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

RecruitingPhase 2Ages 16 Years - anyAllNorthSea Therapeutics B.V.
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ฅ1.5 ร— upper limit of normal \[ULN\]) for โ‰ฅ6 months and/or 2. total bilirubin \> ULN for โ‰ฅ6 months. * Laboratory parameters consistent with stable liver disease without cirrhosis as defined by: 1. ALT and AST \<5 ร— ULN; 2. Total bilirubin โ‰ค2.5 mg/dL in the absence of Gilbert's Syndrome. 3. Serum albumin โ‰ฅ2.5 g/dL; 4. International normalized ratio (INR) โ‰ค1.3 in the absence of anticoagulant therapy; 5. Platelet count โ‰ฅ120,000/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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What it tests

  • NST-6179 Part A
  • NST-6179 Part B
  • Matched Placebo
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Where

13 sites
Mayo Clinic Scottsdale Campus
Scottsdale, United States
University of California San Francisco Medical Center
San Francisco, United States
MedStar Georgetown University Hospital
Washington D.C., United States
Emory University School of Medicine
Atlanta, United States
The University of Chicago Medical Center
Chicago, United States
Boston Children's Hospital
Boston, United States
Henry Ford Hospital
Detroit, United States
Mayo Clinic Rochester Campus
Rochester, United States

โ€ฆand 5 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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