Safety and Efficacy of Simtuzumab (SIM, GS-6624) in Adults With Advanced Liver Fibrosis But Not Cirrhosis Secondary to Non-Alcoholic Steatohepatitis (NASH)

NCT ID: NCT01672866

Last Updated: 2019-03-27

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

222 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-12-05

Study Completion Date

2016-12-29

Brief Summary

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The primary objective of this study is to evaluate whether SIM (formerly referred to as GS-6624) is effective at preventing the histologic progression of liver fibrosis and the clinical progression to cirrhosis in participants with NASH.

It will consist of 2 phases:

* Randomized Double-Blind Phase
* Open-Label Phase (optional)

Detailed Description

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Conditions

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Liver Fibrosis Due to NASH

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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SIM 75 mg

During the Randomized Double-Blind Phase, participants will receive SIM 75 mg via subcutaneous injection once weekly for up to 240 weeks. During the optional Open-Label Phase, participants will receive SIM 125 mg via subcutaneous injection once weekly for up to an additional 240 weeks.

Group Type EXPERIMENTAL

SIM

Intervention Type BIOLOGICAL

Subcutaneous injection every week

SIM 125 mg

During the Randomized Double-Blind Phase, participants will receive SIM 125 mg via subcutaneous injection once weekly for up to 240 weeks. During the optional Open-Label Phase, participants will receive SIM 125 mg via subcutaneous injection once weekly for up to an additional 240 weeks.

Group Type EXPERIMENTAL

SIM

Intervention Type BIOLOGICAL

Subcutaneous injection every week

Placebo

During the Randomized Double-Blind Phase, participants will receive placebo to match SIM via subcutaneous injection once weekly for up to 240 weeks. During the optional Open-Label Phase, participants will receive SIM 125 mg via subcutaneous injection once weekly for up to an additional 240 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type BIOLOGICAL

Placebo to match SIM via subcutaneous injection every week

SIM

Intervention Type BIOLOGICAL

Subcutaneous injection every week

Interventions

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Placebo

Placebo to match SIM via subcutaneous injection every week

Intervention Type BIOLOGICAL

SIM

Subcutaneous injection every week

Intervention Type BIOLOGICAL

Other Intervention Names

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GS-6624

Eligibility Criteria

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Inclusion Criteria

* Adults with chronic liver disease due to NASH defined as macrovesicular steatosis involving \> 5% of hepatocytes on a liver biopsy with associated lobular inflammation
* Stage 3-4 fibrosis by Ishak score on a liver biopsy
* Exclusion of other causes of liver disease including viral hepatitis and alcoholic liver disease
* Must have aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 10 x Central Laboratory Upper Limit of Normal (clULN)
* Must have serum creatinine \< 2.0 mg/dL
* A negative serum pregnancy test is required for females of childbearing potential
* All sexually active females of childbearing potential must agree to use a protocol recommended method of contraception during intercourse throughout the study and for 90 days following the last dose of study medication
* Lactating females must agree to discontinue nursing before starting study treatment
* Males, if not vasectomized, are required to use barrier contraception (condom plus spermicide) during heterosexual intercourse from the screening through the study completion and for 90 days following the last dose of study drug.

Exclusion Criteria

* Pregnant or breast feeding
* Cirrhosis of the liver
* Any history of hepatic decompensation including ascites, hepatic encephalopathy or variceal bleeding
* Weight reduction surgery in the past 5 years
* Positive for hepatitis C virus (HCV) RNA
* Positive for HBsAg
* Alcohol consumption greater than 21oz/week for males or 14oz/week for females
* Positive urine screen for amphetamines, cocaine or opiates (i.e. heroin, morphine) at screening.
* Clinically significant cardiac disease
* History of malignancy, other than non-melanomatous skin cancer, within 5 years prior to screening
* Major surgical procedure within 30 days prior to screening or the presence of an open wound
* Known hypersensitivity to the investigation product or any of its formulation excipients
* History of bleeding diathesis within 6 months of screening
* Unavailable for follow-up assessment or concern for individual's compliance with the protocol procedures;
* Participation in an investigational trial of a drug or device within 30 days prior to screening
* BMI \< 18 kg/m\^2
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Gilead Sciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gilead Study Director

Role: STUDY_DIRECTOR

Gilead Sciences

Locations

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Mayo Clinic Hospital

Phoenix, Arizona, United States

Site Status

Southern California Liver Centers

Coronado, California, United States

Site Status

University of California, San Diego (UCSD)

