver disease with a liver volume of at least 1800 ml/m
- Must experience at least one symptom related to the condition within two weeks before screening, such as bloating, abdominal fullness, lack of appetite, pain, or shortness of breath after physical activity
- Must not be a candidate for surgical intervention or unwilling to undergo surgery during the trial
- Female patients of childbearing potential and male patients must agree to use appropriate contraception methods throughout the study
Who cannot participate:
- Patients with other liver conditions besides polycystic liver disease
- Those outside the specified age range
- Vulnerable populations requiring special protection or care
What the study involves: This is a double-blind study, meaning neither participants nor researchers will know who receives the actual medication versus a placebo. Participants will receive treatment as an injection under the skin and will have regular appointments for up to 53 weeks. Monitoring will include MRI scans to measure liver volume changes and assessments of symptoms. The study includes follow-up visits after treatment completion to monitor long-term effects.
Investigational drug: CAM2029 (octreotide subcutaneous depot) is a somatostatin analog that mimics a natural hormone in the body. It is designed to be injected under the skin and release the medication slowly over time. The drug works by reducing the production of certain bodily fluids and slowing down the growth of cysts in the liver.
Summary
Currently, two clinical trials are actively recruiting patients with polycystic liver disease. Both studies focus on hormonal approaches to managing the condition, though they target different patient populations and use different medications.
The first trial is specifically designed for premenopausal women with severe disease and tests leuprorelin acetate, a hormone-lowering medication. The second trial is open to both men and women who experience symptoms and evaluates octreotide, a medication that mimics a natural hormone to reduce cyst growth.
Geographically, the Netherlands hosts both trials, while Belgium and Germany participate in the octreotide study. Both studies use MRI scans to objectively measure changes in liver volume and closely monitor participants' symptoms and safety throughout the treatment period. These trials represent important research efforts to find effective treatments for a condition that currently has limited therapeutic options.
