A study evaluating Orziloben (NST-6179) in Subjects with Intestinal Failure-Associated Liver Disease
Research type
Research Study
Full title
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Orziloben (NST-6179) in Subjects with Intestinal Failure-Associated Liver Disease (IFALD)
IRAS ID
1011574
Contact name
Rob de Ree
Contact email
Sponsor organisation
NorthSea Therapeutics B.V.
Eudract number
2024-520202-19
Clinicaltrials.gov Identifier
Research summary
Intestinal Failure-Associated Liver Disease (IFALD) is a rare liver disease that can develop in people who cannot get enough nutrients from food because their intestines are not working properly. To stay healthy, they need a treatment called parenteral nutrition (PN), in which nutrients are given directly into the blood stream. Over time, this can put extra strain on the liver and cause damage. NorthSea Therapeutics B.V. is developing an experimental medicine called Orziloben for the treatment of IFALD. The purpose of this study is to find out if Orziloben is safe and effective in subjects with IFALD, how Orziloben behaves in the body (pharmacokinetics), and how Orziloben impacts the body and the symptoms of IFALD (pharmacodynamics).
Up to 36 people in total will take part in this study at several different locations. The study will have 2 parts: Part A and Part B.
The study will include an up to 30-day Screening Period, a 4-week (Part A) and 12-week (Part B) Treatment Period, and a 2-week Follow-Up Period. This study uses a placebo control, which is a ‘dummy’ solution that does not contain the medicine Orziloben but looks just like it. The decision to give a person medicine or placebo will be made by chance. People will have a 2 in 3 chance of getting Orziloben and a 1 in 3 chance of getting placebo. Neither the people taking part in the trial nor the study doctors will know who has taken medicine or placebo. Each dose of study drug should be taken by mouth every day during the Treatment Period at the same time in the morning.
People taking part in this study must be men or women aged at least 18 years, have IFALD, and be on a stable PN therapy.
Safety assessments will include checking for health issues and side effects, physical examination, heart measurements, vital signs, electrical activity of the heart, and blood and urine collection for standard laboratory tests. Diagnostic assessments will include Fibroscan and magnetic resonance imaging (MRI).REC name
London - Westminster Research Ethics Committee
REC reference
25/LO/0573
Date of REC Opinion
3 Sep 2025
REC opinion
Further Information Favourable Opinion