Trial · NCT03439254
Study Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis
NCT03439254, Phase 3, Completed, Intercept Pharmaceuticals; results first posted 2023-10-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis. Trials, record NCT03439254. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03439254
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis
- NCT number
- NCT03439254
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 919 participants, actual
- Lead sponsor
- Intercept Pharmaceuticals
- Conditions
- Compensated Cirrhosis, Nonalcoholic Steatohepatitis
- Interventions
- Obeticholic acid (10 mg), Obeticholic acid (10 mg to 25 mg), Placebo
- First posted
- 2018-02-20
- Last updated
- 2023-10-23
- Results first posted
- 2023-10-23
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) | Up to 18 months | Fibrosis stage was evaluated by NASH Clinical Research Network(CRN)Fibrosis Staging System with stages:0=no fibrosis;1=perisinusoidal/periportal;1A=mild,zone 3,perisinusoidal;1B=moderate,zone 3,perisinusoidal;1C=portal/periportal;2=perisinusoidal and portal/periportal;3=bridging fibrosis;4=cirrhosis.No worsening of steatohepatitis was defined as no worsening of lobular inflammation or hepatocellular ballooning grade as per scoring in relevant nonalcoholic fatty liver disease activity score (NAS) categories.NAS is semiquantitative scoring system based on unweighted sum of:steatosis (0=\<5% to 3=\>66%),lobular inflammation(0=no foci to 3=\>4 foci/200x),hepatocellular ballooning(0=none to 2=many cells/prominent ballooning)scores.Total scale range:0-12;0:no features of fatty liver disease and 12:highest degree of fatty liver disease.Higher scores:worse symptoms.Responders:did not discontinue treatment due to Adverse event(AE) or did not die and had evaluable post-Baseline biopsy assessment |
| Primary | OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | Up to 12 months | An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment. |
| Primary | OLE Phase: Change From Baseline to Month 12 in Liver Stiffness Measurement (LSM) | Baseline and up to Month 12 | Non-invasive radiological methods to assess liver stiffness were conducted at selected study sites where the respective devices were available. These assessments were taken by vibration controlled transient elastography (TE) method using FibroScan®. Participant was included as a random effect and an unstructured covariance matrix was used assuming convergence could be attained. Baseline was defined as the last value collected prior to the first administration of the investigational product (IP). Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Primary | OLE Phase: Fibrosis-4 (FIB-4) at Baseline | Baseline (Day 1) | FIB-4 was a noninvasive assessment of liver disease assessed by a combination of age, alanine aminotransferase (ALT) and platelet results. FIB-4 was the ratio of age in years and aminotransferase to platelet count. It was a non-invasive hepatic fibrosis index score combining standard biochemical values, platelets, ALT, Aspartate aminotransferase (AST) and age that was calculated using formula: FIB-4 = (Age \[years\] x AST \[Units per Liter {U/L}\]) / (platelets \[10\^9/L\] x (square root of ALT \[U/L\])). A FIB-4 index of \<1.45 indicated no or moderate fibrosis and an index of \> 3.25 indicated extensive fibrosis/cirrhosis. Higher ratio indicated worse condition. Baseline was defined as the last value collected prior to the first administration of the IP. |
| Primary | OLE Phase: Enhanced Liver Fibrosis (ELF) at Baseline | Baseline (Day 1) | ELF was non-invasive panel of circulating fibrosis markers calculated from serum biomarkers. The markers of fibrosis comprised hyaluronic acid (HA), tissue inhibitor of metalloproteinase (TIMP1) and procollagen III N-terminal peptide (PIIINP). Each of these markers was measured by an immunoassay and an ELF score was generated, from which a level of fibrosis severity could be determined. The ELF test was a composite score: \< 7.7: no to mild fibrosis; ≥ 7.7 - \< 9.8: Moderate fibrosis; ≥ 9.8 - \< 11.3: Severe fibrosis; ≥ 11.3: Cirrhosis.; higher ELF scores were associated with worsening liver fibrosis. Baseline was defined as the last value collected prior to the first administration of the IP. |
| Primary | OLE Phase: Number of Participants Reporting All-cause Mortality | Up to Month 12 | All-cause mortality is defined as death due to any cause. Number of participants reporting all-cause mortality is presented |
