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

NCT ID: NCT02943447

Last Updated: 2020-09-22

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

71 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-12-01

Study Completion Date

2019-09-04

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 biliary cholangitis (PBC).

Detailed Description

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Conditions

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Primary Biliary 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 30 mg (Blinded Study Phase)

Cilofexor 30 mg + placebo to match cilofexor 100 mg for 12 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 100 mg (Blinded Study Phase)

Cilofexor 100 mg + placebo to match cilofexor 30 mg for 12 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 12 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 may have the option to receive open-label cilofexor 100 mg for an additional 96 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

* Meets all of the following conditions

* Definite or probable PBC as defined by at least 2 of the 3 following criteria:

* Serum alkaline phosphatase (ALP) \> the upper limit of normal (ULN)
* Presence of anti-mitochondrial antibodies (AMA) in serum (≥ 1:40 on immunofluorescence)
* Liver histological findings consistent with PBC including nonsuppurative, destructive cholangitis affecting mainly the interlobular bile and septal bile ducts
* Serum ALP \> 1.67 x ULN and/or total bilirubin \>ULN but ≤ 2 x ULN
* Ursodeoxycholic acid (UDCA) use at a stable dose for at least 12 months or intolerant of UDCA with no UDCA use for at least 12 months before screening
* 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) \> 5 x ULN
* Total bilirubin \> 2 x ULN
* International normalized ratio (INR) \> 1.2 unless on anticoagulant therapy
* Other causes of liver disease including viral, metabolic, alcoholic, and other autoimmune conditions. Participants 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.
* 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)
* History of decompensated liver disease, including ascites, hepatic encephalopathy or variceal bleeding
* Liver stiffness \> 16.9 kPa by FibroScan®
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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Sacramento, California, United States

Site Status

Aurora, Colorado, United States

Site Status

Lakewood Rch, Florida, United States

Site Status

Miami, Florida, United States

Site Status

Marietta, Georgia, United States

Site Status

Saint Paul, Minnesota, United States

Site Status

Arlington, Texas, United States

Site Status

Dallas, Texas, United States

Site Status

Dallas, Texas, United States

Site Status

Houston, Texas, United States

Site Status

Charlottesville, Virginia, United States

Site Status

Newport News, Virginia, United States

Site Status

Seattle, Washington, United States

Site Status

Graz, Styria, Austria

Site Status

Vienna, Vienna, Austria

Site Status

Calgary, Alberta, Canada

Site Status

Vancouver, British Columbia, Canada

Site Status

Vancouver, British Columbia, Canada

Site Status

Winnipeg, Manitoba, Canada

Site Status

Toronto, Ontario, Canada

Site Status

Vaughan, Ontario, Canada

Site Status

Birmingham, England, United Kingdom

Site Status

London, England, United Kingdom

Site Status

London, England, United Kingdom

Site Status

Norwich, England, United Kingdom

Site Status

Countries

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United States Austria Canada United Kingdom

References

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Kowdley KV, Minuk GY, Pagadala MR, Gulamhusein A, Swain MG, Neff GW, et al. The Nonsteroidal Farnesoid X Receptor (FXR) Agonist Cilofexor Improves Liver Biochemistry in Patients with Primary Biliary Cholangitis (PBC): A Phase 2, Randomized, Placebo-Controlled Trial [Abstract 45]. Hepatology AASLD Abstracts 2019;70 (Suppl 1):31A-2A.

Reference Type RESULT

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-002443-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GS-US-427-4024

Identifier Type: -

Identifier Source: org_study_id

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