A Phase 3, Open-Label, Long-Term Safety Extension Study Evaluating the Safety and Tolerability of the Fixed-Dose Combination of Obeticholic Acid and Bezafibrate in Subjects with Primary Biliary Cholangitis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Intercept Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

3 Apr 2025 → 21 Oct 2025

Decision date (initial)

2025-02-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Intercept Pharmaceuticals, Inc., USA

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Safety, Pharmacokinetic, Pharmacodynamic

To assess the long-term safety and tolerability of the OCA + BZF FDC tablet (OCA 5 mg + BZF 400 mg SR) in subjects with PBC:
• Biochemical disease markers, including ALP, GGT, ALT, AST, and total and conjugated bilirubin.
• ALP <1.67 x ULN, total bilirubin ≤ULN, and ALP decrease of ≥15% from baseline.
• Noninvasive assessments of liver fibrosis (TE, ELF score).
• Disease severity scores (GLOBE, UK-PBC, and MELD).
Please refer to the Protocol for details

Secondary objectives 1

1. To assess the effects of OCA + BZF FDC tablet on collagen formation and degradation, PBC-40, NRS, FIS, PK, PD, biomarkers of bile acid synthesis and homeostasis, and the occurrence of all-cause mortality and liver-related clinical outcomes. Please refer to the Protocol for detailed secondary objectives.

Conditions and MedDRA coding

Primary Biliary Cholangitis (PBC)

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Key Target Population: All subjects with PBC who participated and are actively taking investigational product in Study 747-213 or Study 747-214 (or other Sponsor studies) are included. Please refer to the Protocol for detailed Principal inclusion criteria.

Exclusion criteria 1

1. Key Exclusion include: if they have a history or presence in the last 30 days, before rolling over to this study, of other concomitant liver diseases, varices, ascites/hepatic hydrothorax, spontaneous bacterial peritonitis, hepatic encephalopathy, or jaundice. Clinical complication of PBC including prior liver transplantation. Biochemical evidence of hepatic impairment, decompensation, or injury. Medical conditions that may cause non-hepatic increases in ALP. Presence of any other disease or condition that interferes with the absorption, distribution, metabolism, or excretion of drugs including bile salt metabolism in the intestine. History of cholelithiasis or choledocholithiasis unless documented cholecystectomy, chronic pancreatitis/recurrent acute pancreatitis, drug-induced myopathy, chronic kidney disease or undergoing dialysis, HIV, clinically concerning cardiac arrhythmias, severe pruritus, or hypersensitivity to OCA, BZF. Please refer to the Protocol for detailed Principal exclusion criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. 1. Response rate of ≥40% reduction from baseline and normalization rates of ALP;
2. 2. normalization rates of GGT, ALT, AST, total and conjugated bilirubin;
3. 3. change from baseline in GGT, ALT, ALP, AST, total and conjugated bilirubin, GLOBE scores, UK-PBC scores, MELD scores, and noninvasive markers of liver fibrosis, including liver stiffness measured by TE and ELF score;
4. 4. and percentage of subjects with ALP <1.67 x ULN, total bilirubin ≤ULN, and ALP decrease of ≥15% from baseline.
5. 5. Please refer to the Protocol for detailed Primary end point.

Secondary endpoints 3

1. 1. Exploratory endpoints include change from baseline in NRS, PBC-40, FIS, and APRI. In addition, time to first occurrence of any of the following:
2. 2. death (all-cause), liver transplant, MELD score ≥15, hospitalization new onset or recurrence of variceal bleed, hepatic encephalopathy, spontaneous bacterial peritonitis, or hepatocellular carcinoma , portal hypertension syndromes, portal hypertension (clinically evident ascites or endoscopic evidence without bleeding), splenomegaly, and time to first occurrence of each individual component of the composite event endpoint.
3. 3. Please refer to the Protocol for detailed Secondary end point.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Intercept Pharmaceuticals Inc.

Sponsor organisation

Intercept Pharmaceuticals Inc.

Address

305 Madison Avenue

City

Morristown

Postcode

07960-6117

Country

United States

Scientific contact point

Organisation

Intercept Pharmaceuticals Inc.

Contact name

Clinical Reaserch

Public contact point

Organisation

Intercept Pharmaceuticals Inc.

Contact name

Clinical Operations

Third parties 8

Locations

12 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 67 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications