A Study of NST-6179 in Subjects With Intestinal Failure-Associated Liver Disease (IFALD).
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: NST-6179 Part A, NST-6179 Part B, Matched Placebo.
- Who it may be relevant to
- Registry conditions: Intestinal Failure Associated Liver Disease. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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)
Overview
This is a phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NST-6179 in subjects with intestinal failure-associated liver disease (IFALD) receiving parenteral nutrition (PN). The study will be conducted in 2 sequential parts. Up to 36 subjects diagnosed with IFALD will be enrolled in the study, of which up to 18 subjects will be enrolled in each of the 2 parts and randomized (2:1) to receive NST-6179 (N=12/part) or matched placebo (N=6/part). Subjects in Part A will receive once daily (QD) oral administration of 800 mg (32 mL solution) NST-6179 or placebo for 4 weeks. The NST-6179 dose for Part B is planned to be 1200 mg QD for 12 weeks. Actual dose, however, will be determined during the safety review meeting.
Interventions
- Drug NST-6179 Part A
Once daily (QD) oral administration of 800mg (32 mL solution) of NST-6179 for 4 weeks - Drug NST-6179 Part B
Once daily (QD) oral administration of 1200mg of NST-6179 for 12 weeks - Other Matched Placebo
Matched placebo for administration in Part A or Part B
Primary outcome measures
- To assess the safety and tolerability of NST-6179 [Time frame: Up to 14 Weeks]
- To assess the pharmacokinetics of NST-6179 [Time frame: Day 1 and Day 14]
- To assess the pharmacodynamic effects of NST-6179 on hepatic steatosis [Time frame: 12 weeks]
- To assess the pharmacodynamic effects of NST-6179 on hepatic inflammation [Time frame: 12 weeks]
- To assess the pharmacodynamic effects of NST-6179 on hepatic cholestasis (bilirubin, ALP, GGT) [Time frame: 12 weeks]
- To assess the pharmacodynamic effects of NST-6179 on hepatic fibrosis (ELF, Pro-C3, FIB-4) [Time frame: 12 weeks]
Eligibility criteria
Inclusion criteria
- Adult persons aged 16 years or older at the time of informed consent.
- Minimum of 6 months on Parenteral supplementation.
- Established clinical diagnosis of IFALD based on a persistent elevation of
- liver enzymes (ALP, AST, ALT, or GGT ≥1.5 × upper limit of normal \[ULN\]) for ≥6 months and/or
- total bilirubin > ULN for ≥6 months.
- Laboratory parameters consistent with stable liver disease without cirrhosis as defined by:
- ALT and AST <5 × ULN;
- Total bilirubin ≤2.5 mg/dL in the absence of Gilbert's Syndrome.
- Serum albumin ≥2.5 g/dL;
- International normalized ratio (INR) ≤1.3 in the absence of anticoagulant therapy;
- Platelet count ≥120,000/mm3.
Exclusion criteria
- 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.
- Transient elastography read >20.0 kPA within 3 months prior to or during the Screening Period.
- Estimated glomerular filtration rate <45 mL/min based on the 2021 CKD-EPI creatinine equation.
- Poor nutritional status defined as body mass index (BMI) <17 kg/m2.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Sequential
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 13 centers
- Mayo Clinic Scottsdale Campus — Scottsdale
- University of California San Francisco Medical Center — San Francisco
- MedStar Georgetown University Hospital — Washington D.C.
- Emory University School of Medicine — Atlanta
- The University of Chicago Medical Center — Chicago
- Boston Children's Hospital — Boston
- Henry Ford Hospital — Detroit
- Mayo Clinic Rochester Campus — Rochester
- … and 5 more centers
Identifiers
NCT: NCT05919680 · NST-6179-02