Simtuzumab (GS-6624) in the Prevention of Progression of Liver Fibrosis in Adults With Primary Sclerosing Cholangitis (PSC)

NCT ID: NCT01672853

Last Updated: 2019-10-22

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

235 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-03-04

Study Completion Date

2016-08-24

Brief Summary

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The purpose of this study is to evaluate whether simtuzumab (GS-6624) is effective at preventing the progression of liver fibrosis in adults with primary sclerosing cholangitis (PSC).

Detailed Description

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Conditions

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Primary Sclerosing Cholangitis (PSC)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Treatment Arm A

Simtuzumab 75 mg for 96 weeks

Group Type EXPERIMENTAL

Simtuzumab

Intervention Type BIOLOGICAL

Subcutaneous injections weekly for a total of 96 injections

Treatment Arm B

Simtuzumab 125 mg for 96 weeks

Group Type EXPERIMENTAL

Simtuzumab

Intervention Type BIOLOGICAL

Subcutaneous injections weekly for a total of 96 injections

Treatment Arm C

Placebo for 96 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type BIOLOGICAL

Subcutaneous injections weekly for a total of 96 injections

Interventions

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Simtuzumab

Subcutaneous injections weekly for a total of 96 injections

Intervention Type BIOLOGICAL

Placebo

Subcutaneous injections weekly for a total of 96 injections

Intervention Type BIOLOGICAL

Other Intervention Names

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GS-6624

Eligibility Criteria

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Inclusion Criteria

* Adult Individuals (aged 18-70) with chronic cholestatic liver disease of at least 6 months.
* Liver biopsy consistent with PSC: If a liver biopsy has been performed within 3 months of the screening visit, tissue from that biopsy may be used as the screening biopsy. Slides would be re-cut from the existing tissue block and submitted for central reader assessment. Some individuals with PSC may have a normal liver biopsy, in the event of a normal liver biopsy, the individual must have an abnormal magnetic resonance cholangiopancreatography (MRCP).
* MRCP consistent with PSC: Some individuals with PSC may have a normal MRCP; in the event of a normal MRCP, the individual must have an abnormal liver biopsy.
* Exclusion of other causes of liver disease including viral hepatitis ,alcoholic liver disease,primary biliary cirrhosis and secondary sclerosing cholangitis
* Must have aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 10 x the Central Laboratory Upper Limit of Normal (clULN)
* Must have serum creatinine \< 2.0 mg/dL
* A negative serum pregnancy test is required for female individuals of childbearing potential
* All sexually active female individuals of childbearing potential must agree to use a protocol recommended method of contraception during heterosexual 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 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
* Evidence of hepatic decompensation present, including ascites, episodes of hepatic encephalopathy, variceal bleeding or a prolonged prothrombin time/international normalized ratio (PT/INR)
* Positive for hepatitis C virus (HCV) RNA
* Positive for HBsAg
* Positive for anti-mitochondrial antibody
* Alcohol consumption greater than 21oz/week for males or 14oz/week for females
* Moderately active ulcerative colitis (UC) defined as either a partial Mayo score of \> 4, bleeding score of \>1, or current use of oral corticosteroid therapy and/or any inhibitor of Tumor necrosis factor-α (TNF-α) or α4β7 integrin antagonist
* Positive urine screen for amphetamines, cocaine or opiates (i.e. heroin, morphine) at screening. Individuals on stable methadone or buprenorphine maintenance treatment for at least 6 months prior to screening may be included in the study. Individuals with a positive urine drug screen due to prescription opioid-based medication are eligible if the prescription and diagnosis are reviewed and approved by the investigator
* Clinically significant cardiac disease
* History of cholangiocarcinoma
* History of other cancers,other than non-melanomatous skin cancer, within 5 years prior to screening
* Ascending cholangitis within 60 days of screening
* Presence of a percutaneous drain or bile duct stent
* 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
* Major surgical procedure within 30 days prior to screening or the presence of an open wound
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 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

University of Arizona Health Sciences Center

Tucson, Arizona, United States

Site Status

Southern California Liver Center

Chula Vista, California, United States

Site Status

Southern California Liver Centers

Coronado, California, United States

Site Status

Scripps Clinic

La Jolla, California, United States

Site Status

Verterans Adminstration Hospital

Palo Alto, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

University of San Diego Medical Center

San Diego, California, United States

Site Status

University of California San Francisco

San Francisco, California, United States

Site Status

University of Colorado Denver

Aurora, Colorado, United States

Site Status

University of Miami Center for Liver Diseases

Miami, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, 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 Health Sciences Center

