A Phase 3 interventional study of Obeticholic Acid and Placebo in Non Alcoholic Steatohepatitis (NASH), sponsored by Intercept Pharmaceuticals. Terminated at 346 sites in 21 countries. Open to participants aged 18 Years to 85 Years. Per ClinicalTrials.gov, last updated 2024-10-01.
Sponsored by Intercept Pharmaceuticals (part of Alfasigma S.p.A.) · Phase 3, Interventional, and Treatment
The primary objectives of this study are to evaluate the effect of Obeticholic Acid treatment compared to placebo on 1) histological improvement and 2) liver-related clinical outcomes in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis.
1,507 studies on the registry are indexed under Non-alcoholic Fatty Liver Disease; 303 are open to participants now.
This study's enrollment of 2,477 is above the median of 60 across 1,098 interventional studies indexed under Non-alcoholic Fatty Liver Disease.
Browse Non-alcoholic Fatty Liver Disease studies →Intercept Pharmaceuticals is the lead sponsor of 22 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 8 (89%) have results posted.
First submitted before Intercept Pharmaceuticals became part of Alfasigma S.p.A..
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Histologic evidence of fibrosis stage 2 or stage 3 as defined by the NASH CRN scoring of fibrosis, or
Histologic evidence of fibrosis stage 1a or stage 1b if accompanied by ≥1 of the following risk factors:
Exclusion Criteria:
10 mg Obeticholic Acid daily for the remainder of the study
Drug: Obeticholic Acid
25 mg Obeticholic Acid daily for the remainder of the study
Drug: Obeticholic Acid
One tablet daily for the remainder of the study
Drug: Placebo
Time to the First Adjudicated Event for Clinical Outcome Composite Endpoint: Percentage of Participants With an Event
All potential liver-related clinical outcomes that occurred after administration of first dose of investigational product were reviewed and adjudicated by blinded, independent Hepatic Outcomes Committee(HOC). Adjudicated results were used to assess effect of OCA, compared to placebo in conjunction with established local standard of care, on clinical outcomes in participants with NASH as measured by time to first occurrence of any of following adjudicated events, derived as a composite event endpoint of death(all-cause), liver transplant, Model of End-stage Liver Disease(MELD)≥15 Score, hospitalization(defined by a stay of 24 hours or greater) for onset of: variceal bleed, hepatic encephalopathy(defined by a West Haven score of ≥2), and spontaneous bacterial peritonitis(confirmed by diagnostic paracentesis), ascites secondary to cirrhosis requiring medical intervention, and histological progression to Cirrhosis. Clinical events distribution was estimated using Kaplan-Meier methodology.
Time frame: Up to 7 years
Percentage of Responders With Improvement of Fibrosis by at Least One Stage With no Worsening of NASH Using Consensus Read Method of Scheduled Liver Biopsies
Fibrosis stage was evaluated by NASH Clinical Research Network (CRN) Fibrosis Staging System as follows: Stage 0: No fibrosis, Stage 1: Perisinusoidal/periportal fibrosis, Stage 1A: Mild, zone 3, perisinusoidal fibrosis, Stage 1B: Moderate, zone 3, perisinusoidal fibrosis, Stage 1C: Portal/periportal fibrosis, Stage 2: Perisinusoidal and portal/periportal fibrosis, Stage 3: Bridging fibrosis and Stage 4: Cirrhosis. No worsening of NASH was defined as no worsening of hepatocellular ballooning, no worsening of lobular inflammation, and no worsening of steatosis. Responders are defined as participants who did not discontinue treatment due to Adverse event (AE) or did not die and had evaluable post-Baseline biopsy assessment. Mantel-Haenszel method is used to construct the confidence intervals.
Time frame: Up to 7 years
Percentage of Participants Who Showed Improvement in Fibrosis by at Least 1 Stage and/or Resolution of NASH Without Worsening of Either Using Consensus Read Method
Fibrosis stage was evaluated by NASH Clinical Research Network(CRN) Fibrosis Staging System as follows: Stage 0:No fibrosis, Stage 1:Perisinusoidal/periportal fibrosis, Stage 1A:Mild, zone 3, perisinusoidal fibrosis, Stage 1B:Moderate, zone3, perisinusoidal fibrosis, Stage 1C:Portal/periportal fibrosis, Stage 2:Perisinusoidal and portal/periportal fibrosis, Stage 3:Bridging fibrosis and Stage 4:Cirrhosis. Resolution of NASH (Nonalcoholic Steatohepatitis) is defined as absence of fatty liver disease, or presence of simple or isolated steatosis without steatohepatitis, with a NAS(NAFLD Activity Score) of 0 for ballooning and 0 to 1 for inflammation. NAS ranges from 0-12; 0: no features of fatty liver disease and 12: highest degree of fatty liver disease. Higher signify worse symptoms. Responders are who did not discontinue treatment due to Adverse event(AE) or did not die and had evaluable post-Baseline biopsy assessment. Mantel-Haenszel method is used to construct confidence intervals.