| Primary | OLE Phase: Number of Participants With Adjudicated Liver Related Clinical Outcomes: Ascites, Hepatocellular Carcinoma (HCC) and Non-liver Related Death | Up to 12 months | Adjudication was performed under the review of Hepatic Safety Adjudication Committee (HSAC) of all available data for each identified participant to determine liver injury status. Number of participants with adjudicated liver related clinical outcomes for the following is presented: Ascites (secondary to cirrhosis and requiring medical intervention), Hepatocellular carcinoma (HCC) and non-liver related death. |
| Primary | OLE Phase: Number of Participants With Adjudicated Liver Related Clinical Outcomes: Worsening of Child-Pugh Score | Up to 12 months | The Child-Pugh classification was a scoring system used for the classification of the severity of cirrhosis. It included three continuous variables (bilirubin, albumin, and international normalized ratio) and two discrete variables (ascites and encephalopathy). Each variable was scored 1-3 with 3 indicating most severe derangement. The determination of Child-Pugh score ranged from 5 to 15. The higher the score, the sicker the participant. Adjudication was performed under the review of HSAC of all available data for each identified participant to determine liver injury status. Number of participants with adjudicated liver related clinical outcomes for worsening of Child-Pugh score is presented. |
| Primary | OLE Phase: Number of Participants With Adjudicated Liver Related Clinical Outcomes: Model for End-Stage Liver Disease (MELD) Score ≥15 | Up to 12 months | MELD was a scoring system for assessing the severity of chronic liver disease and to assess prognosis and suitability for liver transplantation. It uses the participant's values for total bilirubin, serum creatinine, and the international normalized ratio for prothrombin time to predict survival. MELD score ranges from 6 (less ill) to 40 (gravely ill) with scores and mortality probability being: Score 40=71.3% mortality; Scores 30-39=52.6% mortality; Scores 20-29=19.6% mortality; Scores10-19=6.0% mortality; Score 9 or less=1.9% mortality. Higher scores indicated greater disease severity. Adjudication was performed under the review of HSAC of all available data for each identified participant to determine liver injury status. Number of participants with adjudicated liver related clinical outcomes for MELD score ≥15 is presented. |
| Secondary | DB Phase: Change From Baseline to Month 18 in LSM | Baseline and up to Month 18 | Non-invasive radiological methods to assess liver stiffness were conducted at selected study sites where the respective devices were available. These assessments were taken by vibration controlled TE method using FibroScan®. Participant was included as a random effect and an unstructured covariance matrix was used assuming convergence could be attained. The principal comparison was at Month 18. Baseline was defined as the last value collected prior to the first administration of the IP. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) | Up to 18 months | ITT Population. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | Up to 12 months | Safety Population: included all randomized participants who received at least 1 dose of investigational product (OCA or placebo). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | OLE Phase: Change From Baseline to Month 12 in Liver Stiffness Measurement (LSM) | Baseline and up to Month 12 | ITT Population. Only those participants with data available at the specified data points were analyzed. | Kilopascal (kPa) | MEDIAN |
| PRIMARY | OLE Phase: Fibrosis-4 (FIB-4) at Baseline | Baseline (Day 1) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Ratio | MEAN |
| PRIMARY | OLE Phase: Enhanced Liver Fibrosis (ELF) at Baseline | Baseline (Day 1) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | MEAN |
| PRIMARY | OLE Phase: Number of Participants Reporting All-cause Mortality | Up to Month 12 | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Phase: Change From Baseline to Month 18 in LSM | Baseline and up to Month 18 | ITT Population. Only those participants with data available at the specified data points were analyzed. | Kilopascal (kPa) | MEDIAN |
| SECONDARY | DB Phase: FIB-4 at Baseline | Baseline (Day 1) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Ratio | MEAN |
| SECONDARY | DB Phase: ELF at Baseline | Baseline (Day 1) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | MEAN |
