A Study of NST-6179 in Subjects with Intestinal Failure-Associated Liver Disease (IFALD).

Description

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.

Conditions

Intestinal Failure Associated Liver Disease

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Study Overview

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Study Details

Study 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.

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)

A Study of NST-6179 in Subjects with Intestinal Failure-Associated Liver Disease (IFALD).

Condition
Intestinal Failure Associated Liver Disease
Intervention / Treatment

-

Contacts and Locations

Scottsdale

Mayo Clinic Scottsdale Campus, Scottsdale, Arizona, United States, 85259

San Francisco

University of California San Francisco Medical Center, San Francisco, California, United States, 94143

Washington

MedStar Georgetown University Hospital, Washington, District of Columbia, United States, 20007

Atlanta

Emory University School of Medicine, Atlanta, Georgia, United States, 30322

Chicago

The University of Chicago Medical Center, Chicago, Illinois, United States, 60637

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Detroit

Henry Ford Hospital, Detroit, Michigan, United States, 48202

Rochester

Mayo Clinic Rochester Campus, Rochester, Minnesota, United States, 55905

New York

Mount Sinai Medical Center, New York, New York, United States, 10029

Durham

Duke University Medical Center, Durham, North Carolina, United States, 27710

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

16 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

NorthSea Therapeutics B.V.,

Study Record Dates

2025-06-30