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Orziloben, also known by its development code NST-6179, is an investigational substance that has not been authorised as a medicine in the trial records. The name orziloben is used for the active substance in the clinical trial described on this page. The substance is being developed for people with intestinal failure-associated liver disease, a condition that can affect patients who need long-term parenteral nutrition.[1]
The registry record for the Phase 2a trial does not describe a specific molecular target or mechanism of action for orziloben. The study was designed to assess pharmacodynamic effects on non-invasive biomarkers of hepatic steatosis, hepatic inflammation, hepatic cholestasis, hepatic fibrosis, lipid metabolism, glucose metabolism, bile acid metabolism, circulating incretins, and intestinal function and injury.[1] These biomarker measurements are intended to show whether the substance changes biological processes linked to liver damage in IFALD, but the trial record does not state how orziloben produces any such effects.
In the Phase 2a trial, orziloben was given as an oral solution. The placebo used for comparison was described as a clear colourless to light yellow oral solution. The trial record does not report a specific dose for orziloben, because the intervention entry lists 0 mg, meaning the dose was not reported in the register.[1]
One completed Phase 2a trial of orziloben is registered. The study was randomised, double-blind, and placebo-controlled, with 18 participants. It was divided into two parts: Part A assessed safety, tolerability, and pharmacokinetics over 4 weeks, while Part B extended treatment to 12 weeks and also evaluated pharmacodynamic effects.[1]
The primary outcomes included safety and tolerability measures such as adverse events, laboratory parameters, vital signs, electrocardiograms, physical examinations, and the percentage of participants discontinuing due to treatment-emergent adverse events. Pharmacokinetic parameters included the area under the concentration-time curve from time 0 to last measurable concentration (AUC0-last), maximum observed concentration (Cmax), and time to reach Cmax (Tmax) on Day 1 and Day 14 in Part A, and on Day 1 and Day 28 in Part B.[1]
The study also aimed to evaluate the effect of colon function on the pharmacokinetic profile of orziloben and to assess exposure-response relationships by measuring changes in pharmacodynamic biomarkers. The trial is listed as completed, but the registry record does not report results.[1]
The registry record for this trial does not report side effects or safety findings from the completed study. The trial measured safety and tolerability, including adverse events and discontinuations due to treatment-emergent adverse events, but no results are available in the data provided.
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