Primary Biliary CholangitisPhase 2NCT02516605

A Multi-part, Double Blind Study to Assess Safety, Tolerability and Efficacy of Tropifexor (LJN452) in PBC Patients

A Phase 2 study testing Part 1: LJN452, Part 2: LJN452 Dose level 1 and Part 2: LJN452 Dose level 2 in people with Primary Biliary Cholangitis, run by Novartis Pharmaceuticals at 28 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

A multi-part study to assess safety, tolerability and efficacy of tropifexor (LJN452) in patients with primary biliary cholangitis 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?: Part 1: LJN452, Part 2: LJN452 Dose level 1 and Part 2: LJN452 Dose level 2
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 61 participants
Where: 28 sitesin 6 countries
Study dates: 9 Sep 2015to 2 Aug 2018
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

Age ≥ 18 years
Diagnosis of PBC as demonstrated by the presence of at least 2 of the following 3 diagnostic criteria:
History of alkaline phosphatase (ALP) elevated above upper limit of normal (ULN) for at least 6 months
Positive antimitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti-GP210 and/or anti-SP100 and/or antibodies against the major M2 components (PDC-E2, 2-oxo-glutaric acid dehydrogenase complex))
Previous liver biopsy findings consistent with PBC
At least 1 of the following markers of disease severity:

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ALP ≥ 1.67 × ULN
Total bilirubin > ULN but < 1.5 × ULN
In addition, patients must meet the following biochemical criteria at enrollment:
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 5 × ULN
Total bilirubin ≤ 1.5 × ULN
INR ≤ ULN
Taking UDCA for at least 12 months, or for at least 6 months and has reached maximal response to UDCA with a plateau in alkaline phosphatase, with no changes in dose for ≥ 3 months prior to Day 1.
Patients must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) within the range of 18 - 40 kg/m2. BMI = Body weight (kg) / [Height (m)]2

Who cannot join

Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception for 30 days before randomization, during dosing and for 30 days following the end of treatment.
Presence of other concomitant liver diseases.
Cirrhosis with complications, including history or presence of:
Variceal bleed
Uncontrolled ascites
Encephalopathy

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Spontaneous bacterial peritonitis
Significant hepatic impairment as defined by Child-Pugh classification of B or C, history of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥15.
History of conditions that may cause increases in ALP (e.g., Paget's disease).
Use of investigational drugs, or immunosuppressive drugs at the time of enrollment, or within 5 half-lives, or 30 days of randomization, whichever is longer; or longer if required by local regulations. Use of high dose oral steroids to treat co-morbid conditions (e.g., airways disease) will be allowed but must be properly documented as such in concomitant medications.
Currently taking obeticholic acid or have taken obeticholic acid within 30 days of randomization
Previous participation in CLJN452X2201 and received study medication within three months of randomization (or longer if required by local regulations).

Trial locations and contacts

28 sites in 6 countries.

Country

City, postcode or hospital

28 sites in 6 countries

United States9 sites

Novartis Investigative Site
Atlanta, Georgia, 30308
Novartis Investigative Site
Chicago, Illinois, 60612
Novartis Investigative Site
Dallas, Texas, 75390
Novartis Investigative Site
Manhasset, New York, 11030
Novartis Investigative Site
Marietta, Georgia, 30060
Novartis Investigative Site
Miami, Florida, 33136
Novartis Investigative Site
Rialto, California, 92377
Novartis Investigative Site
San Antonio, Texas, 78215
Novartis Investigative Site
Seattle, Washington, 98104

Other trials in United States

Germany5 sites

Novartis Investigative Site
Hamburg, 20246
Novartis Investigative Site
Hanover, 30625
Novartis Investigative Site
Heidelberg, 69120
Novartis Investigative Site
München, 81377
Novartis Investigative Site
Würzburg, 97080

Other trials in Germany

United Kingdom5 sites

Novartis Investigative Site
Birmingham, West Midlands, B15 2TH
Novartis Investigative Site
Cambridge, CB2 2QQ
Novartis Investigative Site
Hull, HU3 2JZ
Novartis Investigative Site
London, NW3 2PF
Novartis Investigative Site
Newcastle upon Tyne, NE1 4LP

Other trials in United Kingdom

Poland4 sites

Novartis Investigative Site
Lodz, 91-347
Novartis Investigative Site
Mysłowice, 41-400
Novartis Investigative Site
Warsaw, 02-097
Novartis Investigative Site
Wroclaw, 50-449

