Study to determine if the investigational drug obeticholic acid (also known as OCA) in combination with the investigational drug bezafibrate (BZF), has an effect on Primary Biliary Cholangitis (also known as PBC).

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

Phenomena and Processes [G] - Digestive System and Oral Physiological Phenomena [G10]

Trial duration

9 Mar 2023 → 1 Sep 2025

Decision date (initial)

2024-07-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2024-513584-77-00

EudraCT number

2022-001241-20

ClinicalTrials.gov

NCT05239468

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenetic, Pharmacokinetic, Therapy, Efficacy, Pharmacodynamic, Safety, Dose response, Pharmacogenomic

The primary objective is to assess the effects of the combination of OCA and BZF on alkaline phosphatase (ALP) in comparison to BZF alone in subjects with primary biliary cholangitis (PBC)

Secondary objectives 3

1. To assess the effects of the combination of OCA plus BZF versus BZF alone in subjects with PBC on biochemical disease markers, including ALP, gamma-glutamyl transferase (GGT), alanine aminotransferase (ALT), aspartate aminotransferase (AST), total and conjugated bilirubin, and lipid panel
2. To assess the effects of the combination of OCA plus BZF versus BZF alone in subjects with PBC on biomarkers of bile acid synthesis and homeostasis, including 7α-hydroxy-4 cholesten-3-one (C4) and bile acids
3. To assess the effects of the combination of OCA plus BZF versus BZF alone in subjects with PBC onsafety and tolerability

Conditions and MedDRA coding

Primary biliary cholangitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. A define or probable diagnosis of PBC
2. Qualifying ALP and/or bilirubin liver biochemistry values
3. Taking ursodeoxycholic acid (UDCA) for at least 12 months or no UDCA for 3 months before Day 1

Exclusion criteria 7

1. History or presence of other concomitant liver diseases
2. Presence of clinical complications of PBC
3. History or presence of decompensating events
4. Current or history of gallbladder disease
5. If female, known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating.
6. Treatment with commercially available OCA or participation in aprevious study involving OCA within 3 months before Screening
7. Treatment with commercially available fibrates, or participation in a previous study involving fibrate within 3 months before Screening

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary efficacy endpoint is the change in ALP from baseline to Week 12 in the DB Phase

Secondary endpoints 5

1. Safety and tolerability
2. The response rates of ≥10%, ≥20%, ≥30%, and ≥40% reduction from baseline at Week 12, and normalization rates of ALP at Week 12
3. Normalization rates at Week 12 of GGT, ALT, AST, total and conjugated bilirubin, and lipid panel
4. Change from baseline to Week 12 in GGT, ALT, AST, ALP and total and conjugated bilirubin, and lipid panel
5. Change from baseline to Week 12 in 7α-hydroxy-4-cholesten-3-one (C4) and bile acids

Investigational products

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

Test 1

Comparator 2

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 Research

Public contact point

Organisation

Intercept Pharmaceuticals Inc.

Contact name

Clinical Operations

Third parties 5

Locations

1 EU/EEA country · 1 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 6 files

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

Application history

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