Efficacy and safety of Ursodeoxycholic Acid as a new therapy for the treatment of hepatorenal polycystic diseases, genetic study and determination of prognostic biomarkers.
This research study is looking into a new treatment called Ursodeoxycholic Acid for people with polycystic diseases that affect both their liver and kidneys. These conditions cause many fluid-filled sacs, called cysts, to grow. The main goal is to find out if this medication can reduce the size of the liver cysts. Researchers will compare changes in liver cyst volume between people receiving the treatment and those who aren't. They will also check if the medication is safe, how it affects kidney health, overall well-being, and quality of life. This is an early-stage study (Phase II), meaning it's focused on understanding how well the treatment works and its safety before potentially moving to larger trials.
At a glance
What is this study about?
This study is investigating a new treatment for a condition called hepatorenal polycystic disease. This is a genetic condition where fluid-filled sacs, known as cysts, grow in the liver and kidneys. These cysts can sometimes grow quite large and affect how well these important organs work. Doctors are looking for new ways to manage this condition and improve the lives of people who have it.
The medication being tested in this study is called Ursodeoxycholic Acid. It's a type of acid that naturally occurs in the body and is sometimes used for other liver conditions. Researchers want to see if it can help reduce the size of the cysts in the liver and kidneys, and ultimately improve the health of people living with this disease. This is a Phase II study, which means it's an earlier stage of research where the main focus is on understanding if the treatment is safe and if it shows promise in helping patients.
The study will look very closely at how the liver cysts change in size. They'll measure this at the beginning and end of the treatment period to see if the medication has an effect. They will also monitor a wide range of other important health markers, such as kidney function, overall liver health, and how patients feel in terms of their symptoms and quality of life. They will also look at genetic information to understand why some people might respond differently to the treatment.
Key takeaways
- This study tests a new drug for liver and kidney polycystic diseases.
- The primary goal is to see if the drug shrinks liver cysts.
- It's a Phase II study, focusing on safety and early effectiveness.
- Researchers will also monitor kidney function and quality of life.
- Participation involves regular visits, tests, and taking the study medication.
- You can withdraw from the study at any time.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older. The study is open to both men and women.
There might be other specific health requirements or conditions that would mean you can or cannot take part. These are put in place to make sure the study is safe for everyone involved and that the results are clear.
It's really important to discuss your full medical history with the study team. They will carefully check if this study is suitable for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Am I 18 years old or older?
- Do I have a polycystic disease affecting my liver and kidneys?
- Am I able to attend regular appointments at the study centre?
- Am I comfortable completing questionnaires about my health and well-being?
What does participation involve?
If you decide to take part in this study, you would receive the study medication, Ursodeoxycholic Acid, for a period of time. You would have regular visits to the study centre, where the research team will monitor your health very closely. These visits will involve various assessments, such as blood tests, possibly urine tests, and scans to measure the size of your liver and kidney cysts. You'll also be asked to fill out questionnaires about your symptoms and how you're feeling, which helps the researchers understand your quality of life.
The study team will monitor your health throughout your participation to check for any changes or side effects, and to see how well the treatment is working. They will also check on your existing medications and how often you're taking your study medicine. The total duration of your participation in the study, including follow-up, would be fully explained to you by the study team.
Potential risks and benefits
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Common questions
What are polycystic diseases?
These are genetic conditions where many fluid-filled sacs, called cysts, grow inside organs like the liver and kidneys.
What is Ursodeoxycholic Acid?
It's a medication being tested in this study. It's a natural bile acid sometimes used for other liver conditions.
What does 'Phase II' mean for a study?
It means this is an early stage of research, focusing on whether the treatment is safe and if it shows promise in treating the condition.
Will I know if I'm getting the active treatment or a placebo?
The study design will determine this. The research team will explain it to you fully before you decide to take part.
What is the main thing the study hopes to find out?
The main goal is to see if the medication can reduce the size of liver cysts in people with polycystic diseases.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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