RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

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)

Quick Facts

Study Start:2024-01-15
Study Completion:2025-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05919680

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.

Ages Eligible for Study:16 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Adult persons aged 16 years or older at the time of informed consent.
  2. * Minimum of 6 months on Parenteral supplementation.
  3. * Established clinical diagnosis of IFALD based on a persistent elevation of
  4. 1. liver enzymes (ALP, AST, ALT, or GGT ≥1.5 × upper limit of normal \[ULN\]) for ≥6 months and/or
  5. 2. total bilirubin \> ULN for ≥6 months.
  6. * Laboratory parameters consistent with stable liver disease without cirrhosis as defined by:
  7. 1. ALT and AST \<5 × ULN;
  8. 2. Total bilirubin ≤2.5 mg/dL in the absence of Gilbert's Syndrome.
  9. 3. Serum albumin ≥2.5 g/dL;
  10. 4. International normalized ratio (INR) ≤1.3 in the absence of anticoagulant therapy;
  11. 5. Platelet count ≥120,000/mm3.
  1. * Clinical, laboratory, imaging, or histopathologic evidence of other causes of acute or chronic liver disease, including autoimmune, viral, metabolic, or alcoholic liver disease.
  2. * 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).
  3. * Presence of hepatic impairment, end-stage liver disease, and/or a model for end-stage liver disease (MELD) score \>12.
  4. * Transient elastography read \>20.0 kPA within 3 months prior to or during the Screening Period.
  5. * Estimated glomerular filtration rate \<45 mL/min based on the 2021 CKD-EPI creatinine equation.
  6. * Poor nutritional status defined as body mass index (BMI) \<17 kg/m2.

Contacts and Locations

Study Contact

Michelle Yokley
CONTACT
+31 (0) 35 760 65 05
michelle.yokley@northseatherapeutics.com

Study Locations (Sites)

Mayo Clinic Scottsdale Campus
Scottsdale, Arizona, 85259
United States
University of California San Francisco Medical Center
San Francisco, California, 94143
United States
MedStar Georgetown University Hospital
Washington, District of Columbia, 20007
United States
Emory University School of Medicine
Atlanta, Georgia, 30322
United States
The University of Chicago Medical Center
Chicago, Illinois, 60637
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Henry Ford Hospital
Detroit, Michigan, 48202
United States
Mayo Clinic Rochester Campus
Rochester, Minnesota, 55905
United States
Mount Sinai Medical Center
New York, New York, 10029
United States
Duke University Medical Center
Durham, North Carolina, 27710
United States
The Cleveland Clinic
Cleveland, Ohio, 44195
United States
Vanderbilt University School of Medicine
Nashville, Tennessee, 37232
United States
University of Washington
Seattle, Washington, 98195
United States

Collaborators and Investigators

Sponsor: NorthSea Therapeutics B.V.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-15
Study Completion Date2025-06-30

Study Record Updates

Study Start Date2024-01-15
Study Completion Date2025-06-30

Terms related to this study

Additional Relevant MeSH Terms

  • Intestinal Failure Associated Liver Disease