New Orleans, Louisiana, 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

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Minnesota Gastroenterology, PA

Saint Paul, Minnesota, United States

Site Status

St. Louis University

St Louis, Missouri, 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 Clinical Research Institute

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

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Brooke Army Medical Center

Fort Sam Houston, Texas, United States

Site Status

St. Luke Episcopal Hospital

Houston, Texas, United States

Site Status

Alamo Medical Research

San Antonio, Texas, United States

Site Status

Intermountain Medical Center

Murray, Utah, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia Health Center

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

Bon Secours Richmond Health System

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 Antwerpen

Edegem, , Belgium

Site Status

UZ Gent

Ghent, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

University of Calgary

Calgary, Alberta, Canada

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

University of Manitoba

Winnepeg, Manitoba, Canada

Site Status

Dalhousie University

Halifax, Nova Scotia, Canada

Site Status

London Health Science Center

London, Ontario, Canada

Site Status

Toronto Liver Centre

Toronto, Ontario, Canada

Site Status

Århus Universitetshospital, Århus Sygehus

Århus C, , Denmark

Site Status

Hvidovre Hospital

Hvidovre, , Denmark

Site Status

Rigshospitalet

København Ø, , Denmark

Site Status

Klinikum der Johann Wolfgang Goethe Universitat

Frankfurt, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Gastroenterologisch Hepatologisches Zentrum Kiel

Kiel, , Germany

Site Status

EUGASTRO GmbH

Leipzig, , Germany

Site Status

Istituto Clinico Humanitas

Rozzano, Milano, 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

Eramus MC

Rotterdam, , Netherlands

Site Status

Hospital Universitario Vall d'Hebron

Barcelona, Catalonia, Spain

Site Status

Hospital Donostia

Donostia / San Sebastian, , Spain

Site Status

Hospital Universitario Puerta de Hierro

Majadahonda, , Spain

Site Status

Avd för invärtesmedicin och klinisk nutrition

Gothenburg, , Sweden

Site Status

New Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

John Radcliffe Hospital

Headington, , United Kingdom

Site Status

Imperial College Healthcare NHS Trust- St. Mary's Hospital

London, , United Kingdom

Site Status

University College London

London, , United Kingdom

Site Status

Norfolk and Norwich University Hospital

Norwich, , United Kingdom

Site Status

University of Nottingham

Nottingham, , United Kingdom

Site Status

Countries

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United States Belgium Canada Denmark Germany Italy Netherlands Spain Sweden United Kingdom

References

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Shea P, Hirschfield G, Shiffman M, McColgan B, Goodman Z, Myers, R, et al. Common variation near glial-derived neurotrophic factor is associated with progression of hepatic collagen content in a genome-wide association study of liver fibrosis phenotypes in patients with primary sclerosing cholangitis. J Hepatol 2017; 66 (1): S4-S5.

Reference Type RESULT

Muir A, Goodman Z, Levy C, Janssen H, Montano-Loza A, Bowlus C, et al. Efficacy and safety of simtuzumab for the treatment of primary sclerosing cholangitis: results of a phase 2b, dose-ranging, randomized, placebo-controlled trial. J Hepatol 2017; 66 (1): S73.

Reference Type RESULT

Bowlus CL, Patel K, Hirschfield G, Guha I, Chapman R, Chazouilleres O, et al. Prospective validation of the Enhanced Liver Fibrosis test for the prediction of disease progression in a randomized trial of patients with primary sclerosing cholangitis. J Hepatol 2017; 66 (1): S359.

Reference Type RESULT

Levy C, Eksteen B, Shiffman M, Janssen H, Montano-Loza A, Ding D, et al. Prospective validation of serum alkaline phosphatase for the prediction of disease progression in a randomized trial of patients with primary sclerosing cholangitis. J Hepatol 2017; 66 (1): S360-S361.

Reference Type RESULT

Levy C, Shiffman M, Caldwell S, Luketic V, Invernizzi P, Minuk G, et al. Serum fibroblast growth factor 19, 7α-Hydroxy-4-Cholesten-3-one, and bile acids and their associations with clinical characteristics in primary sclerosing cholangitis. J Hepatol 2017; 66 (1): S361.

Reference Type RESULT

French D, Huntzicker EG, Goodman ZD, Shea PR, Ding D, Aguilar Schall, RE, et al. Hepatic expression of lysyl oxidase-like-2 (LOXL2) in primary sclerosing cholangitis (PSC). Hepatol 2016; 64 (1): 194A.

