Evaluating NST-6179 for liver disease related to intestinal failure

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)

This study is testing a new drug called NST-6179 to see if it can help adults with liver disease caused by intestinal failure who are on parenteral nutrition.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 16 Years and up
• Sex: All
• Sponsor: NorthSea Therapeutics B.V. (Industry-sponsored)
• Locations: 13 sites (Scottsdale, Arizona and 12 other locations)
• Trial ID: NCT05919680 on ClinicalTrials.gov

What this trial studies

This phase 2a clinical trial is designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of NST-6179 in adults with intestinal failure-associated liver disease (IFALD) who are on parenteral nutrition. The study will enroll up to 36 participants, who will be randomly assigned to receive either NST-6179 or a placebo in two parts, with varying dosages and durations. Part A involves a 4-week treatment period with a daily dose of 800 mg, while Part B extends to 12 weeks with a planned dose of 1200 mg. The trial aims to gather data on the drug's effects and safety profile in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 16 years or older who have been on parenteral nutrition for at least six months and have a clinical diagnosis of IFALD.

Not a fit: Patients with other underlying liver diseases or those with evidence of cirrhosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve liver function and overall health in patients suffering from IFALD.

How similar studies have performed: While this approach is novel in the context of IFALD, similar studies targeting liver diseases have shown varying degrees of success.

Eligibility criteria

Key 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

  1. liver enzymes (ALP, AST, ALT, or GGT ≥1.5 × upper limit of normal \[ULN\]) for ≥6 months and/or
  2. total bilirubin \> ULN for ≥6 months.
* Laboratory parameters consistent with stable liver disease without cirrhosis as defined by:

  1. ALT and AST \<5 × ULN;
  2. Total bilirubin ≤2.5 mg/dL in the absence of Gilbert's Syndrome.
  3. Serum albumin ≥2.5 g/dL;
  4. International normalized ratio (INR) ≤1.3 in the absence of anticoagulant therapy;
  5. Platelet count ≥120,000/mm3.

Key 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.

Where this trial is running

Scottsdale, Arizona and 12 other locations

• Mayo Clinic Scottsdale Campus — Scottsdale, Arizona, United States (Recruiting)
• University of California San Francisco Medical Center — San Francisco, California, United States (Recruiting)
• MedStar Georgetown University Hospital — Washington D.C., District of Columbia, United States (Not_yet_recruiting)
• Emory University School of Medicine — Atlanta, Georgia, United States (Recruiting)
• The University of Chicago Medical Center — Chicago, Illinois, United States (Recruiting)
• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• Henry Ford Hospital — Detroit, Michigan, United States (Recruiting)
• Mayo Clinic Rochester Campus — Rochester, Minnesota, United States (Recruiting)
• Mount Sinai Medical Center — New York, New York, United States (Recruiting)
• Duke University Medical Center — Durham, North Carolina, United States (Recruiting)
• The Cleveland Clinic — Cleveland, Ohio, United States (Recruiting)
• Vanderbilt University School of Medicine — Nashville, Tennessee, United States (Recruiting)
• University of Washington — Seattle, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Michelle Yokley

Conditions Intestinal Failure Associated Liver Disease