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Study Comparing Tenofovir Disoproxil Fumarate (TDF), Emtricitabine (FTC)/TDF, and Entecavir (ETV) in the Treatment of Chronic HBV in Subjects With Decompensated Liver Disease.

Phase 2
Completed
Conditions
Chronic Hepatitis B
Interventions
Drug: Emtricitabine/tenofovir disoproxil fumarate (FTC/TDF)
Drug: TDF placebo
Drug: FTC/TDF placebo
Drug: ETV placebo
Registration Number
NCT00298363
Lead Sponsor
Gilead Sciences
Brief Summary

This study was designed to evaluate and compare the safety and tolerability of tenofovir disoproxil fumarate (TDF), emtricitabine (FTC)/TDF, and entecavir (ETV) in the treatment of hepatitis B patients with decompensated liver disease. Safety was assessed by evaluating adverse events (AEs) and laboratory abnormalities. Efficacy was assessed by evaluating reductions in Child-Pugh-Turcotte (CPT) and Model for End Stage Liver Disease (MELD) scores, reductions in hepatitis B virus (HBV) deoxyribonucleic acid (DNA), changes in liver enzymes, development of drug-resistant mutations, and generation of antibody to virus.

A maximum randomized treatment duration of 168 weeks was planned. Since subjects with decompensated liver disease were enrolled into this study, it was necessary to provide early intervention strategies if profound viral suppression was not expeditiously achieved. For this reason, subjects with a decrease in plasma HBV DNA from baseline of \< 2 log_10 copies/mL and plasma HBV DNA \> 10,000 copies/mL (or plasma HBV DNA \> 1,000 copies/mL for subjects who entered the study with HBV DNA \< 10,000 copies/mL) at Week 8 had the option to start open-label FTC/TDF and continue in the study. Subjects with a virologic breakthrough or who had plasma HBV DNA levels remaining \> 400 copies/mL (confirmed) at or after 24 weeks of treatment could have been unblinded at the investigator's discretion for selection of alternative anti-HBV therapy that may have included open-label FTC/TDF. If study drug was permanently discontinued, immediate initiation of another anti-HBV regimen was strongly recommended.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
112
Inclusion Criteria

A participant was required to meet all of the following inclusion criteria to be eligible for participation in the study:

  • Chronic Hepatitis B infection

  • 18 through 69 years of age, inclusive

  • HBV DNA ≥ 1000 copies/mL

  • Decompensated liver disease with all of the following:

    • CPT score of 7-12 (inclusive) OR history of CPT score ≥ 7 and any CPT at screen ≤ 12
    • Serum alanine aminotransferase (ALT) < 10 x the upper limit of the normal range (ULN)
    • Hemoglobin ≥ 7.5 g/dL
    • Total white blood cell (WBC) count ≥ 1,500/mm^3
    • Platelet count ≥ 30,000/mm^3
  • Alpha-fetoprotein ≤ 20 ng/mL and ultrasound or other imaging with no evidence of hepatocellular carcinoma (HCC), or alpha-fetoprotein of 21-50 ng/mL and computed tomography (CT)/magnetic resonance imaging (MRI) scan with no evidence of HCC, within 6 months of screening

  • Calculated creatinine clearance ≥ 50 mL/min

  • Negative human immunodeficiency virus (HIV), hepatitis C virus (HCV), and hepatitis D virus (HDV) serologies

  • Less than 24 months of total prior adefovir dipivoxil exposure

  • Willing and able to provide written informed consent

Exclusion Criteria

A participant who met any of the following exclusion criteria could not be enrolled in the study:

  • Pregnant women, women who were breastfeeding or who believed they may have wished to become pregnant during the course of the study
  • Males and females of reproductive potential who were unwilling to use an effective method of contraception during the study
  • Prior use of TDF or ETV
  • History of variceal bleeding, hepatorenal syndrome, Grade 3 or 4 hepatic encephalopathy, or spontaneous bacterial peritonitis within 60 days of screening
  • Grade 2 hepatic encephalopathy at screening
  • History of solid organ or bone marrow transplant
  • Current use of hepatotoxic drugs, nephrotoxic drugs, or drugs that interfere with renal tubular secretion
  • Current therapy with immunomodulators (eg, corticosteroids, interleukin-2, etc.) or investigational drugs
  • Diagnosis of proximal tubulopathy
  • Use of investigational agent within 30 days prior to screening
  • Known hypersensitivity to TDF, FTC, ETV, or formulation excipients of any of the study drug products