Time frame: Up to 7 years
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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. A TEAE was defined as any AE that either newly appeared, increased in frequency, or worsened in severity following treatment up to 30 days from last dose of permanent investigational product discontinuation. Serious AE (SAE) was defined as any AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event.
Time frame: Up to 7 years
This was a phase 3, double-blind, randomized, long-term, placebo-controlled, multicenter study evaluating the safety and efficacy of Obeticholic Acid (OCA) in Participants with Nonalcoholic Steatohepatitis (NASH).
| Milestone | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| Started | 825 | 827 | 825 |
| Completed | 0 | 0 | 0 |
| Not completed | 825 | 827 | 825 |
| Withdrew: Withdrawal by subject | 149 | 154 | 152 |
| Withdrew: Death | 11 | 15 | 12 |
| Withdrew: Adverse event | 22 | 56 | 36 |
| Withdrew: Study terminated by sponsor | 549 | 509 | 528 |
| Withdrew: Site closure | 14 | 5 | 7 |
| Withdrew: Non-compliance with study drug | 4 | 2 | 0 |
| Withdrew: Protocol violation | 2 | 1 | 2 |
| Withdrew: Physician decision | 11 | 18 | 11 |
| Withdrew: Lost to follow-up | 43 | 50 | 57 |
| Withdrew: Other | 19 | 16 | 20 |
| Withdrew: Missing | 1 | 1 | 0 |
All potential liver-related clinical outcomes that occurred after administration of first dose of investigational product were reviewed and adjudicated by blinded, independent Hepatic Outcomes Committee(HOC). Adjudicated results were used to assess effect of OCA, compared to placebo in conjunction with established local standard of care, on clinical outcomes in participants with NASH as measured by time to first occurrence of any of following adjudicated events, derived as a composite event endpoint of death(all-cause), liver transplant, Model of End-stage Liver Disease(MELD)≥15 Score, hospitalization(defined by a stay of 24 hours or greater) for onset of: variceal bleed, hepatic encephalopathy(defined by a West Haven score of ≥2), and spontaneous bacterial peritonitis(confirmed by diagnostic paracentesis), ascites secondary to cirrhosis requiring medical intervention, and histological progression to Cirrhosis. Clinical events distribution was estimated using Kaplan-Meier methodology.
| Percentage of participants | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| Time to the First Adjudicated Event for Clinical Outcome Composite Endpoint: Percentage of Participants With an Event | 15.8 (13.2 to 18.6) | 14.7 (12.2 to 17.4) | 18.8 (16.0 to 21.9) |
Fibrosis stage was evaluated by NASH Clinical Research Network (CRN) Fibrosis Staging System as follows: Stage 0: No fibrosis, Stage 1: Perisinusoidal/periportal fibrosis, Stage 1A: Mild, zone 3, perisinusoidal fibrosis, Stage 1B: Moderate, zone 3, perisinusoidal fibrosis, Stage 1C: Portal/periportal fibrosis, Stage 2: Perisinusoidal and portal/periportal fibrosis, Stage 3: Bridging fibrosis and Stage 4: Cirrhosis. No worsening of NASH was defined as no worsening of hepatocellular ballooning, no worsening of lobular inflammation, and no worsening of steatosis. Responders are defined as participants who did not discontinue treatment due to Adverse event (AE) or did not die and had evaluable post-Baseline biopsy assessment. Mantel-Haenszel method is used to construct the confidence intervals.
| Percentage of participants | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| Percentage of Responders With Improvement of Fibrosis by at Least One Stage With no Worsening of NASH Using Consensus Read Method of Scheduled Liver Biopsies | 17.1 | 19.5 | 10.0 |
Fibrosis stage was evaluated by NASH Clinical Research Network(CRN) Fibrosis Staging System as follows: Stage 0:No fibrosis, Stage 1:Perisinusoidal/periportal fibrosis, Stage 1A:Mild, zone 3, perisinusoidal fibrosis, Stage 1B:Moderate, zone3, perisinusoidal fibrosis, Stage 1C:Portal/periportal fibrosis, Stage 2:Perisinusoidal and portal/periportal fibrosis, Stage 3:Bridging fibrosis and Stage 4:Cirrhosis. Resolution of NASH (Nonalcoholic Steatohepatitis) is defined as absence of fatty liver disease, or presence of simple or isolated steatosis without steatohepatitis, with a NAS(NAFLD Activity Score) of 0 for ballooning and 0 to 1 for inflammation. NAS ranges from 0-12; 0: no features of fatty liver disease and 12: highest degree of fatty liver disease. Higher signify worse symptoms. Responders are who did not discontinue treatment due to Adverse event(AE) or did not die and had evaluable post-Baseline biopsy assessment. Mantel-Haenszel method is used to construct confidence intervals.