| PRIMARY | OLE Phase: Number of Participants With Adjudicated Liver Related Clinical Outcomes: Ascites, Hepatocellular Carcinoma (HCC) and Non-liver Related Death | Up to 12 months | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group45
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) | DB: Placebo | 31 | Participants | ||
| DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) | DB: OCA 10 Milligrams (mg) | 33 | Participants | ||
| DB Phase: Number of Participants Who Were Responders and Showed Improvement in Fibrosis by at Least 1 Stage Without Worsening of Nonalcoholic Steatohepatitis (NASH) | DB: OCA 10 mg Titrated to OCA 25 mg | 37 | Participants | ||
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: OCA 10 mg (DB Placebo) | AEs | 199 | Participants | |
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: OCA 10 mg (DB OCA 10 mg) | AEs | 213 | Participants | |
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) | AEs | 197 | Participants | |
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: OCA 10 mg (DB Placebo) | SAEs | 26 | Participants | |
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: OCA 10 mg (DB OCA 10 mg) | SAEs | 50 | Participants | |
| OLE Phase: Number of Participants With Non-serious Adverse Events (AEs) and Serious Adverse Events (SAEs) | OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) | SAEs | 48 | Participants | |
| OLE Phase: Change From Baseline to Month 12 in Liver Stiffness Measurement (LSM) | OLE: OCA 10 mg (DB Placebo) | -2.10 | Inter-Quartile Range -7.40 to 3.40 | Kilopascal (kPa) |
1 to 10 of 45Source ClinicalTrials.gov
See all 45 results by groupParticipant flow36
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind Phase (18 Months) | STARTED | DB: Placebo | 313 | |
| Double-blind Phase (18 Months) | STARTED | DB: OCA 10 Milligrams (mg) | 296 | |
| Double-blind Phase (18 Months) | STARTED | DB: OCA 10 mg Titrated to OCA 25 mg | 310 | |
| Double-blind Phase (18 Months) | STARTED | OLE: OCA 10 mg (DB Placebo) | 0 | |
| Double-blind Phase (18 Months) | STARTED | OLE: OCA 10 mg (DB OCA 10 mg) | 0 | |
| Double-blind Phase (18 Months) | STARTED | OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) | 0 | |
| Double-blind Phase (18 Months) | COMPLETED | DB: Placebo | 284 | |
| Double-blind Phase (18 Months) | COMPLETED | DB: OCA 10 Milligrams (mg) | 274 | |
| Double-blind Phase (18 Months) | COMPLETED | DB: OCA 10 mg Titrated to OCA 25 mg | 275 | |
| Double-blind Phase (18 Months) | COMPLETED | OLE: OCA 10 mg (DB Placebo) | 0 |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 participant flowAdverse events1,398
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Nausea | Gastrointestinal disorders | DB: Placebo | 43 | 312 | |
| other | Nausea | Gastrointestinal disorders | DB: OCA 10 Milligrams (mg) | 32 | 295 | |
| other | Nausea | Gastrointestinal disorders | DB: OCA 10 mg Titrated to OCA 25 mg | 48 | 309 | |
| other | Nausea | Gastrointestinal disorders | OLE: OCA 10 mg (DB Placebo) | 19 | 231 | |
| other | Nausea | Gastrointestinal disorders | OLE: OCA 10 mg (DB OCA 10 mg) | 28 | 221 | |
| other | Nausea | Gastrointestinal disorders | OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) | 39 | 207 | |
| other | Constipation | Gastrointestinal disorders | DB: Placebo | 20 | 312 | |
| other | Constipation | Gastrointestinal disorders | DB: OCA 10 Milligrams (mg) | 28 | 295 | |
| other | Constipation | Gastrointestinal disorders | DB: OCA 10 mg Titrated to OCA 25 mg | 33 | 309 | |
| other | Constipation | Gastrointestinal disorders | OLE: OCA 10 mg (DB Placebo) | 12 | 231 |
1 to 10 of 1,398Source ClinicalTrials.gov
See all 1,398 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB: Placebo | 4 | 312 |
| Total, all-cause mortality | DB: OCA 10 Milligrams (mg) | 3 | 295 |
| Total, all-cause mortality | DB: OCA 10 mg Titrated to OCA 25 mg | 3 | 309 |
| Total, all-cause mortality | OLE: OCA 10 mg (DB Placebo) | 1 | 231 |
| Total, all-cause mortality | OLE: OCA 10 mg (DB OCA 10 mg) | 0 | 221 |
| Total, all-cause mortality | OLE: Titrated to OCA 25 mg (DB OCA 10 mg Titrated to OCA 25 mg) | 1 | 207 |
| Total, other adverse events | DB: Placebo | 290 | 312 |
| Total, other adverse events | DB: OCA 10 Milligrams (mg) | 276 | 295 |
| Total, other adverse events | DB: OCA 10 mg Titrated to OCA 25 mg | 291 | 309 |
| Total, other adverse events | OLE: OCA 10 mg (DB Placebo) | 199 | 231 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03439254
GET https://conductscience.org/api/trials/v1/NCT03439254/results?page=2