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A Study of Baricitinib (LY3009104) in Participants With Primary Biliary Cholangitis Who do Not Respond or Cannot Take UDCA

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03742973
Recruitment Status : Completed
First Posted : November 15, 2018
Last Update Posted : October 22, 2019
Information provided by (Responsible Party):
Eli Lilly and Company

Brief Summary:
This study evaluates the safety and efficacy of baricitinib in participants with primary biliary cholangitis (PBC) who do not respond or are unable to take ursodeoxycholic acid (UDCA).

Condition or disease Intervention/treatment Phase
Primary Biliary Cholangitis Drug: Baricitinib Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Proof-of-Concept Study Evaluating the Efficacy and Safety of Baricitinib (LY3009104) in Patients With Primary Biliary Cholangitis Who Have an Inadequate Response or Are Intolerant to UDCA
Actual Study Start Date : March 28, 2019
Actual Primary Completion Date : September 26, 2019
Actual Study Completion Date : September 26, 2019

Arm Intervention/treatment
Experimental: Baricitinib Low Dose Cohort A
Baricitinib administered orally.
Drug: Baricitinib
Administered orally.
Other Name: LY3009104

Placebo Comparator: Placebo Cohort A
Placebo administered orally.
Drug: Placebo
Administered orally.

Experimental: Baricitinib High Dose Cohort B
Baricitinib administered orally.
Drug: Baricitinib
Administered orally.
Other Name: LY3009104

Placebo Comparator: Placebo Cohort B
Placebo administered orally.
Drug: Placebo
Administered orally.

Primary Outcome Measures :
  1. Change from Baseline in Alkaline Phosphatase (ALP) [ Time Frame: Baseline, Week 12 ]
    Change from baseline in ALP

Secondary Outcome Measures :
  1. Proportion of Participants with ALP<1.67 x Upper Limit of Normal (ULN) (and at Least 15% Decrease from Baseline) and Total Bilirubin < ULN [ Time Frame: Week 12 ]
    Proportion of participants with ALP<1.67 x ULN (and at least 15% decrease from baseline) and total bilirubin < ULN

  2. Change from Baseline in Itch Numeric Rating Scale (NRS) [ Time Frame: Baseline, Week 12 ]
    Change from baseline in itch NRS

  3. Change from Baseline in Fatigue NRS [ Time Frame: Baseline, Week 12 ]
    Change from baseline in fatigue NRS

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Have a diagnosis of PBC (consistent with American Association for the Study of Liver Disease [AASLD] and European Association for Study of the Liver [EASL] Practice Guidelines; as demonstrated by the presence of at least 2 of the following 3 diagnostic factors:

    • History of elevated ALP levels for at least 6 months
    • Positive antimitochondrial antibodies titer
    • Liver biopsy consistent with PBC
  • Have ALP ≥1.67 x ULN but <6 x ULN.
  • Taking UDCA for at least 52 weeks (stable dose for at least 12 weeks) prior to Week 0, or have previously taken, but are intolerant (in the opinion of the investigator) to UDCA and have not received UDCA for at least 12 weeks prior to Week 0.
  • Nonpregnant, nonbreastfeeding female participants of childbearing potential.

Exclusion Criteria:

  • History or presence of other concomitant liver diseases including:

    • Hepatitis C virus (HCV) infection
    • Hepatitis B virus (HBV) infection
    • Primary sclerosing cholangitis
    • Alcoholic liver disease
    • Autoimmune liver disease other than PBC, such as overlap hepatitis
    • Nonalcoholic steatohepatitis
    • Gilbert's syndrome
  • Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:

    • Liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥15
    • Portal hypertension with complications, including known gastric or esophageal varices, ascites, history of variceal bleeds or related therapeutic or prophylactic interventions (e.g., beta blockers, insertion of variceal bands or transjugular intrahepatic portosystemic shunt), or hepatic encephalopathy
    • Cirrhosis, including history or presence of one or more of the following:

