Study to Evaluate the Efficacy and Safety of K-808 (Pemafibrate) in Participants With Primary Biliary Cholangitis (PBC) With Inadequate Response to Ursodeoxycholic Acid (UDCA) and/or Obeticholic Acid (OCA) Treatment.

NCT ID: NCT06247735

Last Updated: 2025-09-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-07

Study Completion Date

2026-06-30

Brief Summary

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Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.

Detailed Description

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Conditions

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Primary Biliary Cholangitis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo + K-877 (Group A)

Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks

Group Type PLACEBO_COMPARATOR

K-808 (Dose A)

Intervention Type DRUG

Administered orally once daily

Placebo

Intervention Type DRUG

Administered orally once daily

Placebo + K-877 (Group B)

Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks

Group Type PLACEBO_COMPARATOR

K-808 (Dose B)

Intervention Type DRUG

Administered orally once daily

Placebo

Intervention Type DRUG

Administered orally once daily

K-808 Group A

K-808 (Dose A) for 64 Weeks

Group Type EXPERIMENTAL

K-808 (Dose A)

Intervention Type DRUG

Administered orally once daily

K-808 Group B

K-808 (Dose B) for 64 Weeks

Group Type EXPERIMENTAL

K-808 (Dose B)

Intervention Type DRUG

Administered orally once daily

Interventions

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K-808 (Dose A)

Administered orally once daily

Intervention Type DRUG

K-808 (Dose B)

Administered orally once daily

Intervention Type DRUG

Placebo

Administered orally once daily

Intervention Type DRUG

Other Intervention Names

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Pemafibrate Pemafibrate

Eligibility Criteria

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Inclusion Criteria

* Male or female participant who has a PBC diagnosis as demonstrated by the presence of ≥2 of the following three diagnostic criteria:

* History of ALP above ULN for at least 6 months
* History of positive antimitochondrial antibody (AMA) titer or positive PBC-specific antinuclear antibody (ANA) titer
* Historical liver biopsy consistent with PBC
* Participant has the following qualifying biochemistry value at Screening:

* ALP ≥1.5 × ULN
* Participant is ≥18 years of age at consent.
* Participant meets all other eligibility criteria outlined in the Clinical Study Protocol.

Exclusion Criteria

* Participant meets any one of the following criteria at Screening:

* ALP\>10 × ULN
* ALT or AST \>5 × ULN
* Hepatitis C treatment within 5 years of Screening, or active hepatitis C as defined by positive hepatitis C antibody with the presence of hepatitis C virus ribonucleic acid; subjects with active hepatitis B (HBV) infection (hepatitis B surface antigen \[HbsAg\] positive) will be excluded. A subject with resolved hepatitis A at least 3 months prior to the Screening Visit can be screened.
* Primary sclerosing cholangitis and secondary sclerosing cholangitis (eg, due to cholangiolithiasis, ischemia, telangiectasia, vasculitis, infectious diseases)
* Alcoholic liver disease
* History of definite autoimmune hepatitis or PBC/autoimmune hepatitis overlap, defined as both of the following: 1) IgG \>2 × ULN and/or positive anti-smooth muscle antibodies, 2) liver histology revealing moderate or severe periportal or periseptal inflammation
* Nonalcoholic steatohepatitis (NASH)
* Gilbert's Syndrome
* Alpha-1-antitrypsin deficiency, cystic fibrosis, Wilson's disease, hemochromatosis based on historically established diagnosis
* Drug-induced liver injury (DILI) as defined by typical exposure and history
* Known condition that involves bile duct obstruction or cholestasis other than PBC, eg, vascular diseases (eg, Budd-Chiari syndrome, sinusoidal obstruction syndrome, congestive hepatopathy), congenital conditions (ductal plate malformations, Caroli syndrome, congenital liver fibrosis), idiopathic ductopenia
* Hepatocellular carcinoma
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kowa Research Institute, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Andre Belous, MD, PhD

Role: STUDY_DIRECTOR

Kowa Pharma Development Co.

Locations

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UA Thomas D. Boyer Liver Institute

Tucson, Arizona, United States

Site Status

Southern California Research Center - Coronado

Coronado, California, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

Velocity Clinical Research

Santa Ana, California, United States

Site Status

Peak Gastroenterology Associates Colorado Springs

Colorado Springs, Colorado, United States

Site Status

University of Florida Hepatology Research at CTRB

Gainesville, Florida, United States

Site Status

Florida Research Institute

Lakewood Rch, Florida, United States

Site Status

University of Miami Leonard M. Miller School of Medicine

Miami, Florida, United States

Site Status

Springfield Clinic

Springfield, Illinois, United States

Site Status

Mercy Medical Center - Mcauley Plaza

Baltimore, Maryland, United States

Site Status

CommonSpirit Health Research Institute

Omaha, Nebraska, United States

Site Status

New York University Hepatology Associates

New York, New York, United States

Site Status

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

Einstein Medical Center

Philadelphia, Pennsylvania, United States

Site Status

Rapid City Medical Center

Rapid City, South Dakota, United States

Site Status

Vanderbilt Digestive Disease Center

Nashville, Tennessee, United States

Site Status

Pioneer Research Solutions

Houston, Texas, United States

Site Status

UVA Health - University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

Gastrointestinal and Liver Specialists of Tidewater - Digestive and Liver Disease Specialists

Norfolk, Virginia, United States

Site Status

Liver Institute Northwest

Seattle, Washington, United States

Site Status

(G.I,R,I) GI Research Institute

Vancouver, British Columbia, Canada

Site Status

Office of Dr. Gauthier

North Bay, Ontario, Canada

Site Status

Toronto General Hospital

Toronto, Ontario, Canada

Site Status

Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CRCHUM)

Montreal, Quebec, Canada

Site Status

306

Fukui, , Japan

Site Status

313

Fukuoka, , Japan

Site Status

305

Hamamatsu, , Japan

Site Status

308

Hirakata, , Japan

Site Status

309

Hiroshima, , Japan

Site Status

304

Isehara, , Japan

Site Status

303

Itabashi-ku, , Japan

Site Status

312

Kanazawa, , Japan

Site Status

307

Matsumoto, , Japan

Site Status

311

Ōmura, , Japan

Site Status

302

Sapporo, , Japan

Site Status

Teine Keijinkai Hospital

Sapporo, , Japan

Site Status

314

Yokohama, , Japan

Site Status

Countries

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United States Canada Japan

Other Identifiers

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K-808-2.01

Identifier Type: -

Identifier Source: org_study_id

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