Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Double-blind, randomized, placebo-controlled, phase III study comparing norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis

This research study is testing a new medicine, NUC01, for people with a long-term liver disease called primary sclerosing cholangitis (PSC). The study is designed to be very thorough, comparing NUC01 against a placebo (a tablet with no active medicine) to carefully observe its effects. Scientists want to know if NUC01 can help improve key markers of liver health and prevent the disease from getting worse. This is a "Phase III" study, which means it's a final big step before a medicine might be made available more widely. The aim is to find out if NUC01 is a safe and effective treatment option for PSC.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Dr. Falk Pharma GmbH

Enrolment target

195

Start

14 Oct 2024

primary sclerosing cholangitis (PSC)

What is this study about?

This study is all about finding new ways to help people living with primary sclerosing cholangitis, often called PSC. PSC is a long-term liver disease that affects the bile ducts – the small tubes that carry digestive liquid (bile) from your liver. Over time, these tubes can become scarred and narrowed, which can cause serious problems for your liver.

The main goal of this study is to test a new medication, NUC01, to see if it can make a real difference for people with PSC. Researchers want to find out if NUC01 can help improve certain measures of liver health and prevent the disease from getting worse. To do this fairly, some participants will receive NUC01, while others will receive a 'placebo' – a dummy medicine that looks the same but contains no active drug. This comparison helps scientists understand the true effects of the new medicine.

This kind of study is called a 'Phase III' trial, which means it’s one of the final and most important steps in testing a new medicine. It’s designed to confirm whether the medicine is safe and effective on a larger group of people before it can potentially be approved for use by doctors. The overall aim is to improve the lives of people with PSC by finding better treatment options.

Key takeaways

This study is testing a new medicine (NUC01) for Primary Sclerosing Cholangitis (PSC).
It aims to see if NUC01 can improve liver health and slow down the disease.
The study involves comparing NUC01 to a dummy medicine (placebo).
It's a Phase III trial, a critical step before a medicine can be widely available.
Participation involves regular clinic visits, tests, and taking capsules for nearly two years.
You can discuss with your doctor if this study might be right for you.

Who may be eligible?

To be considered for this study, you need to be an adult, aged 18 or over. The study is open to both men and women.

The researchers are looking for people who have been diagnosed with primary sclerosing cholangitis (PSC). There will be specific medical checks to make sure your PSC fits the study's requirements. These checks help ensure that the study can accurately measure how the new medicine works.

It's important to remember that not everyone with PSC will be able to join. There will be other health conditions or medications that might mean you can't take part. These rules are in place to keep participants safe and to make sure the study results are clear and reliable. Your doctor will be able to go through all the detailed requirements with 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.

Are you 18 years old or older?
Have you been diagnosed with Primary Sclerosing Cholangitis (PSC)?
Are you comfortable with regular hospital or clinic visits and tests?
Could you commit to taking medication for almost two years?
Are you able to provide informed consent (understand and agree to participate)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would be involved for a total of 96 weeks, which is about 1 year and 10 months. During this time, you would receive either the new medication, NUC01, or a placebo (a dummy medicine) in capsule form. Neither you nor your study doctor would know which one you are receiving, which helps make the study fair.

You would have regular visits to your clinic or hospital for check-ups. These visits will involve blood tests to measure important markers of your liver health, such as s-ALP. You might also have imaging scans to look at your liver and bile ducts, and possibly tissue samples (biopsies) taken from your liver to see how the disease is progressing. Researchers will also check your general health and how you're feeling, including your quality of life.

There will be follow-up appointments after the main treatment period to ensure your health is monitored. Throughout the study, the research team will explain all procedures, answer your questions, and support you.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new treatment before it's widely available and closer monitoring of your health. However, there are also potential risks involved, which could include side effects from the study medication, or discomfort from procedures like blood tests or biopsies. It's crucial to understand that you are free to withdraw from the study at any time, for any reason, without affecting your usual medical care, and your safety will be a top priority throughout the trial.

Locations (9)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Denmark
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Unverified
Austria
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Unverified
Norway
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Unverified
Finland
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Unverified
Poland
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Unverified
Germany
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Unverified
France
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Unverified
Netherlands
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Unverified
Hungary

Common questions

What is primary sclerosing cholangitis (PSC)?

PSC is a long-term liver disease where the bile ducts inside and outside your liver become inflamed and scarred, blocking bile flow and potentially damaging the liver over time.

What is NUC01?

NUC01 is the name of the new medication being tested in this study. It's hoped to help improve liver health in people with PSC.

What does 'placebo' mean?

A placebo is a dummy pill that looks just like the study medication but contains no active drug. It helps researchers compare the effects of the active medicine fairly.

How long will the study last?

If you join, the study medication and main assessments will last for 96 weeks (about 1 year and 10 months), with follow-up after that.

Will I know if I'm getting the real medicine or the placebo?

No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you are receiving NUC01 or the placebo. This helps keep the study fair and unbiased.