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
FTC/TDFTDF placeboFTC 200 mg/TDF 300 mg + TDF placebo + ETV placebo QD
FTC/TDFETV placeboFTC 200 mg/TDF 300 mg + TDF placebo + ETV placebo QD
EntecavirEntecavir (ETV)ETV 0.5 mg or 1 mg + TDF placebo + FTC/TDF placebo QD
Tenofovir DFTenofovir disoproxil fumarate (tenofovir DF; TDF)TDF 300 mg + FTC/TDF placebo + ETV placebo once daily (QD)
Tenofovir DFFTC/TDF placeboTDF 300 mg + FTC/TDF placebo + ETV placebo once daily (QD)
Tenofovir DFETV placeboTDF 300 mg + FTC/TDF placebo + ETV placebo once daily (QD)
FTC/TDFEmtricitabine/tenofovir disoproxil fumarate (FTC/TDF)FTC 200 mg/TDF 300 mg + TDF placebo + ETV placebo QD
EntecavirTDF placeboETV 0.5 mg or 1 mg + TDF placebo + FTC/TDF placebo QD
EntecavirFTC/TDF placeboETV 0.5 mg or 1 mg + TDF placebo + FTC/TDF placebo QD
Primary Outcome Measures
NameTimeMethod
Percent Probability of Tolerability FailureBaseline to Week 168

Tolerability failure was defined as permanent discontinuation of study drug due to a treatment-emergent adverse event (AE), including any subject who temporarily discontinued study drug due to an AE and did not restart. Results are expressed as proportions of participants who experience tolerability failure using the Kaplan-Meier (KM) method of estimation.

Percent Probability of a Confirmed Increase in Serum Creatinine of ≥ 0.5 mg/dL From Baseline or a Confirmed Serum Phosphorus Level < 2.0 mg/dLBaseline to Week 168

Results are expressed as proportions of participants who experience a confirmed increase in serum creatinine of ≥ 0.5 mg/dL from baseline or a confirmed serum phosphorus level \< 2.0 mg/dL using the KM method of estimation.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With HBsAg Loss and HBsAg Seroconversion at Week 168Baseline to Week 168

Loss of HBsAg was defined as change of detectable HBsAg from positive to negative. HBsAg seroconversion was defined as change of detectable antibody to HBsAg from negative to positive.

Median DAVG in Plasma HBV DNA Levels at 144 Weeks Relative to BaselineBaseline to 144 weeks

Change from baseline was evaluated by subtracting baseline HBV DNA log_10 copies/mL from Week 144 HBV DNA log_10 copies/mL. DAVG is defined as the area of the trapezoid under the response-time curve divided by time to the last available evaluation of the patient minus the baseline value.

Percentage of Participants With Normalized ALT (for Subjects With Elevated ALT at Baseline) at Week 96Baseline to Week 96

Normalized ALT is defined as having a baseline ALT value \> ULN, and a decrease in ALT value to ≤ ULN at the given time point.

Percentage of Participants With an Increase in CPT Score of ≥ 2 Points at Week 144Baseline to Week 144

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Percentage of Participants With Hepatitis B Early Antigen (HBeAg) Loss and HBeAg Seroconversion at Week 48 (for Participants Who Were HBeAg Positive at Baseline)Baseline to Week 48

Loss of HBeAg was defined as change of detectable HBeAg from positive to negative. HBeAg seroconversion was defined as change of detectable antibody to HBeAg from negative to positive.

Percentage of Participants With HBeAg Loss and HBeAg Seroconversion at Week 96 (for Participants Who Were HBeAg Positive at Baseline)Baseline to Week 96

Loss of HBeAg was defined as change of detectable HBeAg from positive to negative. HBeAg seroconversion was defined as change of detectable antibody to HBeAg from negative to positive.

Percentage of Participants With HBeAg Loss and HBeAg Seroconversion at Week 168 (for Participants Who Were HBeAg Positive at Baseline)Baseline to Week 168

Loss of HBeAg was defined as change of detectable HBeAg from positive to negative. HBeAg seroconversion was defined as change of detectable antibody to HBeAg from negative to positive.

Percentage of Participants With Hepatitis B Surface Antigen (HBsAg) Loss and HBsAg Seroconversion at Week 48Baseline to Week 48

Loss of HBsAg was defined as change of detectable HBsAg from positive to negative. HBsAg seroconversion was defined as change of detectable antibody to HBsAg from negative to positive.

Median DAVG in Plasma HBV DNA Levels at 168 Weeks Relative to BaselineBaseline to 168 weeks

Change from baseline was evaluated by subtracting baseline HBV DNA log_10 copies/mL from Week 168 HBV DNA log_10 copies/mL. DAVG is defined as the area of the trapezoid under the response-time curve divided by time to the last available evaluation of the patient minus the baseline value.

