Active Digestion, Kidneys & Other Organs NIHR-supported project Cancer

An open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets (NUT002)

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust Birmingham Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Feb 2025 — Sept 2027

In plain English

AI plain-English summary

Patients who took part in an earlier trial of a new drug for primary sclerosing cholangitis (PSC) can now continue receiving it for up to 72 weeks in a follow-up study, with a simpler three-tablet daily dose instead of six capsules. PSC is a rare, progressive liver disease that scars the bile ducts, often leading to liver failure or cancer. No approved drug currently slows its progression. The medication, norucholic acid (NCA), has already been tested in over 700 patients. This open-label study—where everyone knows they are getting the drug—aims to gather longer-term safety and effectiveness data. Around 125 patients globally, including 20 in the UK, will attend check-ups every three months, with routine blood draws and non-invasive imaging. If NCA proves safe and effective over this extended period, it could become the first treatment to slow PSC’s progression, potentially delaying or preventing the need for liver transplants. That would directly improve quality of life for patients with this debilitating condition, and reduce pressure on transplant waiting lists. The study also tests whether switching from capsules to tablets improves patient comfort and adherence, a practical change that could make daily treatment easier to sustain.

View original technical description
During the development of norucholic acid (NCA) over 700 patients have been treated with this new medication so far. This study, called NUT-022/PSC is a kind of follow-up study for the NUC-5/PSC study; it offers continuous NCA treatment to patients from the NUC-5/PSC study. Another goal of the current study is to gather more information about the safety and efficacy of NCA treatment for PSC patients over a longer time period. This is an open-label, single-arm study. Open-label means that the label of the study medication is not hidden, and that participants know which treatment they get. Single-arm means that all participants receive the same treatment. To be able to take part, patients must have participated in the previous NUC-5/PSC study. If patients are eligible, they will be asked if they would like to enrol in the NUT-022/PSC study. They will receive the medication NCA at a dose of 1500 mg per day, taken as three tablets (containing 500 mg NCA each), instead of the 6 capsules (250mg) in NUC-5/PSC study. This should make it easier and more comfortable for them to take the medication. Earlier studies showed that the capsules and the tablets both work in the body in the same way. The patient will come to regular interim visits every 3 months. The final visit will take place at the end of the treatment phase or at the time of participant’s withdrawal. Participants will have a total of 8 visits over a period of up to 72 weeks. Diagnostic procedures and examinations done in this study are mostly routine or non-invasive procedures. However, taking blood samples may cause discomfort or even clotting of the vein or nerve injury in rare cases. The total amount of blood withdrawn per visit is about 30 ml. Imaging (like ultrasound) is a non-invasive procedure. The trial will take place at 6 sites in UK which previously participated in the NUC-5/PSC study. Around 125 patients are expected to join globally and 20 expected in UK

Researchers

Palak Trivedi (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

Double-blind, randomized, placebo-controlled, phase III study comparing norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis(NUC-5 )
An open-label study of patients with primary sclerosing cholangitis (PSC) treatedwith norucholic acid tablets
Pharmacokinetic (PK) study to investigate the pharmacokinetic profile of norucholic acid (NCA) in primary sclerosing cholangitis (PSC) patients treated with NCA capsules within open-label extension (OLE) NUC-5/PSC (NUC-011)
NUT-2 - Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of norucholic acid tablets with placebo in the treatment of primary biliary cholangitis in patients with an inadequate response to ursodeoxycholic acid
Double-blind, randomised, placebo-controlled, phase IIb trial on the efficacy and safety of norursodeoxycholic acid tablets in patients with non-alcoholic steatohepatitis (NASH) (NUT3 STUDY)

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