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A study to evaluate the effectiveness and safety of ursodeoxycholic acid in patients with hepatorenal polycystic diseases.

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Hepatorenal polycystic diseases, which are conditions where fluid-filled sacs, known as cysts, grow in both the liver and the kidneys. The goal of the research is to evaluate the effectiveness and safety of ursodeoxycholic acid compared to a placebo in slowing the growth of these cysts in the liver.

During the study, participants will be monitored to see how the medication affects the size of the liver and kidney cysts, as well as overall organ function. This includes checking renal function, which refers to how well the kidneys filter waste from the blood, and monitoring liver profile markers through blood tests to ensure safety. Researchers will also look at genetic information and certain proteins or molecules in the blood, often called biomarkers, to better understand how the disease progresses and how individuals respond to the treatment.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Start of the trial
The trial begins with an initial assessment to establish a baseline for the condition, which involves measuring the total liver cyst volume (the total size of fluid-filled sacs in the liver) and the total kidney cyst volume (the total size of fluid-filled sacs in the kidneys).
Physical measurements such as body weight, height, blood pressure, and heart rate are recorded.
Blood tests are performed to check haematology (blood cell counts), biochemistry, hormones, coagulation (how the blood clots), and liver profile (enzymes and markers that show how well the liver is working).
Renal function, which refers to how well the kidneys are filtering waste, is assessed by measuring the glomerular filtration rate (a measure of kidney filtration) and serum creatinine (a waste product in the blood).
Symptoms are documented using a specific questionnaire for polycystic liver disease, and quality of life is assessed using a standard survey.
Step 2
Medication administration
The treatment involves taking ursodeoxycholic acid, which is administered by oral use (swallowed as a pill or liquid).
The dosage is 30 mg/kg, meaning the amount of medicine is calculated based on each kilogram of body weight.
The medication is administered to evaluate its ability to slow the growth of liver cysts compared to a placebo (an inactive substance that looks like the medicine but has no active effect).
Step 3
Monitoring and follow-up
During the treatment period, regular visits are required to monitor safety and any adverse events (unwanted side effects or health changes).
Periodic evaluations are conducted to track changes in liver volume, kidney volume, and kidney cyst volume.
Genetic analysis is performed to study the genetic profile (the specific genes related to the disease) and to see how these genes might relate to the response to the treatment.
Exploratory tests, such as a liquid biopsy (testing blood for proteins, RNA, or other metabolites), may be used to find markers that show how the body is responding to the therapy.
Step 4
End of treatment
At the conclusion of the treatment period, final measurements of the total liver cyst volume and other clinical parameters are taken to determine the overall effect of the medication on the progression of the disease.

Who can join the trial?

6 criteria

You must be between 18 and 80 years old.
You must have a diagnosis of liver disease or hepatorenal polycystic disease, which is a condition where fluid-filled sacs called cysts grow in both the liver and the kidneys.
Your total liver volume, which is the total size or amount of space your liver takes up, must be between 2000 and 5000 mL.
To meet the criteria for polycystic liver disease (PLD), you must have more than 10 cysts in your liver.
To meet the criteria for polycystic kidney disease (PKD), the number of cysts required depends on your age: at least 2 kidney cysts (or 1 cyst in each kidney) if you are under 30; at least 2 cysts in each kidney if you are between 30 and 59; or at least 4 cysts in each kidney if you are 60 or older.
You must not be a candidate for aspiration (a procedure where a needle is used to drain fluid from a cyst) or surgical intervention (an operation) at the time you join the study.

Who cannot join the trial?

16 criteria

If the total volume of liver cysts (fluid-filled sacs in the liver), known as TLV, is greater than 5000 mL.
Having a chronic liver disease (a long-term liver problem) that is not caused by polycystic liver disease (PLD).
Having undergone any aspiration (using a needle to drain fluid) or surgical intervention (surgery) within the last 6 months.
Having used somatostatin analogues (medicines that mimic a natural hormone to slow certain processes) within the last 6 months.
Having an allergy or hypersensitivity (an extreme reaction) to UDCA (the study medication).
Having galactose intolerance (the inability to digest a specific sugar), malabsorption (the body's inability to absorb nutrients properly), or lactase deficiency (the inability to digest the sugar found in milk).
Having acute cholecystitis (sudden inflammation of the gallbladder) or frequent episodes of biliary colic (sudden pain caused by gallstones).
Having used tolvaptan (a medication used for certain kidney or liver conditions) within the last 6 months.
Having renal dysfunction (kidney problems), specifically a GFR (a measure of how well the kidneys filter blood) of 30 ml/min/1.73m2 or less.
Using hormonal contraceptives (birth control pills) or estrogen supplements within the last 6 months.
Participating in another clinical trial (a medical research study) at the same time as this one.
Being pregnant or lactating (breastfeeding), or being able to become pregnant without using an effective form of contraception (birth control).
Having any other serious illness or health condition that makes the doctor believe it is unsafe to participate.
Having gastric ulcers (sores in the stomach) or duodenal ulcers (sores in the first part of the small intestine).
Using medicines that may interfere with the study drug, such as cholestyramine or aluminum hydroxide.
Having used UDCA (the study medication) within the last 6 months.

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Hospital Universitario Donostia
Donostia / San Sebastian, Spain

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Investigated drugs

Ursodeoxycholic acid is a medication used in this study to see if it can help slow down the growth of cysts in the liver for people with certain genetic liver diseases.

Ursodeoxycholic Acid

What is already known about the treatment

Ursodeoxycholic acid - This medication is taken by mouth in pill or capsule form to help treat conditions where fluid-filled sacs grow in the liver and kidneys. It belongs to a group of drugs known as bile acids and is currently used in medical practice to support liver health. On a microscopic level, it works by improving the flow of bile and protecting liver cells from being damaged by harmful substances.

Investigated diseases

Hepatorenal polycystic diseases - This is a group of genetic conditions that cause multiple fluid-filled sacs, known as cysts, to grow in both the liver and the kidneys. As the condition progresses, the number and size of these cysts increase over time. This growth can lead to an enlargement of the total volume of both the liver and the kidneys. The expansion of these organs may eventually affect how well the liver and kidneys function.