Adults 18 to 75, any sex, with Primary Sclerosing Cholangitis (PSC). Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
DB Phase: Change From Baseline In Serum Alkaline Phosphatase (ALP)Primary· Baseline, Week 24
The primary efficacy analysis compared the Week 24 change from Baseline in ALP between OCA treatment group and placebo using an analysis of covariance (ANCOVA) model with fixed effects for treatment group and randomization strata, and Baseline as a covariate. Results are reported in U/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
-105.05
± 38.02
5 mg OCA Titrating to 10 mg OCA
-110.19
± 33.77
Placebo
-26.76
± 36.65
LTSE Phase: Incidence Of Adverse Events Of Special Interest (AESIs)Primary· LTSE Baseline (DB Week 24) to Month 26
The primary safety analysis evaluated the effects of OCA treatment on AESIs of pruritus, hepatic disorders, and dyslipidemia. All adverse event (AE) summaries were restricted to treatment emergent AEs (TEAEs), which were defined as any AEs that newly appeared, increased in frequency, or worsened in severity following initiation of investigational product. Treatment-emergent pruritus was defined as any preferred term (PT) including "Prur-". Hepatic disorder AESIs were defined using specific Hepatic Disorders Standardized Medical Dictionary for Regulatory Activities (MedDRA) Query (SMQ) terms. A
Dyslipidaemia
Group
Value
95% CI
LTSE OCA Total
1
Hepatic Disorder
Group
Value
95% CI
LTSE OCA Total
23
Pruritus
Group
Value
95% CI
LTSE OCA Total
34
DB Phase: Change From Baseline In Serum Alanine Transaminase (ALT)Secondary· Baseline, Week 24
As a marker of hepatic biochemistry and liver function, the median change in ALT from Baseline at Week 24 is reported. Results are reported in U/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
-33.0
-50.5 – 5.0
5 mg OCA Titrating to 10 mg OCA
-5.5
-30.0 – 4.5
Placebo
-19.5
-49.0 – 4.0
DB Phase: Change From Baseline In Serum Aspartate Aminotransferase (AST)Secondary· Baseline, Week 24
As a marker of hepatic biochemistry and liver function, the median change in AST from Baseline at Week 24 is reported. Results are reported in U/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
-8.0
-20.0 – 19.0
5 mg OCA Titrating to 10 mg OCA
0.5
-17.5 – 32.8
Placebo
-14.0
-35.5 – 8.0
DB Phase: Change From Baseline In Serum Total BilirubinSecondary· Baseline, Week 24
As a marker of hepatic biochemistry and liver function, the median change in serum total bilirubin from Baseline at Week 24 is reported. Results are reported in umol/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
0.8
-1.7 – 4.3
5 mg OCA Titrating to 10 mg OCA
1.3
-1.3 – 6.9
Placebo
0.0
-5.1 – 4.3
DB Phase: Change From Baseline In Serum Direct BilirubinSecondary· Baseline, Week 24
As a marker of hepatic biochemistry and liver function, the median change in serum direct bilirubin from Baseline at Week 24 is reported. Results are reported in umol/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
0.8
-0.9 – 0.9
5 mg OCA Titrating to 10 mg OCA
0.9
-0.9 – 6.4
Placebo
0.0
-1.7 – 4.3
DB Phase: Change From Baseline In Serum Gamma-glutamyl Transferase (GGT)Secondary· Baseline, Week 24
As a marker of hepatic biochemistry and liver function, the median change in serum GGT from Baseline at Week 24 is reported. Results are reported in U/L.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
-79.0
-171.0 – -9.7
5 mg OCA Titrating to 10 mg OCA
-78.5
-235.5 – 14.0
Placebo
-89.0
-167.0 – 20.0
DB Phase: Change From Baseline In Plasma Fibroblast Growth Factor-19 (FGF-19)Secondary· Baseline, Week 24
To assess farnesoid X receptor (FXR) activity, the change in FGF-19 from Baseline at Week 24 is reported. Results are reported in picograms/milliliter (pg/mL).
