Recruiting
Phase 2

NST-6179

Sponsor:

NorthSea Therapeutics B.V.

Code:

NCT05919680

Conditions

Intestinal Failure Associated Liver Disease

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

NST-6179 Part A

NST-6179 Part B

Matched Placebo

Study Details

Brief summary:

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

Study ID

NCT05919680

Start date

Jan 15, 2024

Status verified date

Jan, 2025

Completion date

Jun 30, 2025

Anticipated

Primary completion date

Jun 30, 2025

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

36 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part A-800 mg NST-6179

up to 12 subjects

experimental: Part A matched NST-6179 placebo

up to 6 subjects

experimental: Part B- 1200mg NST-6179

up to 12 subjects

experimental: Part B matched NST-6179 placebo

up to 6 subjects

Interventions

NST-6179 Part A

Once daily (QD) oral administration of 800mg (32 mL solution) of NST-6179 for 4 weeks

NST-6179 Part B

Once daily (QD) oral administration of 1200mg of NST-6179 for 12 weeks

Matched Placebo

Matched placebo for administration in Part A or Part B

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

Michelle Yokley

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

Locations

Mayo Clinic Scottsdale Campus

Recruiting

Scottsdale, Arizona, United States, 85259

University of California San Francisco Medical Center

Recruiting

San Francisco, California, United States, 94143

Emory University School of Medicine

Recruiting

Atlanta, Georgia, United States, 30322

The University of Chicago Medical Center

Recruiting

Chicago, Illinois, United States, 60637

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Henry Ford Hospital

Recruiting

Detroit, Michigan, United States, 48202

Mayo Clinic Rochester Campus

Recruiting

Rochester, Minnesota, United States, 55905

Mount Sinai Medical Center

Recruiting

New York, New York, United States, 10029

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

The Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Vanderbilt University School of Medicine

Recruiting

Nashville, Tennessee, United States, 37232

University of Washington

Recruiting

Seattle, Washington, United States, 98195

More Information

Sponsor

NorthSea Therapeutics B.V.

Last update posted

Jan 7, 2025

Last verified

Jan, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by NorthSea Therapeutics B.V. on 2025-01-07.