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Evaluation of HepQuant SHUNT to Assess Liver Disease; Substudy Within GS-US-416-2124

Early Phase 1
Withdrawn
Conditions
Severe Alcoholic Hepatitis
Interventions
Device: HepQuant SHUNT Test
Drug: Placebo
Registration Number
NCT03087968
Lead Sponsor
HepQuant, LLC
Brief Summary

This clinical investigation is a substudy within GS-US-416-2124, IND 129570, which is A Phase 2, Double-Blind, Randomized Study Evaluating the Safety, Tolerability, and Efficacy of GS-4997 in Combination with Prednisolone versus Prednisolone Alone in Subjects with Severe Alcoholic Hepatitis. The use of the HepQuant SHUNT test is to assess liver disease severity before, during, and after treatment with GS-4997 or placebo, to assess liver disease severity.

Detailed Description

The main study is a Phase 2, double blind, proof-of-concept, randomized study evaluating the safety, tolerability, and biological activity of GS-4997 in combination with prednisolone, compared to prednisolone alone, in subjects with severe, histologically-confirmed AH.

This substudy uses the HepQuant SHUNT Liver Diagnostic test to assess severity of disease at baseline and to track disease progression or improvement over the 24 weeks of the study. The HepQuant SHUNT test will be performed at baseline (Day 1) and at Weeks 1, 2, 4, 12, and 24 regardless of treatment Arm.

GS-4997 Dose and Mode of Administration. Subjects will be randomized 1:1 to either:

* Treatment Group A: GS-4997 18 mg (1 x 18 mg tablet) AND prednisolone 40 mg (4 x 10 mg tablets), both administered orally once daily

* Treatment Group B: GS-4997 placebo (1 tablet) AND prednisolone 40 mg (4 x 10 mg tablets), both administered orally once daily

Recruitment & Eligibility

Status
WITHDRAWN
Sex
All
Target Recruitment
Not specified
Inclusion Criteria
  1. Willing and able to give informed consent prior to any study specific procedures being performed. In individuals with hepatic encephalopathy (HE) which may impair decision-making, consent will be obtained per hospital procedures (eg, by Legally Authorized Representative)
  2. Clinical diagnosis of severe AH
  3. Maddrey's DF ≥ 32 at screening
Exclusion Criteria

Key Exclusion Criteria:

  1. Pregnant or lactating females;
  2. Other causes of liver disease including chronic hepatitis B (hepatitis B surface antigen [HBsAg] positive), chronic hepatitis C (HCV RNA positive), acetaminophen hepatotoxicity, biliary obstruction, and autoimmune liver disease;
  3. Serum AST >400 U/L or ALT >300 U/L;
  4. MELD >30 at screening;
  5. Maddrey's DF >60 at screening;
  6. Grade 4 Hepatic Encephalopathy (HE) by West Haven criteria;
  7. Concomitant or previous history of hepatocellular carcinoma;
  8. History of liver transplantation;
  9. HIV Ab positive;
  10. Clinical suspicion of pneumonia;
  11. Uncontrolled sepsis;
  12. Uncontrolled gastrointestinal (GI) bleeding or controlled GI bleeding within 7 days of screening that was associated with shock or required transfusion of more than 3 units of blood;
  13. Type 1 hepatorenal syndrome (HRS) or renal failure defined as a serum creatinine >221 μmol/L (>2.5 mg/dL) or the requirement for renal replacement therapy;
  14. Individuals dependent on inotropic (eg, epinephrine or norepinephrine) or ventilatory support (ie, endotracheal intubation or positive-pressure ventilation);
  15. Portal vein thrombosis;
  16. Acute pancreatitis;
  17. Cessation of alcohol consumption for more than 2 months before Baseline/ Day 1 NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
GS-4997 + PrednisoloneHepQuant SHUNT TestGS-4997 + Prednisolone for 28 days HepQuant SHUNT Test
Prednisolone + PlaceboHepQuant SHUNT TestPlacebo + Prednisolone for 28 days HepQuant SHUNT Test
Prednisolone + PlaceboPlaceboPlacebo + Prednisolone for 28 days HepQuant SHUNT Test
GS-4997 + PrednisoloneGS-4997GS-4997 + Prednisolone for 28 days HepQuant SHUNT Test
GS-4997 + PrednisolonePrednisoloneGS-4997 + Prednisolone for 28 days HepQuant SHUNT Test
Prednisolone + PlaceboPrednisolonePlacebo + Prednisolone for 28 days HepQuant SHUNT Test
Primary Outcome Measures
NameTimeMethod
To compare the change in (DSI ) Disease Severity Index between GS-4997 treatment and placebo armsHepQuant Shunt testing will be done at baseline (Day1), Week 1, Week 4, Week 12, Week 24

Using the SHUNT DSI to evaluate the liver, this outcome will compare the two arms to determine if SHUNT DSI is able to measure a change between the experimental and control groups.

Secondary Outcome Measures
NameTimeMethod
Secondary Outcome 1HepQuant Shunt testing will be done at baseline (Day1), Week 1, Week 4, Week 12, Week 24

To determine the relationship of baseline Disease Severity Index (DSI) to mortality risk;

Secondary Outcome 2HepQuant Shunt testing will be done at baseline (Day1), Week 1, Week 4, Week 12, Week 24

To determine the relationship of change in Disease Severity Index (DSI) to mortality risk

Secondary Outcome 3HepQuant Shunt testing will be done at baseline (Day1), Week 1, Week 4, Week 12, Week 24

To correlate baseline Disease Severity Index (DSI) with baseline Maddrey, MELD, and Lille scorestest with the pharmacokinetics of GS-4997

Secondary Outcome 4HepQuant Shunt testing will be done at baseline (Day1), Week 1, Week 4, Week 12, Week 24

To determine the relationship between baseline Disease Severity Index (DSI), Maddrey, MELD, and Lille scores and mortality

Trial Locations

Locations (12)

Emory University

🇺🇸

Atlanta, Georgia, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

The Liver Institute at Methodist Dallas Medical Center

🇺🇸

Dallas, Texas, United States

University of Miami

🇺🇸

Miami, Florida, United States

American Research Corporation at the Texas Liver Institute

🇺🇸

San Antonio, Texas, United States

Southern California Research Center

🇺🇸

Coronado, California, United States

University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

Intermountain Medical Center

🇺🇸

Murray, Utah, United States

University of Washington

🇺🇸

Seattle, Washington, United States

VCU Health System

🇺🇸

Richmond, Virginia, United States

Liver Institute of Virginia

🇺🇸

Richmond, Virginia, United States

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