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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)
EU CTIS ID: 2024-520202-19-00
What this study is testing
- Part A: To assess the safety and tolerability of multiple oral doses of NST-6179 for 4 weeks in subjects with IFALD. To characterize the PK of NST-6179 in subjects with IFALD. To evaluate the effect of colon function on the PK profile of NST-6179 in subjects with IFALD. - Part B: To assess the safety and tolerability of multiple oral doses of NST-6179 for 12 weeks in subjects with IFALD. To characterize the PK of NST-6179 in subjects with IFALD. To evaluate the effect of colon function on the PK profile of NST-6179 in subjects with IFALD. To assess the PD effects of NST-6179 in subjects with IFALD 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. To evaluate the exposure-response relationship of NST-6179 by assessing the changes in PD biomarkers.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Persons aged 18 years or older at the time of informed consent.
- Agree to use appropriate contraceptive methods based on biological sex and child-bearing potential.
- Agree to abstain from sperm or egg donation through 90 or 30 days, respectively, after administration of the last dose of IP.
- Able to understand the purpose and procedures that are involved in the study and willing to sign a written informed consent form and agrees to comply with the study protocol.
- Negative severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) result obtained from test performed according to site standard of care between Day -3 and Day 1.
- Minimum of 6 months on PN supplementation prior to Visit 1. Any PN prescription is allowed provided the PN is part of the participant’s caloric needs and the participant plans to stay on stable PN therapy throughout the Study.
You likely can't join if
- Major intestinal surgery within 3 months of screening or planned during the Study Period.
- The use of feeding tubes for the administration of study medication.
- Clinical, laboratory, imaging, or histopathologic evidence of other causes of acute or chronic liver disease, including autoimmune, viral, metabolic, or alcoholic liver disease.
- Clinical evidence of compensated or decompensated hepatic cirrhosis as assessed by historical liver histology, ultrasound-based and/or signs and symptoms of hepatic decompensation (including, but not limited to, jaundice, ascites, variceal hemorrhage, and/or hepatic encephalopathy).
- Presence of hepatic impairment, end-stage liver disease, and/or a model for end-stage liver disease (MELD) score >12.
- History of solid organ transplant (except for corneal transplant) or planned transplantation during the Study Period.
See the full eligibility criteria
- Persons aged 18 years or older at the time of informed consent.
- Agree to use appropriate contraceptive methods based on biological sex and child-bearing potential.
- Agree to abstain from sperm or egg donation through 90 or 30 days, respectively, after administration of the last dose of IP.
- Able to understand the purpose and procedures that are involved in the study and willing to sign a written informed consent form and agrees to comply with the study protocol.
- Negative severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) result obtained from test performed according to site standard of care between Day -3 and Day 1.
- Minimum of 6 months on PN supplementation prior to Visit 1. Any PN prescription is allowed provided the PN is part of the participant’s caloric needs and the participant plans to stay on stable PN therapy throughout the Study.
- Stable PN regimen for 4 weeks prior to and during the Screening Period. Stable regimen is defined as actual PN usage by volume and energy content and composition consistent with the prescribed regimen (±10%).
- Willingness to maintain current habitual level of physical activity, oral diet, and lifestyle throughout the entire study.
- Established clinical diagnosis of IFALD based on a persistent elevation of: a) liver enzymes: ALP and/or AST and/or ALT and/or GGT ≥1.5 × ULN for ≥6 months and/or b) total bilirubin > ULN for ≥6 months
- Major intestinal surgery within 3 months of screening or planned during the Study Period.
- The use of feeding tubes for the administration of study medication.
- Clinical, laboratory, imaging, or histopathologic evidence of other causes of acute or chronic liver disease, including autoimmune, viral, metabolic, or alcoholic liver disease.
- Clinical evidence of compensated or decompensated hepatic cirrhosis as assessed by historical liver histology, ultrasound-based and/or signs and symptoms of hepatic decompensation (including, but not limited to, jaundice, ascites, variceal hemorrhage, and/or hepatic encephalopathy).
- Presence of hepatic impairment, end-stage liver disease, and/or a model for end-stage liver disease (MELD) score >12.
- History of solid organ transplant (except for corneal transplant) or planned transplantation during the Study Period.
- Laboratory parameters consistent with unstable or deteriorating liver function as defined by: a. ALP ≥10 × ULN; b. ALT and AST ≥5 × ULN; c. Total bilirubin >2.5 mg/dL in the absence of Gilbert’s Syndrome. i. Gilbert’s Syndrome patients are eligible as long as the direct bilirubin is ≤0.5 mg/dL and ≤20% of the total bilirubin level.
- Transient elastography read >20.0 kPA within 3 months prior to or during the Screening Period.
- Estimated glomerular filtration rate <45 mL/min per 1.73 m2 based on the 2021 CKD-EPI creatinine equation
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.