Percentage of Participants With Plasma HBV DNA < 400 Copies/mL at Week 96Week 96

The percentage of participants with plasma HBV DNA \< 400 copies/mL at Week 96 was summarized.

Percentage of Participants With Plasma HBV DNA < 400 Copies/mL at Week 168Week 168

The percentage of participants with plasma HBV DNA \< 400 copies/mL at Week 168 was summarized.

Percentage of Participants With Normalized ALT (for Subjects With Elevated ALT at Baseline) at Week 168Baseline to Week 168

Normalized ALT is defined as having a baseline ALT value \> ULN, and a decrease in ALT value to ≤ ULN at the given time point.

Percentage of Participants With HBsAg Loss and HBsAg Seroconversion at Week 96Baseline to Week 96

Loss of HBsAg was defined as change of detectable HBsAg from positive to negative. HBsAg seroconversion was defined as change of detectable antibody to HBsAg from negative to positive.

Median DAVG in Plasma HBV DNA Levels at 96 Weeks Relative to BaselineBaseline to 96 weeks

Change from baseline was evaluated by subtracting baseline HBV DNA log_10 copies/mL from Week 96 HBV DNA log_10 copies/mL. DAVG is defined as the area of the trapezoid under the response-time curve divided by time to the last available evaluation of the patient minus the baseline value.

Percentage of Participants With Plasma HBV DNA < 400 Copies/mL at Week 48Week 48

The percentage of participants with plasma HBV DNA \< 400 copies/mL at Week 48 was summarized.

Percentage of Participants With an Increase in Child-Pugh Turcotte (CPT) Score of ≥ 2 Points at Weeks 48Baseline to Week 48

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Percentage of Participants With an Increase in CPT Score of ≥ 2 Points at Week 168Baseline to Week 168

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Median Change in MELD Score From Baseline at Week 96Baseline to Week 96

MELD scores, used to assess prognosis and suitability for transplant, are calculated based on laboratory values only and can range from 6 to 40, with higher scores indicating greater disease severity.

Percentage of Participants With HBeAg Loss and HBeAg Seroconversion at Week 144 (for Participants Who Were HBeAg Positive at Baseline)Baseline to Week 144

Loss of HBeAg was defined as change of detectable HBeAg from positive to negative. HBeAg seroconversion was defined as change of detectable antibody to HBeAg from negative to positive.

Median Time-averaged Change (DAVG) in Plasma Hepatitis B Virus (HBV) Deoxyribonucleic Acid (DNA) Levels at 48 Weeks Relative to BaselineBaseline to 48 weeks

Change from baseline was evaluated by subtracting baseline HBV DNA log_10 copies/mL from Week 48 HBV DNA log_10 copies/mL. DAVG is defined as the area of the trapezoid under the response-time curve divided by time to the last available evaluation of the patient minus the baseline value.

Percentage of Participants With Plasma HBV DNA < 400 Copies/mL at Week 144Week 144

The percentage of participants with plasma HBV DNA \< 400 copies/mL at Week 144 was summarized.

Percentage of Participants With Normalized Alanine Aminotransferase (ALT) (for Subjects With Elevated ALT at Baseline) at Week 48Baseline to Week 48

Normalized ALT is defined as having a baseline ALT value \> the upper limit of the normal range (ULN), and a decrease in ALT value to ≤ ULN at the given time point.

Percentage of Participants With a Decrease in CPT Score of ≥ 2 Points From Baseline at Week 96Baseline to Week 96

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Median Change in Model for End-Stage Liver Disease (MELD) Score From Baseline at Week 48Baseline to Week 48

MELD scores, used to assess prognosis and suitability for transplant, are calculated based on laboratory values only and can range from 6 to 40, with higher scores indicating greater disease severity.

Percentage of Participants With HBsAg Loss and HBsAg Seroconversion at Week 144Baseline to Week 144

Loss of HBsAg was defined as change of detectable HBsAg from positive to negative. HBsAg seroconversion was defined as change of detectable antibody to HBsAg from negative to positive.

Percentage of Participants With Normalized ALT (for Subjects With Elevated ALT at Baseline) at Week 144Baseline to Week 144

Normalized ALT is defined as having a baseline ALT value \> ULN, and a decrease in ALT value to ≤ ULN at the given time point.

