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

ID#: NCT05919680

Age: 18 years - 66+

Gender: All

Healthy Subjects: No

Study Phase: Phase 2

Recruitment Status: Recruiting

Start Date: January 01, 2024

End Date: June 30, 2025

Contact Information:
Michelle Yokley
+31 (0) 35 760 65 05
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 adult subjects with intestinal failure-associated liver disease (IFALD) receiving parenteral nutrition (PN). The study will be conducted in 2 sequential parts. Up to 36 adult subjects diagnosed with IFALD will be enrolled in the study, of which 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.
Eligibility: Key

Inclusion Criteria:

- Adult persons aged 18 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 ≤2.0 mg/dL in the absence of Gilbert's Syndrome.

3. Serum albumin ≥3 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.
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