Study to Evaluate the Safety, Tolerability, and Efficacy of Cilofexor in Adults With Primary Sclerosing Cholangitis Without Cirrhosis

NCT ID: NCT02943460

Last Updated: 2021-06-07

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

52 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-11-29

Study Completion Date

2020-05-18

Brief Summary

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The primary objective of this study is to evaluate the safety and tolerability of cilofexor in adults with primary sclerosing cholangitis (PSC).

Detailed Description

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Conditions

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Primary Sclerosing Cholangitis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Cilofexor 100 mg (Blinded Study Phase)

Cilofexor 100 mg + placebo to match cilofexor 30 mg for up to 12.6 weeks

Group Type EXPERIMENTAL

Cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Placebo to match cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Cilofexor 30 mg (Blinded Study Phase)

Cilofexor 30 mg + placebo to match cilofexor 100 mg for up to 12.7 weeks

Group Type EXPERIMENTAL

Cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Placebo to match cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Placebo (Blinded Study Phase)

Placebo to match cilofexor 30 mg + placebo to match cilofexor 100 mg for up to 12.3 weeks

Group Type PLACEBO_COMPARATOR

Placebo to match cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Cilofexor (Open Label Extension Phase)

Following the Blinded Study Phase, eligible participants received cilofexor for an additional up to 97.4 weeks.

Group Type EXPERIMENTAL

Cilofexor

Intervention Type DRUG

Tablet(s) administered orally once daily with food

Interventions

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Cilofexor

Tablet(s) administered orally once daily with food

Intervention Type DRUG

Placebo to match cilofexor

Tablet(s) administered orally once daily with food

Intervention Type DRUG

Other Intervention Names

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GS-9674

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of PSC based on cholangiogram (magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), or percutaneous transhepatic cholangiogram (PTC)) within the previous 12 months
* Serum alkaline phosphatase (ALP) \> 1.67 x upper limit of the normal range (ULN)
* For individuals on ursodeoxycholic acid (UDCA), the dose of UDCA must have been stable for at least 12 months prior to screening through the end of treatment. For individuals not on UDCA, no UDCA use for at least 12 months before screening through the end of treatment
* For individuals being administered biologic treatments (eg, antitumor necrosis factor (TNF) or anti-integrin monoclonal antibodies), immunosuppressants or systemic corticosteroids, the dose must have been stable at least 3 months prior to screening and anticipated to remain stable throughout the trial
* Screening FibroSURE/FibroTest® \<0.75 unless a historical liver biopsy within 12 months of screening does not reveal cirrhosis. In adults with Gilbert's syndrome or hemolysis, FibroSURE/FibroTest® will be calculated using direct bilirubin instead of total bilirubin.

Exclusion Criteria

* Alanine aminotransferase (ALT) \> 10 x ULN
* Total bilirubin \> 2 x ULN
* International normalized ratio (INR) \> 1.2 unless on anticoagulant therapy
* Small-duct PSC (histologic evidence of PSC with normal bile ducts on cholangiography)
* Other causes of liver disease including secondary sclerosing cholangitis and viral, metabolic, alcoholic, and other autoimmune conditions. Individuals with hepatic steatosis may be included if there is no evidence of nonalcoholic steatohepatitis (NASH) in the opinion of the investigator or on liver biopsy;
* Ascending cholangitis within 60 days of screening
* Presence of a percutaneous drain or bile duct stent
* Use of fibrates or obeticholic acid within 3 months prior to screening through the end of treatment
* Cirrhosis of the liver as defined by any of the following:

* Historical liver biopsy demonstrating cirrhosis (eg, Ludwig stage 4 or Ishak stage ≥ 5)
* Prior history of decompensated liver disease, including ascites, hepatic encephalopathy or variceal bleeding
* Liver stiffness \> 14.4 kilopascal (kPa) by FibroScan
* Current, active inflammatory bowel disease (IBD) defined as a partial Mayo score of \> 1 and/or a score on the Rectal Bleeding domain \> 0.
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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University of California, Davis Medical Center

Sacramento, 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

Florida Digestive Health Specialists

Lakewood Rch, Florida, United States

Site Status

Schiff Center for Liver Diseases/University of Miami

Miami, Florida, United States

Site Status

Indiana University Health University Hospital

Indianapolis, Indiana, United States

Site Status

Minnesota Gastroenterology, P.A.

Saint Paul, Minnesota, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

The Liver Institute at Methodist Dallas Medical Center

Dallas, Texas, United States

Site Status

Intermountain Medical Center - Transplant Services

Murray, Utah, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Bon Secours St. Mary's Hospital of Richmond, Inc.

Richmond, Virginia, United States

Site Status

McGuire VA Medical Center

Richmond, Virginia, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Swedish Organ Transplant and Liver Center

Seattle, Washington, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Universitätsklinik Klinik für Innere Medizin III

Vienna, , Austria

Site Status

University of Calgary Liver Unit (Heritage Medical Research Clinic)

Calgary, Alberta, Canada

Site Status

Toronto Liver Centre

Toronto, Ontario, Canada

Site Status

New Queen Elizabeth Hospital NHS Foundation Trust

Birmingham, , United Kingdom

Site Status

Royal Free Hospital

London, , United Kingdom

Site Status

King's College Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

Norfolk and Norwich University Hospital

Norwich, , United Kingdom

Site Status

Countries

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United States Austria Canada United Kingdom

References

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Trauner M, Gulamhusein A, Hameed B, Caldwell S, Shiffman ML, Landis C, Eksteen B, Agarwal K, Muir A, Rushbrook S, Lu X, Xu J, Chuang JC, Billin AN, Li G, Chung C, Subramanian GM, Myers RP, Bowlus CL, Kowdley KV. The Nonsteroidal Farnesoid X Receptor Agonist Cilofexor (GS-9674) Improves Markers of Cholestasis and Liver Injury in Patients With Primary Sclerosing Cholangitis. Hepatology. 2019 Sep;70(3):788-801. doi: 10.1002/hep.30509. Epub 2019 Mar 10.

Reference Type RESULT
PMID: 30661255 (View on PubMed)

Provided Documents

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Document Type: Study Protocol: Original

View Document

Document Type: Study Protocol: Amendment 1

View Document

Document Type: Study Protocol: Amendment 2

View Document

Document Type: Study Protocol: Amendment 3

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-002442-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS-US-428-4025

Identifier Type: -

Identifier Source: org_study_id

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