Other trials in Poland

Russia3 sites

Novartis Investigative Site
Moscow, 117198
Novartis Investigative Site
Saint Petersburg, 194044
Novartis Investigative Site
Samara, 443011

Other trials in Russia

Canada2 sites

Novartis Investigative Site
Calgary, Alberta, T2N 4N1
Novartis Investigative Site
Edmonton, Alberta, T6G 2B7

Other trials in Canada

About this study

Official title: A Multi-part, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability and Efficacy of Tropifexor (LJN452) in Patients With Primary Biliary Cholangitis

A multi-part study to assess safety, tolerability and efficacy of tropifexor (LJN452) in patients with primary biliary cholangitis

Conditions: Primary Biliary Cholangitis
Being tested: Part 1: LJN452, Part 1: Placebo, Part 2: LJN452 Dose level 1, Part 2: Placebo, Part 2: LJN452 Dose level 2
Keywords: Primary Biliary Cirrhosis, PBC, Primary Biliary Cholangitis
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.

Part 1: LJN452Drug
Also called tropifexor
LJN452 capsules administered once daily for 28 days
Part 1: PlaceboDrug
Matching placebo capsules administered once daily for 28 days
Part 2: LJN452 Dose level 1Drug
Also called tropifexor
LJN452 capsules administered once a day for 12 weeks
Part 2: PlaceboDrug
Matching placebo to LJN452 administered once a day for 12 weeks
Part 2: LJN452 Dose level 2Drug
Also called tropifexor
LJN452

Study groups in NCT02516605
Study group	Role	What they receive
LJN452	Experimental	Not described Receives: Drug: Part 1: LJN452; Drug: Part 2: LJN452 Dose level 1; Drug: Part 2: LJN452 Dose level 2
Placebo	Placebo comparator	Not described Receives: Drug: Part 1: Placebo; Drug: Part 2: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Fold Change in Serum Gamma-glutamyl Transferase (GGT)
Fold change in serum gamma-glutamyl transferase (GGT) from baseline to Day 28
Measured: Baseline to Day 28
Blood Pressure
Vital signs - Systolic Blood pressure
Measured: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Pulse Rate
Vital signs
Measured: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Body Temperature
Vital signs
Measured: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
ECG - Heart Rate
Electrocardiogram (ECG)
Measured: Screening, Baseline, day 1, day 28
ECG Intervals - PR Interval
Electrocardiogram (ECG)
Measured: Screening, Baseline, day 1, day 28
Haemoglobin
Hematology panel for safety laboratory assessments.
Measured: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84

Secondary outcomes (6)

Plasma PK Parameter - AUC 0-8h
Tropifexor levels were determined in plasma using a validated LC-MS/MS method. AUC0-t=The area under the plasma concentration-time curve from time zero to time 't' where t is a defined time point after administration [mass x time / volume]
Measured: Day 1, Day 28
Plasma PK Parameter - Cmax
Tropifexor levels were determined in plasma using a validated LC-MS/MS method. Cmax=The observed maximum plasma concentration following drug administration [mass /volume]
Measured: Day 1, Day 28
Plasma PK Parameter - Tmax
Tropifexor levels were determined in plasma using a validated LC-MS/MS method. Tmax = The time to reach the maximum concentration after drug administration [time]
Measured: Day 1, Day 28
Changes From Baseline in Total PBC-40 Score
Baseline is defined as the latest available predose value. The PBC-40 is a paper-based, patient-derived, disease-specific health-related quality of life (HRQOL) patient reported outcome (PRO) measure which was developed and validated for use in PBC patients (Jacoby et al 2005). It consists of 40 questions arranged in 6 domains with between 3 and 11 questions in each domain. Each question is scored from 1 to 5 in increasing order of severity. All questions within a domain are summed and all domain totals are summed to obtain a total score. The total score range is between a minimum of 40 and a maximum of 200. Higher scores represent a poorer quality of life. The median difference from baseline in total sum score for each treatment group is presented.
Measured: Baseline, Day 28, Day 56, Day 84
Change From Baseline in Itch Subdomain of PBC-40 Score
Baseline is defined as the latest available predose value. The PBC-40 is a paper-based, patient-derived, disease-specific health-related quality of life (HRQOL) patient reported outcome (PRO) measure which was developed and validated for use in PBC patients (Jacoby et al 2005). It consists of 40 questions arranged in 6 domains with between 3 and 11 questions in each domain. Each question is scored from 1 to 5 in increasing order of severity. This dataset focuses on the itch subdomain which consists of 3 questions. These 3 questions within the itch subdomain are summed to obtain a total score for the itch subdomain. The total score range is between a minimum of 3 and a maximum of 15. Higher scores represent a poorer quality of life. The median change from baseline in total itch subdomain score in each treatment group is presented.
Measured: Baseline, Day 28, Day 56, Day 84
Change From Baseline in Global Itch Visual Analogue Scale (VAS)
Baseline is defined as the latest available predose value. The Global Itch Visual Analogue Scale, a 100 mm visual analogue scale (VAS), was used to assess the severity of patients itch (ranging from 0 = none at all to 100 = the worst imaginable itch). The score range is between a minimum of 0 and a maximum of 100. The score (distance in mm from left) on the VAS was recorded by the patient marking with a line and used to test for an effect of tropifexor over placebo. The mean change from baseline in itch VAS score in each treatment group is presented.
Measured: Day 7, Day 14, Day 21, Day 28, Day 56, and Day 84

