Norucholic Acid Open-Label Treatment in PSC
Research type
Research Study
Full title
An open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets.
IRAS ID
1011119
Contact name
Michael Stiess
Contact email
Sponsor organisation
Dr Falk Pharma GmbH
Eudract number
2024-514292-18
ISRCTN Number
ISRCTN58756615
Research summary
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 The total amount of blood withdrawn per visit is about 30 ml.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
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
24/EM/0262
Date of REC Opinion
7 Jan 2025
REC opinion
Further Information Favourable Opinion