Reference Type RESULT

Bowlus CL, Montano-Loza AJ, Invernizzi P, Chazouilleres O, Hirschfield G, Metselaar HJ, et al. Liver stiffness measurement by transient elastography for the prediction of fibrosis in patients with primary sclerosing cholangitis in a randomized trial of simtuzumab. J Hepatol 2016; 64 (2): S434.

Reference Type RESULT

Goodman Z, Patel K, Guha I, Hameed B, Kowdley K, Alaparthi L, et al. Correlations between hepatic morphometric collagen content, histologic fibrosis stage, and serum markers in patients with primary sclerosing cholangitis (PSC). J Hepatol 2016; 64 (2): S652-S653.

Reference Type RESULT

Muir AJ, Goodman Z, Bowlus CL, Caldwell S, Invernizzi P, Luketic V, et al. Serum lysyl oxidase-like-2 (SLOXL2) levels correlate with disease severity in patients with primary sclerosing cholangitis. J Hepatol 2016; 64 (2): S428.

Reference Type RESULT

Shea PR, Eksteen B, Hirschfield GM, Shiffman ML, Janssen HLA, Montano-Loza AJ, et al. Genome-wide association study (GWAS) of liver fibrosis phenotypes in patients with primary sclerosing cholangitis (PSC) reveals common genetic variation influencing serum levels of lysyl oxidase-like-2 (LOXL2). J Hepatol 2016; 64 (2): S180-S182.

Reference Type RESULT

Manns MP, Eksteen B, Shiffman ML, Levy C, Kowdley KV, Montano-Loza AJ, et al. Association between elevated serum IgG4 (sIgG4) concentrations and the phenotype of patients with primary sclerosing cholangitis (PSC). Hepatol 2015; 62 (1): 515A.

Reference Type RESULT

Bowlus CL, Patel K, Chuha IN, Chapman RW, Chazouilleres O, Chalasani NP, et al. Validation of serum fibrosis marker panels in patients with primary sclerosing cholangitis (PSC) in a randomized trial of simtuzumab. Hepatol 2015; 62 (1): 519A.

Reference Type RESULT

French D, Goodman ZD, Huntzicker EG, Newstrom D, Karnik S, Smith V, et al. Expression of matrix metalloproteinase 9 (MMP9) and the apoptosis signal-regulating kinase 1 (ASK1) pathway activation marker, phospho-P38 (p-P38), in primary sclerosing cholangitis (PSC). Hepatol 2015; 62 (1): 523A-524A.

Reference Type RESULT

Laschtowitz A, Lindberg EL, Liebhoff AM, Liebig LA, Casar C, Steinmann S, Guillot A, Xu J, Schwinge D, Trauner M, Lohse AW, Bonn S, Hubner N, Schramm C. Liver transcriptome analysis reveals PSC-attributed gene set associated with fibrosis progression. JHEP Rep. 2024 Nov 12;7(3):101267. doi: 10.1016/j.jhepr.2024.101267. eCollection 2025 Mar.

Reference Type DERIVED
PMID: 39996122 (View on PubMed)

Thorburn D, Leeming DJ, Barchuk WT, Wang Y, Lu X, Malkov VA, Ito KL, Bowlus CL, Levy C, Goodman Z, Karsdal MA, Muir AJ, Xu J. Serologic extracellular matrix remodeling markers are related to fibrosis stage and prognosis in a phase 2b trial of simtuzumab in patients with primary sclerosing cholangitis. Hepatol Commun. 2024 Jul 5;8(7):e0467. doi: 10.1097/HC9.0000000000000467. eCollection 2024 Jul 1.

Reference Type DERIVED
PMID: 38967589 (View on PubMed)

Muir AJ, Levy C, Janssen HLA, Montano-Loza AJ, Shiffman ML, Caldwell S, Luketic V, Ding D, Jia C, McColgan BJ, McHutchison JG, Mani Subramanian G, Myers RP, Manns M, Chapman R, Afdhal NH, Goodman Z, Eksteen B, Bowlus CL; GS-US-321-0102 Investigators. Simtuzumab for Primary Sclerosing Cholangitis: Phase 2 Study Results With Insights on the Natural History of the Disease. Hepatology. 2019 Feb;69(2):684-698. doi: 10.1002/hep.30237. Epub 2019 Jan 11.

Reference Type DERIVED
PMID: 30153359 (View on PubMed)

Other Identifiers

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2012-002473-61

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS-US-321-0102

Identifier Type: -

Identifier Source: org_study_id

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