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
32.00
-16.00 – 110.00
5 mg OCA Titrating to 10 mg OCA
147.00
-6.35 – 714.50
Placebo
-19.50
-79.56 – 28.00
DB Phase: Change From Baseline In Plasma 7α-Hydroxy-4-cholesten-3-one (C4)Secondary· Baseline, Week 24
To assess FXR activity, the change in plasma C4 from Baseline at Week 24 is reported. Results are reported in nanograms (ng)/mL.
Group
Value
95% CI
1.5 mg OCA Titrating to 3 mg OCA
-2.80
-7.50 – 0.55
5 mg OCA Titrating to 10 mg OCA
-2.90
-6.94 – -1.12
Placebo
0.05
-2.25 – 10.84
LTSE Phase: Change From Baseline In Serum ALP At Month 12Secondary· LTSE Baseline (DB Week 24), Month 12
The median change in serum ALP from Baseline to the last available visit is reported. The DB value at Week 24 was used as the Baseline. Results are reported in U/L.
Group
Value
95% CI
LTSE OCA Total
-91.5
-144.0 – -17.0
LTSE Phase: Change From Baseline In Serum ALT At Month 12Secondary· LTSE Baseline (DB Week 24), Month 12
As a marker of hepatic biochemistry and liver function, the median change in ALT from Baseline at Month 12 is reported. The DB value at Week 24 was used as the Baseline. Results are reported in U/L.
Group
Value
95% CI
LTSE OCA Total
-37.0
-60.5 – -7.5
LTSE Phase: Change From Baseline In Serum AST At Month 12Secondary· LTSE Baseline (DB Week 24), Month 12
As a marker of hepatic biochemistry and liver function, the median change in AST from Baseline at Month 12 is reported. The DB value at Week 24 was used as the Baseline. Results are reported in U/L.
Group
Value
95% CI
LTSE OCA Total
-14.5
-30.5 – 9.5
Adverse events — posted to ClinicalTrials.gov
Time frame: DB Phase: From informed consent to end of double-blind phase study participation, up to 24 weeks. LTSE Phase: Baseline (DB Week 24) to Month 26..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This was a phase 2, double-blind (DB), placebo-controlled trial in participants with primary sclerosing cholangitis to evaluate the effect of obeticholic acid on liver biochemistry, in particular, serum alkaline phosphatase; and, safety. The long-term safety extension (LTSE) phase was conducted to evaluate the safety, tolerability, and efficacy of long-term, open-label use of OCA in participants with PSC who had completed the DB phase of the study.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03633227 — Study of Obeticholic Acid (OCA) Evaluating Pharmacokinetics and Safety in Participants With Primary Biliary Cholangitis
· Phase 4
· terminated
NCT02308111 — Phase 4 Study of Obeticholic Acid Evaluating Clinical Outcomes in Patients With Primary Biliary Cholangitis
· Phase 4
· terminated
NCT00570765 — Study of INT-747 as Monotherapy in Participants With Primary Biliary Cirrhosis (PBC)
· Phase 2
· completed
Other recruiting trials for Primary Sclerosing Cholangitis (PSC)
Currently open trials in the same condition.
NCT06026865 — S-adenosylmethionine (SAMe) in Patients With Primary Sclerosing Cholangitis (PSC)
· NA
· active not recruiting
NCT03146936 — Swiss Primary Sclerosing Cholangitis Cohort Study
· recruiting
Other Intercept Pharmaceuticals trials
Trials by the same sponsor.
NCT06121375 — Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OC
· Phase 2, PHASE3
· terminated
NCT06488911 — To Evaluate Safety and Tolerability of the Fixed- Dose Combination of Obeticholic Acid and Bezafibrate
· Phase 3
· terminated
NCT05639543 — FXR Effect on Severe Alcohol-Associated Hepatitis (FRESH) Study
· Phase 2
· recruiting
NCT05239468 — Study of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC
· Phase 2
· completed
NCT04594694 — Study of OCA in Combination With BZF Evaluating Efficacy, Safety, and Tolerability in Participants With PBC
· Phase 2
· terminated
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Intercept Pharmaceuticals
Last refreshed: 8 July 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02177136.