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

9 Sep 20152 Aug 2018

First posted6 Aug 2015 (estimated)
Study start9 Sep 2015
Primary completion2 Aug 2018
Study completion2 Aug 2018

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe 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: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 61 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02516605
Sponsor's protocol ID: CLJN452X2201
Other IDs: 2015-001590-41, 2015
Status last verified: Oct 2019
First submitted: 4 Aug 2015
Last updated: 5 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

Schramm C, Wedemeyer H, Mason A, Hirschfield GM, Levy C, Kowdley KV, Milkiewicz P, Janczewska E, Malova ES, Sanni J, Koo P, Chen J, Choudhury S, Klickstein LB, Badman MK, Jones D. Farnesoid X receptor agonist tropifexor attenuates cholestasis in a randomised trial in patients with primary biliary cholangitis. JHEP Rep. 2022 Jul 21;4(11):100544. doi: 10.1016/j.jhepr.2022.100544. eCollection 2022 Nov.
PubMed 36267872

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multi-part, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability and Efficacy of Tropifexor (LJN452) in Patients With Primary Biliary Cholangitis
ClinicalTrials.gov ID: NCT02516605
Acronym / sponsor ID: CLJN452X2201
Conditions: Primary Biliary Cholangitis
Keywords: Primary Biliary Cirrhosis, PBC, Primary Biliary Cholangitis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 61 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 9 Sep 2015 (actual)
Primary completion: 2 Aug 2018 (actual)
Study completion: 2 Aug 2018 (actual)
First posted: 6 Aug 2015 (estimated)
Results: Posted 13 Nov 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

A multi-part study to assess safety, tolerability and efficacy of tropifexor (LJN452) in patients with primary biliary cholangitis

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
LJN452	Experimental	Drug: Part 1: LJN452, Drug: Part 2: LJN452 Dose level 1, Drug: Part 2: LJN452 Dose level 2
Placebo	Placebo comparator	Drug: Part 1: Placebo, Drug: Part 2: Placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (14 listed)

Age ≥ 18 years
Diagnosis of PBC as demonstrated by the presence of at least 2 of the following 3 diagnostic criteria:
At least 1 of the following markers of disease severity:
In addition, patients must meet the following biochemical criteria at enrollment:
Taking UDCA for at least 12 months, or for at least 6 months and has reached maximal response to UDCA with a plateau in alkaline phosphatase, with no changes in dose for ≥ 3 months prior to Day 1.

Key exclusion criteria (12 listed)

Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception for 30 days before randomization, during dosing and for 30 days following the end of treatment.
Presence of other concomitant liver diseases.
Significant hepatic impairment as defined by Child-Pugh classification of B or C, history of liver transplantation, current placement on a liver transplant list or current Model for End Stage Liver Disease (MELD) score ≥15.
History of conditions that may cause increases in ALP (e.g., Paget's disease).
Use of investigational drugs, or immunosuppressive drugs at the time of enrollment, or within 5 half-lives, or 30 days of randomization, whichever is longer; or longer if required by local regulations. Use of high dose oral steroids to treat co-morbid conditions (e.g., airways disease) will be allowed but must be properly documented as such in concomitant medications.

