Primary Biliary CholangitisPhase 2NCT03742973
A Study of Baricitinib (LY3009104) in Participants With Primary Biliary Cholangitis Who do Not Respond or Cannot Take UDCA
A Phase 2 study testing Baricitinib in people with Primary Biliary Cholangitis, run by Eli Lilly and Company at 12 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- Baricitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 28 Mar 2019to 26 Sep 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 Alkaline Phosphatase (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 Upper Limit Normal (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.
Who cannot join
- 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
Show all 42Show fewer
- 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.
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
United States10 sites
University of Colorado School of Medicine
Aurora, Colorado, 80045
The Institute for Digestive Health and Liver Disease at Mercy
Baltimore, Maryland, 21202
UH Cleveland Medical Center
Cleveland, Ohio, 44106
Southern California GI and Liver Centers (SCLC)
Coronado, California, 92118
Henry Ford Hospital
Detroit, Michigan, 48202
Baylor College of Medicine
Houston, Texas, 77030
Schiff Center for Liver Diseases/University of Miami
Miami, Florida, 33136
NYU Langone
New York, New York, 10016
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
University of California, Davis - Health Systems
Sacramento, California, 95817
Puerto Rico2 sites
Klinical Investigations Group, LLC
San Juan, PR, 00909
University of Puerto Rico, Medical Sciences Campus
San Juan, PR, 00936
About this study
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
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).
- Conditions
- Primary Biliary Cholangitis
- Being tested
- Baricitinib, Placebo
- Keywords
- Janus kinase inhibitor, JAK
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaricitinibDrug
Also called LY3009104
Administered orally.
PlaceboDrug
Administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Baricitinib Cohort A | Experimental | Participants received 2 milligram (mg) of Baricitinib tablet orally once a day for 12 weeks. Cohort A is not reported due to protection of personal identifiable information based on enrollment futility. Receives: Drug: Baricitinib |
| Placebo Cohort A | Placebo comparator | Participants received placebo orally once a day for 12 weeks. Cohort A is not reported due to protection of personal identifiable information based on enrollment futility. Receives: Drug: Placebo |
| Baricitinib Cohort B | Experimental | Participants received 4 mg of Baricitinib orally once a day for 12 weeks. Cohort B was planned, but due to enrollment futility, the strategic decision was made to terminate the study. Receives: Drug: Baricitinib |
| Placebo Cohort B | Placebo comparator | Placebo administered orally. Cohort B was planned, but due enrollment futility, the strategic decision was made to terminate the study. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Alkaline Phosphatase (ALP)
Change from baseline in Alkaline Phosphatase (ALP)
Measured: Baseline, Week 12
Secondary outcomes (3)
Percentage of Participants With Alkaline Phosphatase (ALP) <1.67 x Upper Limit of Normal (ULN) (and at Least 15% Decrease From Baseline) and Total Bilirubin Level Less Than ULN
Percentage of participants with alkaline phosphatase (ALP) <1.67 x Upper Limit of Normal (ULN) (and at least 15% decrease from baseline) and total bilirubin level less than ULN.
Measured: Week 12
Change From Baseline in Itch Numeric Rating Scale (NRS)
Change from baseline in itch NRS. The Itch NRS is a participant-administered, 11-point horizontal scale anchored at 0 and 10, with 0 representing "no itch" and 10 representing "worst itch imaginable." Overall severity of a participant's itching is indicated by circling the number that best describes the worst level of itching in the past 7 days.
Measured: Baseline, Week 12
Change From Baseline in Fatigue NRS
Change from baseline in fatigue NRS. The Fatigue NRS is a single-item, patient-administered, 11-point horizontal scale anchored at 0 and 10, with 0 representing "no fatigue" and 10 representing "as bad as you can imagine." Overall severity of a participant's fatigue is indicated by selecting the number that describes the worst level of fatigue during the past 7 days.
Measured: Baseline, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2018
- Study start28 Mar 2019
- Primary completion26 Sep 2019
- Study completion26 Sep 2019
Why it stopped: Study was terminated due to enrollment futility.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- 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), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03742973
- Sponsor's protocol ID
- 17039
- Other IDs
- I4V-MC-JAIV
- Status last verified
- Oct 2019
- First submitted
- 14 Nov 2018
- Last updated
- 14 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03742973
- Acronym / sponsor ID
- 17039
- Conditions
- Primary Biliary Cholangitis
- Keywords
- Janus kinase inhibitor, JAK
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated due to enrollment futility.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Study start
- 28 Mar 2019 (actual)
- Primary completion
- 26 Sep 2019 (actual)
- Study completion
- 26 Sep 2019 (actual)
- First posted
- 15 Nov 2018 (actual)
- Results
- Posted 14 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Baricitinib Cohort A | Experimental | Drug: BaricitinibParticipants received 2 milligram (mg) of Baricitinib tablet orally once a day for 12 weeks. Cohort A is not reported due to protection of personal identifiable information based on enrollment futility. |
| Placebo Cohort A | Placebo comparator | Drug: PlaceboParticipants received placebo orally once a day for 12 weeks. Cohort A is not reported due to protection of personal identifiable information based on enrollment futility. |
| Baricitinib Cohort B | Experimental | Drug: BaricitinibParticipants received 4 mg of Baricitinib orally once a day for 12 weeks. Cohort B was planned, but due to enrollment futility, the strategic decision was made to terminate the study. |
| Placebo Cohort B | Placebo comparator | Drug: PlaceboPlacebo administered orally. Cohort B was planned, but due enrollment futility, the strategic decision was made to terminate the study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- Have ALP ≥1.67 x ULN but ≤6 x Upper Limit Normal (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.
Key exclusion criteria (42 listed)
- History or presence of other concomitant liver diseases including:
- Presence of clinical complications of PBC or clinically significant hepatic decompensation, including:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BaricitinibAlso: LY3009104Administered orally. | Drug | Baricitinib Cohort A, Baricitinib Cohort B |
| PlaceboAdministered orally. | Drug | Placebo Cohort A, Placebo Cohort B |
Primary outcome
Change From Baseline in Alkaline Phosphatase (ALP)
Time frame: Baseline, Week 12
Change from baseline in Alkaline Phosphatase (ALP)
3 secondary outcomes
Percentage of Participants With Alkaline Phosphatase (ALP) <1.67 x Upper Limit of Normal (ULN) (and at Least 15% Decrease From Baseline) and Total Bilirubin Level Less Than ULN
Time frame: Week 12
Change From Baseline in Itch Numeric Rating Scale (NRS)
Time frame: Baseline, Week 12
Change From Baseline in Fatigue NRS
Time frame: Baseline, Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Click here for more information about this study: A Study of Baricitinib (LY3009104) in Participants With Primary Biliary Cholangitis Who do Not Respond or Cannot Take UDCALink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Primary Biliary Cholangitis
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FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy
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Fenofibrate for Patients With Primary Biliary Cirrhosis Who Had An Inadequate Response to Ursodeoxycholic Acid
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
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) study director
Eli Lilly and Company
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Baricitinib in people with Primary Biliary Cholangitis, run by Eli Lilly and Company at 12 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 42 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Alkaline Phosphatase (ALP) (time frame: Baseline, Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2020.