San Diego, California, United States

Site Status

University of California San Francisco (UCSF)

San Francisco, California, United States

Site Status

University of Colorado

Aurora, Colorado, United States

Site Status

Miami Veterans Administration Healthcare System

Miami, Florida, United States

Site Status

Tampa General Hospital

Tampa, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Indianapolis Gastroenterology Research Foundation

Indianapolis, Indiana, United States

Site Status

Iowa Digestive Disease Center

Clive, Iowa, United States

Site Status

University of Louisville

Louisville, Kentucky, United States

Site Status

Tulane University

New Orleans, Louisiana, United States

Site Status

Mercy Medical Center

Baltimore, Maryland, United States

Site Status

Walter Reed National Military Medical Center

Bethesda, Maryland, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

Lahey Clinic

Burlington, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Minnnesota Gastroenterology, PA

Saint Paul, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Saint Louis University Hospital

St Louis, Missouri, United States

Site Status

State University Of New York

Buffalo, New York, United States

Site Status

Weill Cornell Medical College

New York, New York, United States

Site Status

Mount Sinai Hospital

New York, New York, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University Gastroenterology

Providence, Rhode Island, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Texas Clinical Research Institute, LLC

Arlington, Texas, United States

Site Status

Brooke Army Medical Center

Fort Sam Houston, Texas, United States

Site Status

St. Luke's Episcopal Hospital

Houston, Texas, United States

Site Status

Alamo Clinical Research Associates

San Antonio, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Liver Institute of Virginia

Newport News, Virginia, United States

Site Status

Digestive and Liver Disease Specialists

Norfolk, Virginia, United States

Site Status

Bucheon St. Marys Hospital

Richmond, Virginia, United States

Site Status

Virginia Commonwealth University Health System

Richmond, Virginia, United States

Site Status

Virginia Mason Medical Center

Seattle, Washington, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Hôpital Erasme

Brussels, , Belgium

Site Status

Université Catholique de Louvain

Brussels, , Belgium

Site Status

UZ Ghent

Ghent, , Belgium

Site Status

University of Calgary

Calgary, Alberta, Canada

Site Status

University of Manitoba

Winnipeg, Manitoba, Canada

Site Status

London Health Science Center

London, Ontario, Canada

Site Status

Toronto Liver Centre

Toronto, Ontario, Canada

Site Status

Hopital Beaujon

Clichy, , France

Site Status

Hospital Saint-Antoine

Paris, , France

Site Status

Groupe Hospitalier Pitié- Salpétrière

Paris, , France

Site Status

CHU Strasbourg Hôpital Civil

Strasbourg, , France

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Gastroenterologisch-Hepatologisches Zentrum Kiel

Kiel, , Germany

Site Status

EUGASTRO GmbH

Leipzig, , Germany

Site Status

Azienda Ospedaliero-Universitaria di Modena Policlinico

Modena, , Italy

Site Status

Azienda Ospedaliera San Camillo Forlanini

Roma, , Italy

Site Status

Azienda Ospedaliera Città della Salute e della Scienza di Torino

Torino, , Italy

Site Status

Fundacion De Investigacion

San Juan, , Puerto Rico

Site Status

Hospital Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital Donostia

Donostia / San Sebastian, , Spain

Site Status

Hospital Universitario Puerta de Hierro

Majadahonda, , Spain

Site Status

John Radcliffe Hospital

Headington, , United Kingdom

Site Status

The Royal London Hospital

London, , United Kingdom

Site Status

Royal Free Hospital, Pond Street

London, , United Kingdom

Site Status

King's College Hospital NHS Foundation Trust No. 1 Account

London, , United Kingdom

Site Status

Nottingham University Hospitals Queens Medica

Nottingham, , United Kingdom

Site Status

Countries

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United States Belgium Canada France Germany Italy Puerto Rico Spain United Kingdom

References

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Sanyal A, Harrison S, Ratziu V, Abdelmalek M, Diehl A, Caldwell S, et al. Changes in fibrosis, but not the NAFLD Activity Score (NAS), are associated with disease progression in patients with nonalcoholic steatohepatitis (NASH) and advanced fibrosis. J Hepatol 2017; 66 (1): S2.

Reference Type RESULT

Sanyal A, Abdelmalek M, Diehl A, Caldwell S, Shiffman M, Ghalib R, et al. Efficacy and safety of simtuzumab for the treatment of nonalcoholic steatohepatitis with bridging fibrosis or cirrhosis: results of two phase 2b, dose-ranging, randomized, placebo-controlled trials. J Hepatol 2017; 66 (1): S54.