      • spontaneous bacterial peritonitis
      • hepatocellular carcinoma
    • Hepatorenal syndrome (type I or II)
  • Have an estimated glomerular filtration rate (eGFR) based on the most recent available serum creatinine of <90 milliliters/minute/1.73 m2.
  • Have screening electrocardiogram (ECG) abnormalities that in the opinion of the investigator or the sponsor are clinically significant and indicate an unacceptable risk for the participant's participation in the study.
  • Have experienced any of the following within 12 weeks of screening: myocardial infarction, unstable ischemic heart disease, stroke, or New York Heart Association Stage III/IV heart failure.
  • Have a history of venous thromboembolism (VTE) (deep vein thrombosis/pulmonary embolism [DVT/PE]).
  • Have a history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, hematological, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data.
  • Have a current or recent (<4 weeks prior to randomization) clinically serious infection or any other active or recent infection that, in the opinion of the investigator, would pose an unacceptable risk to the participant if participating in the study.
  • Have had symptomatic herpes zoster infection within 12 weeks prior to randomization.
  • Have active tuberculosis (TB) disease determined on the basis of a positive medical history, physical examination, or chest radiography (per local standard of care) or latent TB infection (LTBI).
  • Have any of the following specific abnormalities based on screening central lab test results:

    • Hemoglobin <10 grams per deciliter (100.0 grams per liter)
    • Alanine aminotransferase (ALT) >3 x ULN
    • aspartate aminotransferase (AST) >3 x ULN
    • alkaline phosphatase (ALP) >6 x ULN
    • Total bilirubin level (TBL) >ULN
    • Creatine phosphokinase (CPK) > ULN
    • Serum albumin < lower limit of normal (LLN)
    • International Normalized Ratio of Prothrombin Time (INR) > ULN
    • Total white blood cell (WBC) count <LLN
    • Absolute neutrophil count [ANC] <LLN
    • Lymphocyte count <LLN
    • Platelet (thrombocyte) count <LLN
  • Are receiving unstable treatment for pruritus within 6 weeks prior to Week 0.
  • Have been treated with systemic (oral or parenteral) corticosteroids within 6 weeks prior to Week 0.
  • Have received biologic treatments for an immunologic disease within 4 weeks of screening.
  • Have received a Janus kinase (JAK) inhibitor.
  • Have received obeticholic acid.
  • Have received fenofibrate or other fibrates for the treatment of PBC.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03742973

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United States, California
Southern California GI and Liver Centers (SCLC)
Coronado, California, United States, 92118
University of California, Davis - Health Systems
Sacramento, California, United States, 95817
United States, Colorado
University of Colorado School of Medicine
Aurora, Colorado, United States, 80045
United States, Florida
Schiff Center for Liver Diseases/University of Miami
Miami, Florida, United States, 33136
United States, Maryland
The Institute for Digestive Health and Liver Disease at Mercy
Baltimore, Maryland, United States, 21202
United States, Michigan
Henry Ford Hospital
Detroit, Michigan, United States, 48202
United States, New York
NYU Langone
New York, New York, United States, 10016
United States, Ohio
UH Cleveland Medical Center
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Puerto Rico
Klinical Investigations Group, LLC
San Juan, Puerto Rico, 00909
University of Puerto Rico, Medical Sciences Campus
San Juan, Puerto Rico, 00936
Sponsors and Collaborators
Eli Lilly and Company
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Study Director: Call 1-877-CTILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) Eli Lilly and Company
Additional Information:
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Responsible Party: Eli Lilly and Company Identifier: NCT03742973    
Other Study ID Numbers: 17039
I4V-MC-JAIV ( Other Identifier: Eli Lilly and Company )
First Posted: November 15, 2018    Key Record Dates
Last Update Posted: October 22, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and EU, whichever is later. Data will be indefinitely available for requesting.
Access Criteria: A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Eli Lilly and Company:
Janus kinase inhibitor
Additional relevant MeSH terms:
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Liver Cirrhosis, Biliary
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Cholestasis, Intrahepatic
Liver Diseases
Liver Cirrhosis