| Percentage of participants | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| Percentage of Participants Who Showed Improvement in Fibrosis by at Least 1 Stage and/or Resolution of NASH Without Worsening of Either Using Consensus Read Method | 18.5 | 20.8 | 11.8 |
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. A TEAE was defined as any AE that either newly appeared, increased in frequency, or worsened in severity following treatment up to 30 days from last dose of permanent investigational product discontinuation. Serious AE (SAE) was defined as any AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event.
| Participants | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| TEAEs | 798 | 813 | 781 |
| SAEs | 273 | 276 | 248 |
Collected over Up to 7 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| OCA 10 Milligrams (mg) | 14/825 (1.7%) | 273/825 (33.1%) | 798/825 (96.7%) |
| OCA 25 mg | 19/827 (2.3%) | 276/827 (33.4%) | 813/827 (98.3%) |
| Placebo | 14/825 (1.7%) | 248/825 (30.1%) | 781/825 (94.7%) |
| Event | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| Acute kidney injuryRenal and urinary disorders | 6/825 | 13/827 | 4/825 |
| Pneumonia viralInfections and infestations | 9/825 | 10/827 | 4/825 |
| Cholecystitis acuteHepatobiliary disorders | 2/825 | 9/827 | 1/825 |
| PneumoniaInfections and infestations | 5/825 | 9/827 | 7/825 |
| Atrial fibrillationCardiac disorders | 8/825 | 5/827 | 4/825 |
| Hepatocellular carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 8/825 | 6/827 | 3/825 |
| Chest painGeneral disorders | 5/825 | 8/827 | 6/825 |
| CholelithiasisHepatobiliary disorders | 4/825 | 8/827 | 4/825 |
| OsteoarthritisMusculoskeletal and connective tissue disorders | 6/825 | 8/827 | 6/825 |
| Coronary artery diseaseCardiac disorders | 5/825 | 4/827 | 7/825 |
| Event | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo |
|---|---|---|---|
| PruritusSkin and subcutaneous tissue disorders | 294/825 | 470/827 | 210/825 |
| Low-density lipoprotein increasedInvestigations | 175/825 | 183/827 | 92/825 |
| NauseaGastrointestinal disorders | 148/825 | 165/827 | 150/825 |
| Urinary tract infectionInfections and infestations | 147/825 | 151/827 | 133/825 |
| FatigueGeneral disorders | 148/825 | 135/827 | 138/825 |
| DiarrhoeaGastrointestinal disorders | 112/825 | 108/827 | 145/825 |
| Abdominal painGastrointestinal disorders | 139/825 | 135/827 | 132/825 |
| Back painMusculoskeletal and connective tissue disorders | 136/825 | 105/827 | 120/825 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 117/825 | 132/827 | 134/825 |
| ConstipationGastrointestinal disorders | 115/825 | 128/827 | 92/825 |
| Age, Continuous(Years) | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo | Total |
|---|---|---|---|---|
| Mean | 55.3 ± 10.76 | 55.3 ± 11.71 | 54.4 ± 11.21 | 55.3 ± 11.24 |
| Sex: Female, Male(Participants) | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo | Total |
|---|---|---|---|---|
| Female | 475 | 494 | 478 | 1447 |
| Male | 350 | 333 | 347 | 1030 |
| Ethnicity (NIH/OMB)(Participants) | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo | Total |
|---|---|---|---|---|
| Hispanic or Latino | 129 | 149 | 147 | 425 |
| Not Hispanic or Latino | 620 | 594 | 592 | 1806 |
| Unknown or Not Reported | 76 | 84 | 86 | 246 |
| Race (NIH/OMB)(Participants) | OCA 10 Milligrams (mg) | OCA 25 mg | Placebo | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 5 | 8 | 6 | 19 |
| Asian | 47 | 43 | 29 | 119 |
| Native Hawaiian or Other Pacific Islander | 4 | 4 | 2 | 10 |
| Black or African American | 14 | 20 | 12 | 46 |
| White | 679 | 674 | 685 | 2038 |
| More than one race | 4 | 3 | 7 | 14 |
| Unknown or Not Reported | 72 | 75 | 84 | 231 |
Showing the first 100 of 346 sites across 21 countries.
Documents are hosted by the registry — open the source record to download them.
Ran from Sep 2015 to primary completion in Sep 2023, 95 months.
Results were first posted in Sep 2024, 11 months after primary completion.
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Studies of the drug tested here: Obeticholic acid
Non-alcoholic Fatty Liver Disease →