Percentage of Participants With an Increase in CPT Score of ≥ 2 Points at Week 96Baseline to Week 96

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Percentage of Participants With a Decrease in CPT Score of ≥ 2 Points From Baseline at Week 48Baseline to Week 48

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Percentage of Participants With a Decrease in CPT Score of ≥ 2 Points From Baseline at Week 144Baseline to Week 144

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Percentage of Participants With a Decrease in CPT Score of ≥ 2 Points From Baseline at Week 168Baseline to Week 168

CPT scores, widely used to grade the severity of cirrhosis and to determine the need for liver transplantation, are calculated based on a combination of laboratory values and clinical features. CPT scores can range from 5 to 15, with higher scores indicating a greater severity of disease.

Median Change in MELD Score From Baseline at Week 144Baseline to Week 144

MELD scores, used to assess prognosis and suitability for transplant, are calculated based on laboratory values only and can range from 6 to 40, with higher scores indicating greater disease severity.

Median Change in MELD Score From Baseline at Week 168Baseline to Week 168

MELD scores, used to assess prognosis and suitability for transplant, are calculated based on laboratory values only and can range from 6 to 40, with higher scores indicating greater disease severity.

In the Subset of Participants Undergoing Liver Transplantation, Time to Recurrence of Hepatitis B, Defined as 2 Consecutive Plasma HBV DNA Concentrations ≥ 400 Copies/mL or 2 Consecutive HBsAg(+) ResultsBaseline to Week 168

Trial Locations

Locations (38)

Wojewodzki Szpital Zakazny

🇵🇱

Warsaw, Poland

Hospital Clinic i Provincial de Barcelona (HCPB)

🇪🇸

Barcelona, Spain

Hospital General Universitario Gregorio Maranon

🇪🇸

Madrid, Spain

Pfleger Liver Institute

🇺🇸

Los Angeles, California, United States

California Pacific Medical Center Research Institute

🇺🇸

San Francisco, California, United States

University of Miami, Center for Liver Diseases

🇺🇸

Miami, Florida, United States

Henry Ford Hospital and Health System

🇺🇸

Detroit, Michigan, United States

Metropolitan Research

🇺🇸

Fairfax, Virginia, United States

Virginia Mason Medical Center

🇺🇸

Seattle, Washington, United States

Toronto General Hospital

🇨🇦

Toronto, Ontario, Canada

Wojewodzki Szpital Specjalistyczny im Dluskeigo

🇵🇱

Bialystok, Poland

Tan Tock Seng Hospital

🇸🇬

Singapore, Singapore

Vancouver General Hospital

🇨🇦

Vancouver, British Columbia, Canada

General Hospital of Athens "Ippokratio"

🇬🇷

Athens, Greece

Singapore General Hospital

🇸🇬

Singapore, Singapore

Changi General Hospital

🇸🇬

Singapore, Singapore

National Cheng Kung University Hospital

🇨🇳

Tainan, Taiwan

The Gordon & Leslie Diamond Centre

🇨🇦

Vancouver, British Columbia, Canada

Wojewodzki Szpital Obserwacy

🇵🇱

Bydgoszcz, Poland

Chang Gung Memorial Hospital - Kaohsiung

🇨🇳

Kaoshiung Hsien, Taiwan

Chang-Gung Memorial Hospital

🇨🇳

Taipei City, Taiwan

Cathay General Hospital

🇨🇳

Taipei, Taiwan

Rush Presbyterian - St. Luke's Medical Center

🇺🇸

Chicago, Illinois, United States

Columbia Presbyterian Medical Center

🇺🇸

New York, New York, United States

Mt. Sinai School of Medicine/ Mt. Sinai Medical Center

🇺🇸

New York, New York, United States

Heritage Medical Research Clinic

🇨🇦

Calgary, Alberta, Canada

Hopital Conception

🇫🇷

Marseille, France

Medizinische Klinik mit Schwerpunkt Hepatologie und Gastroenterologie

🇩🇪

Berlin, Germany

Universitat Heidelberg

🇩🇪

Heidelberg, Germany

Johannes Gutenberg-Universitat

🇩🇪

Mainz, Germany

Universita de Padova

🇮🇹

Padova, Italy

Policlinico Universitario

🇮🇹

Udine, Italy

National University Hospital Dept. of Gastroenterology & Hepatology

🇸🇬

Singapore, Singapore

Ege Universitesi Tip Fakultesi Hastanesi

🇹🇷

Izmir, Turkey

Hospital General Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital Universitario de Bellvitge

🇪🇸

Barcelona, Spain

Hospital Universitario y Politecnico la Fe

🇪🇸

Valencia, Spain

Marmara Universitesi School of Medicine

🇹🇷

Istanbul, Turkey

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