Safety and Tolerability of Cilofexor in Participants With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis

NCT ID: NCT04060147

Last Updated: 2023-07-27

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

11 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-17

Study Completion Date

2021-09-02

Brief Summary

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The primary objective of this study is to assess the safety and tolerability of escalating doses of cilofexor (CILO) in participants with primary sclerosing cholangitis (PSC) and compensated cirrhosis.

Detailed Description

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Conditions

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Primary Sclerosing Cholangitis Compensated Cirrhosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cilofexor

Participants will receive escalating doses of cilofexor 30 mg, 60 mg, and 100 mg.

Group Type EXPERIMENTAL

Cilofexor

Intervention Type DRUG

Tablets administered orally once daily

Interventions

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Cilofexor

Tablets administered orally once daily

Intervention Type DRUG

Other Intervention Names

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CILO GS-9674

Eligibility Criteria

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Inclusion Criteria

* Diagnosis PSC based on cholangiogram (magnetic resonance cholangiopancreatography \[MRCP\], endoscopic retrograde cholangiopancreatography \[ERCP\], or percutaneous transhepatic cholangiogram \[PTC\]) or liver biopsy
* Individuals have evidence of cirrhosis based on historical liver biopsy, abdominal imaging \[magnetic resonance imaging (MRI), computed tomography (CT), or Ultrasound\], or a screening FibroScan®, enhanced liver fibrosis (ELF)™, or FibroTest®.
* Individual has the following laboratory parameters at the Screening visit, as determined by the central laboratory:

* Estimated glomerular filtration rate (eGFR) \> 60 milliliter/minute (mL/min), as calculated by the Cockcroft-Gault equation
* Alanine aminotransferase (ALT) ≤ 5 x upper limit of the normal (ULN)
* Total 2 milligram/deciliter (mg/dL), unless the individual is known to have Gilbert's syndrome or hemolytic anemia
* International normalized ratio (INR) ≤ 1.4, unless due to therapeutic anticoagulation
* Platelet count ≥ 75,000/microliter (μL). Individuals with evidence of high-risk esophageal or gastric varices in the opinion of the investigator are excluded
* Negative anti-mitochondrial antibody

Exclusion Criteria

* Current or prior history of any of the following

* Decompensated liver disease, including ascites, hepatic encephalopathy (HE), or variceal hemorrhage
* Liver transplantation
* Cholangiocarcinoma or hepatocellular carcinoma (HCC).
* Model for End-stage Liver Disease (MELD) score \> 12 at Screening, unless due to an alternate etiology such as therapeutic anticoagulation
* Child-Pugh (CP) score \> 6 at Screening, unless due to an alternative etiology such as Gilbert's syndrome or therapeutic anticoagulation
* Current moderate to severely active inflammatory bowel disease (IBD) (including ulcerative colitis, Crohn's disease, and indeterminate colitis).

* Note: Individuals with IBD who currently have an external ostomy bag and/or proctocolectomy are not subject to this exclusion criterion and need not undergo IBD Symptom Severity Assessment.
Minimum Eligible Age

18 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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Arizona Liver Health

Chandler, Arizona, United States

Site Status

California Liver Research Institute

Pasadena, California, United States

Site Status

University of California San Francisco, Liver Clinic

San Francisco, California, United States

Site Status

Schiff Center for Liver Diseases/University of Miami

Miami, Florida, United States

Site Status

Piedmont Atlanta Hospital

Atlanta, Georgia, United States

Site Status

Indiana University Health University Hospital

Indianapolis, Indiana, United States

Site Status

Louisiana Research Center, LLC

Shreveport, Louisiana, United States

Site Status

Minnesota Gastroenterology, PA

Maplewood, Minnesota, United States

Site Status

Northwell Health Center for Liver Diseases and Transplantation

Manhasset, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

The Liver Institute at Methodist Dallas Medical Center

Dallas, Texas, United States

Site Status

University of Virginia Medical Center

Charlottesville, Virginia, United States

Site Status

Bon Secours Richmond Community Hospital, Inc. d/b/a Bon Secours Liver Institute of Richmond

Richmond, Virginia, United States

Site Status

VCU Clinical Research Services Unit (CRSU) [Patient Site Address]

Richmond, Virginia, United States

Site Status

University of Washington at Harborview Medical Center

Seattle, Washington, United States

Site Status

Liver Institute Northwest

Seattle, Washington, United States

Site Status

Countries

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United States

References

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Levy C, Caldwell S, Mantry P, Luketic V, Landis CS, Huang J, Mena E, Maheshwari R, Rank K, Xu J, Malkov VA, Billin AN, Liu X, Lu X, Barchuk WT, Watkins TR, Chung C, Myers RP, Kowdley KV. Cilofexor in Patients With Compensated Cirrhosis Due to Primary Sclerosing Cholangitis: An Open-Label Phase 1B Study. Clin Transl Gastroenterol. 2024 Aug 1;15(8):e00744. doi: 10.14309/ctg.0000000000000744.

Reference Type DERIVED
PMID: 38976363 (View on PubMed)

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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GS-US-428-5443

Identifier Type: -

Identifier Source: org_study_id

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