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NCT Number: NCT06247735

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.

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

(G.I,R,I) GI Research Institute, Vancouver, British Columbia, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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
  • Participant meets any other exclusion criteria outlined in the Clinical Study Protocol.

Treatment and study plan

K-808 (Dose A)

Drug

Administered orally once daily

Other names: Pemafibrate

K-808 (Dose B)

Drug

Administered orally once daily

Other names: Pemafibrate

Placebo

Drug

Administered orally once daily

Primary outcomes

  1. Percent change from baseline in serum alkaline phosphatase (ALP)

    Time frame: Baseline to Week 12

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

Secondary outcomes

  1. Achievement of normalization of ALP level

    Time frame: Baseline to Week 12

    ALP ≤1 × upper limit of normal (ULN)

  2. Achievement of target levels of ALP and total bilirubin (TB)

    Time frame: Baseline to Weeks 12 and 64

    After two doses of K-808

  3. Change from baseline in liver function parameters

    Time frame: Baseline to Weeks 12 and 64

    liver function test results including ALP, total and conjugated bilirubin, albumin, international normalized ratio [INR], γ-GT, ALT, AST, albumin, platelets count

  4. Change from baseline in GLOBE risk score

    Time frame: Baseline to Weeks 12 and 64

    calculated by GLOBE scoring system which is calculated based on serum values of bilirubin, ALP, albumin and platelet count.

  5. Change from baseline in UK-PBC score

    Time frame: Baseline to Weeks 12 and 64

    PBC risk score developed by United Kingdom (UK)-PBC Consortium is a scoring system and the calculation is based on laboratory test measurements and upper limits of normal (ULN) for the total bilirubin (BIL12); alanine transaminase or aspartate transaminase (TA12), and alkaline phosphatase (ALP12) after at least 12 months of UDCA, and the laboratory test measurements and lower limits of normal (LLN) for the serum albumin and platelet count in the same timeframe. A high number is indicative of a worse score.

  6. Incidence of Treatment Emergent Adverse Events (TEAEs)

    Time frame: Baseline to Week 68

    Coded using the most recent version of Medical Dictionary of Regulatory Activities (MedDRA).

Sponsors and collaborators

Lead sponsor

Kowa Research Institute, Inc.

Industry

Registry information

Official study title

A Phase 2, Randomized, Placebo-controlled, Parallel Group, Multicenter 12-week Study With a 52-week Extension to Evaluate the Efficacy and Safety of Two Doses of K-808 (Pemafibrate) in Subjects With Primary Biliary Cholangitis With Inadequate Response to Ursodeoxycholic Acid and/or Obeticholic Acid Treatment

Important dates

Study start
2024
Primary completion
2025
Study completion
2026
First posted
Feb 8, 2024
Registry last updated
Jun 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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