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
Part 1: LJN452Also: tropifexorLJN452 capsules administered once daily for 28 days	Drug	LJN452
Part 1: PlaceboMatching placebo capsules administered once daily for 28 days	Drug	Placebo
Part 2: LJN452 Dose level 1Also: tropifexorLJN452 capsules administered once a day for 12 weeks	Drug	LJN452
Part 2: PlaceboMatching placebo to LJN452 administered once a day for 12 weeks	Drug	Placebo
Part 2: LJN452 Dose level 2Also: tropifexorLJN452	Drug	LJN452

Primary outcome

Fold Change in Serum Gamma-glutamyl Transferase (GGT)
Time frame: Baseline to Day 28
Fold change in serum gamma-glutamyl transferase (GGT) from baseline to Day 28
Blood Pressure
Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Vital signs - Systolic Blood pressure
Pulse Rate
Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Vital signs
Body Temperature
Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Vital signs
ECG - Heart Rate
Time frame: Screening, Baseline, day 1, day 28
Electrocardiogram (ECG)
ECG Intervals - PR Interval
Time frame: Screening, Baseline, day 1, day 28
Electrocardiogram (ECG)
Haemoglobin
Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Hematology panel for safety laboratory assessments.

6 secondary outcomes

Plasma PK Parameter - AUC 0-8h
Time frame: Day 1, Day 28
Plasma PK Parameter - Cmax
Time frame: Day 1, Day 28
Plasma PK Parameter - Tmax
Time frame: Day 1, Day 28
Changes From Baseline in Total PBC-40 Score
Time frame: Baseline, Day 28, Day 56, Day 84
Change From Baseline in Itch Subdomain of PBC-40 Score
Time frame: Baseline, Day 28, Day 56, Day 84
Change From Baseline in Global Itch Visual Analogue Scale (VAS)
Time frame: Day 7, Day 14, Day 21, Day 28, Day 56, and Day 84

Results and safety

Results posted to the registry on 13 Nov 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
LJN452 - 0.03 mg qd	11	11
LJN452 - 0.06 mg qd	9	9
LJN452 - 0.09 mg qd	12	12
LJN452 - 0.15 mg qd	8	7
Placebo qd	21	20

Primary outcome: Fold Change in Serum Gamma-glutamyl Transferase (GGT)

Time frame: Baseline to Day 28. Mean (90% Confidence Interval). Unit: fold-change

Measure	LJN452 - 0.03 mg qd n = 10	LJN452 - 0.06 mg qd n = 9	LJN452 - 0.09 mg qd n = 12	LJN452 - 0.15 mg qd n = 8	Pooled Placebo n = 20
Value	0.74 (0.61 to 0.90)	0.41 (0.33 to 0.50)	0.28 (0.23 to 0.33)	0.31 (0.24 to 0.40)	0.86 (0.75 to 0.99)

LJN452 - 0.03 mg qd vs Pooled Placebo: adjusted fold change from baseline: 0.86 (90% CI 0.68 to 1.09), p = 0.293, ANCOVA
LJN452 - 0.06 mg qd vs Pooled Placebo: adjusted fold change from baseline: 0.47 (90% CI 0.37 to 0.60), p <0.001, ANCOVA
LJN452 - 0.09 mg qd vs Pooled Placebo: adjusted fold change from baseline: 0.32 (90% CI 0.26 to 0.40), p <.001, ANCOVA

Population analysed: The PD analysis set included all subjects with any available PD data and no protocol deviations with relevant impact on PD data.

Primary outcome: Blood Pressure

Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84. Mean (Standard Deviation). Unit: mm Hg

Measure	LJN452 - 0.03 mg qd n = 11	LJN452 - 0.06 mg qd n = 9	LJN452 - 0.09 mg qd n = 12	LJN452 - 0.15 mg qd n = 8	Placebo qd n = 21
Screening	122.5 (12.21)	125.8 (11.79)	129.3 (14.42)	129.5 (17.42)	123.0 (15.40)
Baseline	124.7 (17.66)	128.4 (13.83)	122.0 (18.18)	132.3 (18.09)	118.7 (12.76)
Day 1	129.6 (20.48)	122.4 (20.24)	119.9 (11.84)	129.1 (25.12)	123.0 (21.84)
Day 7	127.2 (20.18)	123.7 (13.04)	125.6 (14.22)	128.0 (26.58)	120.9 (19.57)
Day 14	122.4 (16.30)	124.6 (20.67)	127.1 (16.45)	127.3 (12.45)	118.7 (12.88)
Day 21	121.5 (11.25)	126.3 (27.65)	123.9 (13.14)	124.0 (14.23)	124.5 (19.8)
Day 28	118.0 (11.22)	127.1 (28.55)	121.4 (12.82)	126.0 (7.71)	120.1 (22.38)
Day 56	121.4 (10.76)	125.4 (18.48)	121.5 (11.55)	129.1 (26.45)	123.8 (19.74)

Population analysed: Safety Analysis Set

Primary outcome: Pulse Rate

Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84. Mean (Standard Deviation). Unit: bpm