Reference Type RESULT

Ratziu V, Sanyal A, Torres D, Hinrichsen H, Serfaty L, Bambha K, et al. Impact of weight reduction on serum markers and liver histology including progression to cirrhosis in patients with nonalcoholic steatohepatitis (NASH) and bridging fibrosis. J Hepatol 2017; 66 (1): S594.

Reference Type RESULT

Shea PR, Sanyal A, Harrison S, Ratziu V, Loomba R, Caldwell S, et al. PNPLA3 variants confer an increased risk of advanced fibrosis due to non-alcoholic steatohepatitis. J Hepatol 2016; 64 (2): S493.

Reference Type RESULT

Shea PR, Sanyal A, Rockey DC, Loomba R, Diehl AM, Kleinstein SE, et al. Genome-wide association study of clinically significant portal hypertension in patients with nonalcoholic steatohepatitis and advanced fibrosis. J Hepatol 2016; 64 (2): S280.

Reference Type RESULT

Goodman ZD, Alaparthi L, Monge F, Patel K, Loomba R, Caldwell SH, et al. Correlations between hepatic morphometric collagen content, histologic fibrosis staging, and serum markers in patients with advanced fibrosis due to nonalcoholic steatohepatitis (NASH). Hepatol 2015; 62 (1): 906A.

Reference Type RESULT

Harrison SA, Goodman ZD, Ratziu V, Loomba R, Diehl AM, Lawitz E, et al. Serum lysyl oxidase-like-2 (sLOXL2) levels correlate with fibrosis stage in patients with nonalcoholic steatohepatitis (NASH). Hepatol 2015; 62 (1): 910A.

Reference Type RESULT

Ratziu V, Sanyal AJ, Loomba R, Caldwell SH, Ghalib RH, Torres DM, et al. Characterization of insulin resistance in patients with advanced fibrosis due to nonalcoholic steatohepatitis (NASH). Hepatol 2015; 62 (1): 1298A-1299A.

Reference Type RESULT

Loomba R, Huang DQ, Sanyal AJ, Anstee QM, Trauner M, Lawitz EJ, Ding D, Ma L, Jia C, Billin A, Huss RS, Chung C, Goodman Z, Wong VW, Okanoue T, Romero-Gomez M, Abdelmalek MF, Muir A, Afdhal N, Bosch J, Harrison S, Younossi ZM, Myers RP. Liver stiffness thresholds to predict disease progression and clinical outcomes in bridging fibrosis and cirrhosis. Gut. 2023 Mar;72(3):581-589. doi: 10.1136/gutjnl-2022-327777. Epub 2022 Sep 9.

Reference Type DERIVED
PMID: 36750244 (View on PubMed)

Younossi ZM, Anstee QM, Wai-Sun Wong V, Trauner M, Lawitz EJ, Harrison SA, Camargo M, Kersey K, Subramanian GM, Myers RP, Stepanova M. The Association of Histologic and Noninvasive Tests With Adverse Clinical and Patient-Reported Outcomes in Patients With Advanced Fibrosis Due to Nonalcoholic Steatohepatitis. Gastroenterology. 2021 Apr;160(5):1608-1619.e13. doi: 10.1053/j.gastro.2020.12.003. Epub 2020 Dec 8.

Reference Type DERIVED
PMID: 33307033 (View on PubMed)

Harrison SA, Abdelmalek MF, Caldwell S, Shiffman ML, Diehl AM, Ghalib R, Lawitz EJ, Rockey DC, Schall RA, Jia C, McColgan BJ, McHutchison JG, Subramanian GM, Myers RP, Younossi Z, Ratziu V, Muir AJ, Afdhal NH, Goodman Z, Bosch J, Sanyal AJ; GS-US-321-0105 and GS-US-321-0106 Investigators. Simtuzumab Is Ineffective for Patients With Bridging Fibrosis or Compensated Cirrhosis Caused by Nonalcoholic Steatohepatitis. Gastroenterology. 2018 Oct;155(4):1140-1153. doi: 10.1053/j.gastro.2018.07.006. Epub 2018 Jul 7.

Reference Type DERIVED
PMID: 29990488 (View on PubMed)

Other Identifiers

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2012-002488-88

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS-US-321-0105

Identifier Type: -

Identifier Source: org_study_id

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