CompletedPhase IIIInterventional

An open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets

Primary sclerosing cholangitis (PSC) is a rare condition that causes swelling and scarring in the liver's bile ducts. This phase 3 study, named NUT-022/PSC, offers ongoing treatment to adults who previously finished or took part in an earlier trial called NUC-5/PSC. Participants will take three tablets of norucholic acid (1500 mg total) each day. This replaces the six capsules taken in the previous trial, making the daily dose easier to take. Because this is an 'open-label' study, there is no dummy pill (placebo); every person receives the active medicine. The main aim is to understand how well norucholic acid works and how safe it is over a longer period of up to four years. Researchers will monitor liver health through regular blood tests and painless ultrasound scans at hospital visits every three months.

At a glance

Status

Completed

Phase

Phase III

Sponsor

Dr Falk Pharma (Germany)

Enrolment target

125

Start

31 Mar 2025

Estimated completion

09 Apr 2026

Primary sclerosing cholangitis (PSC)

What is this study about?

Primary sclerosing cholangitis (PSC) is a long-term liver disease where the bile ducts—the tiny tubes carrying digestive juices out of the liver—become inflamed and scarred. Over time, this can lead to bile build-up and liver damage. Finding treatments that can safely protect the liver over many years is an urgent goal for doctors and researchers.

This trial builds directly on an earlier study known as NUC-5/PSC. Researchers have developed a new tablet form of a medicine called norucholic acid (NCA). In earlier stages, participants took six capsules every day. The new tablets provide the exact same daily dose in just three tablets, which aims to make taking the treatment simpler and more convenient.

Because this is an 'open-label' study, you and your medical team will both know that you are taking the real study drug. No one will receive a dummy pill (placebo). The results will help doctors understand whether norucholic acid can safely improve liver health over a prolonged period and support its possible approval as a future treatment for PSC.

Key takeaways

This study is open only to people who took part in the previous NUC-5/PSC trial.
All participants receive the active medicine (norucholic acid); there is no placebo.
The daily treatment involves three tablets instead of the previous six capsules.
Participation lasts up to four years, with check-ups every three months.
Joining is voluntary, and you can withdraw at any time without losing your usual care.

Who may be eligible?

To join this study, you must be 18 years of age or older and have a confirmed diagnosis of primary sclerosing cholangitis (PSC). Crucially, you must have already taken part in the earlier NUC-5/PSC clinical trial.

If you take ursodeoxycholic acid (UDCA), a common medicine used for liver bile conditions, your daily dose must be within an approved limit and kept steady.

Women who can become pregnant must agree to use reliable, highly effective birth control throughout the trial and for four weeks after their last dose. Your study doctor will discuss suitable options with you to ensure your safety.

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.

I am aged 18 or older.
I have a confirmed diagnosis of primary sclerosing cholangitis (PSC).
I previously took part in the NUC-5/PSC study.
If female and able to have children, I am willing to use reliable contraception.
If taking ursodeoxycholic acid (UDCA), my daily dose is stable and not above 20 mg/kg.

Answer every question to see your result.

What does participation involve?

If you decide to join, you will take three norucholic acid tablets once a day by mouth, giving you a total dose of 1500 mg. The study can last for up to 216 weeks (around four years). During this time, you will visit the hospital roughly once every three months, totalling around 20 visits. Most check-ups include routine physical exams and small blood tests (around 30 ml, or two tablespoons). At weeks 72 and 144, you will have more comprehensive health reviews, which include quick, non-invasive imaging tests such as an ultrasound to check the stiffness of your liver. Five hospital centres across the UK are taking part.

Potential risks and benefits

You might experience improvements in your liver condition, but direct health benefits cannot be promised, and your PSC could stay the same or worsen. However, your participation will provide valuable scientific knowledge to help future PSC patients. The medicine may cause unwanted side effects or allergic reactions, which your study doctor will closely manage. Having blood taken can cause minor bruising, discomfort, or rarely, slight inflammation or nerve irritation around the vein. Taking part is completely your choice, and you are free to leave the trial at any time without this affecting your everyday medical care.

Locations (5)

Norfolk and Norwich University Hospital
Approximate
Norwich, England
University Hospitals Birmingham NHS Foundation Trust
City only
Birmingham, England
Royal Free London NHS Foundation Trust
City only
London, England
Kings College Hospital
Approximate
London, England
Freeman Hospital
Approximate
Newcastle upon Tyne, England

Common questions

Will I receive a dummy treatment (placebo)?

No. This is an open-label study, meaning every participant receives the real norucholic acid tablets.

How are these tablets different from the medicine in the last study?

The medicine itself is the same, but it is now pressed into tablets so you only need to take three tablets a day instead of six capsules.

How often do I need to attend the clinic?

You will have regular follow-up visits every three months, with around 20 hospital visits planned across four years.

Can I continue taking my other liver medicines?

Yes, common medicines like ursodeoxycholic acid (UDCA) are generally allowed, provided your dose remains stable and within trial guidelines.

What happens if I change my mind about taking part?

You can leave the study at any point without giving a reason. Your standard NHS care will continue as normal.

How to find out more

Clara Grundmann

clinical.studies@drfalkpharma.de + 49 76115140 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.