Measure	LJN452 - 0.03 mg qd n = 11	LJN452 - 0.06 mg qd n = 9	LJN452 - 0.09 mg qd n = 12	LJN452 - 0.15 mg qd n = 8	Placebo qd n = 21
Screening	68.6 (10.63)	61.2 (7.05)	75.3 (8.22)	72.8 (8.26)	69.3 (10.69)
Baseline	64.3 (7.79)	64.1 (8.70)	70.8 (8.63)	74.4 (5.15)	66.3 (9.30)
Day 1	67.7 (11.93)	64.2 (8.07)	69.7 (7.50)	74.1 (11.62)	67.6 (8.35)
Day 7	65.2 (9.98)	66.8 (11.31)	70.1 (11.64)	73.5 (8.33)	65.2 (8.92)
Day 14	62.9 (10.03)	64.8 (8.09)	70.5 (14.16)	75.1 (6.74)	66.1 (9.61)
Day 21	65.5 (8.26)	65.8 (11.18)	73.2 (8.43)	72.5 (6.75)	67.7 (10.18)
Day 28	65.5 (10.83)	68.9 (10.60)	68.5 (9.01)	67.2 (3.42)	65.8 (9.29)
Day 56	66.8 (9.64)	67.7 (7.53)	69.7 (8.17)	69.7 (4.07)	68.1 (10.11)

Population analysed: Safety Analysis Set

Primary outcome: Body Temperature

Time frame: Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84. Mean (Standard Deviation). Unit: Celsius

Measure	LJN452 - 0.03 mg qd n = 11	LJN452 - 0.06 mg qd n = 9	LJN452 - 0.09 mg qd n = 12	LJN452 - 0.15 mg qd n = 8	Placebo qd n = 21
Screening	36.605 (0.2813)	36.567 (0.2000)	36.424 (0.3429)	36.650 (0.1604)	36.460 (0.4500)
baseline	36.582 (0.2960)	36.644 (0.2128)	36.398 (0.3385)	36.638 (0.1506)	36.376 (0.5118)
day 1	36.436 (0.3139)	36.537 (0.3076)	36.482 (0.2636)	36.575 (0.1982)	36.390 (0.4867)
day 7	36.582 (0.2040)	36.533 (0.1323)	36.258 (0.3965)	36.600 (0.1195)	36.345 (0.4639)
day 14	36.516 (0.1793)	36.444 (0.2007)	36.291 (0.4109)	36.500 (0.1826)	36.310 (0.4576)
day 21	36.500 (0.2490)	36.267 (0.4637)	36.308 (0.3288)	36.433 (0.1966)	36.455 (0.4236)
day 28	36.382 (0.2228)	36.444 (0.2128)	36.308 (0.3397)	36.520 (0.2950)	36.380 (0.4047)
day 56	36.527 (0.2102)	36.500 (0.2179)	36.350 (0.4602)	36.443 (0.3309)	36.285 (0.4246)

Population analysed: Safety Analysis Set

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 56 days post last dose.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
LJN452 - 0.03 mg qd	0 of 11 (0%)	9 of 11 (81.8%)	0 of 11
LJN452 - 0.06 mg qd	0 of 9 (0%)	8 of 9 (88.9%)	0 of 9
LJN452 - 0.09 mg qd	0 of 12 (0%)	11 of 12 (91.7%)	0 of 12
LJN452 - 0.15 mg qd	0 of 8 (0%)	8 of 8 (100%)	0 of 8
Placebo qd	0 of 21 (0%)	16 of 21 (76.2%)	0 of 21

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 36267872Schramm C, Wedemeyer H, Mason A, Hirschfield GM, Levy C, Kowdley KV, Milkiewicz P, Janczewska E, Malova ES, Sanni J, Koo P, Chen J, Choudhury S, Klickstein LB, Badman MK, Jones D. Farnesoid X receptor agonist tropifexor attenuates cholestasis in a randomised trial in patients with primary biliary cholangitis. JHEP Rep. 2022 Jul 21;4(11):100544. doi: 10.1016/j.jhepr.2022.100544. eCollection 2022 Nov.
A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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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.

The record lists no contact. Use the official record to reach the sponsor.

28 sites in 6 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 14 inclusion and 12 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: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in United States, Germany, United Kingdom, Poland, Russia and 1 more country. 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 13 Nov 2019). 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?

Fold Change in Serum Gamma-glutamyl Transferase (GGT) (time frame: Baseline to Day 28). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02516605, which also lists 1 linked publication.

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 5 Jan 2021.