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.

NCT06247735 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: K-808-2.01
• Study Type: interventional
• Target: 46
• Locations: 3 countries
• Sites: 38

Brief Summary

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

Conditions

Primary Biliary Cholangitis

Outcome Measures

Primary Outcomes (1)

• Percent change from baseline in serum alkaline phosphatase (ALP)

  Two doses of K-808 compared to placebo after 12 weeks of treatment

  Baseline to Week 12

Secondary Outcomes (6)

• Achievement of normalization of ALP level

  Baseline to Week 12

• Achievement of target levels of ALP and total bilirubin (TB)

  Baseline to Weeks 12 and 64

• Change from baseline in liver function parameters

  Baseline to Weeks 12 and 64

• Change from baseline in GLOBE risk score

  Baseline to Weeks 12 and 64

• Change from baseline in UK-PBC score

  Baseline to Weeks 12 and 64

Study Arms (4)

Placebo + K-877 (Group A)

PLACEBO COMPARATOR

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

Drug: K-808 (Dose A); Drug: Placebo

Placebo + K-877 (Group B)

PLACEBO COMPARATOR

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

Drug: K-808 (Dose B); Drug: Placebo

K-808 Group A

EXPERIMENTAL

K-808 (Dose A) for 64 Weeks

Drug: K-808 (Dose A)

K-808 Group B

EXPERIMENTAL

K-808 (Dose B) for 64 Weeks

Drug: K-808 (Dose B)

Interventions

K-808 (Dose A) DRUG

Administered orally once daily

Also known as: Pemafibrate

K-808 Group A; Placebo + K-877 (Group A)

K-808 (Dose B) DRUG

Administered orally once daily

Also known as: Pemafibrate

K-808 Group B; Placebo + K-877 (Group B)

Placebo DRUG

Administered orally once daily

Placebo + K-877 (Group A); Placebo + K-877 (Group B)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Kowa Research Institute, Inc.: lead

Study Sites (38)

UA Thomas D. Boyer Liver Institute

Tucson, Arizona, 85724, United States

Location

Southern California Research Center - Coronado

Coronado, California, 92118, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Velocity Clinical Research

Santa Ana, California, 92704, United States

Location

Peak Gastroenterology Associates Colorado Springs

Colorado Springs, Colorado, 80921, United States

Location

University of Florida Hepatology Research at CTRB

Gainesville, Florida, 32610, United States

Location

Florida Research Institute

Lakewood Rch, Florida, 34211, United States

Location

University of Miami Leonard M. Miller School of Medicine

Miami, Florida, 33136, United States

Location

Springfield Clinic

Springfield, Illinois, 62702, United States

Location

Mercy Medical Center - Mcauley Plaza

Baltimore, Maryland, 21202, United States

Location

CommonSpirit Health Research Institute

Omaha, Nebraska, 68124, United States

Location

New York University Hepatology Associates

New York, New York, 10016, United States

Location

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Einstein Medical Center

Philadelphia, Pennsylvania, 19141, United States

Location

Rapid City Medical Center

Rapid City, South Dakota, 57701, United States

Location

Vanderbilt Digestive Disease Center

Nashville, Tennessee, 37232, United States

Location

Pioneer Research Solutions

Houston, Texas, 77099, United States

Location

UVA Health - University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

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

Norfolk, Virginia, 23502, United States

Location

Liver Institute Northwest

Seattle, Washington, 98105, United States

Location

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

Vancouver, British Columbia, V6Z 2K5, Canada

Location

Office of Dr. Gauthier

North Bay, Ontario, P1B 2H3, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

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

Montreal, Quebec, H2X 0A9, Canada

Location

306

Fukui, Japan

Location

313

Fukuoka, Japan

Location

305

Hamamatsu, Japan

Location

308

Hirakata, Japan

Location

309

Hiroshima, Japan

Location

304

Isehara, Japan

Location

303

Itabashi-ku, Japan

Location

312

Kanazawa, Japan

Location

307

Matsumoto, Japan

Location

311

Ōmura, Japan

Location

302

Sapporo, Japan

Location

Teine Keijinkai Hospital

Sapporo, Japan

Location

314

Yokohama, Japan

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

(R)-2-(3-((benzoxazol-2-yl-d4 (3-(4-methoxyphenoxy-d7)propyl)amino)methyl)phenoxy) butanoic acid

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Andre Belous, MD, PhD

  Kowa Pharma Development Co.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2024
• First Posted: February 8, 2024
• Study Start: February 7, 2024
• Primary Completion: June 26, 2025
• Study Completion (Estimated): June 1, 2026
• Last Updated: September 10, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CA (4); JP (13); US (21)