Purpose

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

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo + K-877 (Group A)
Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks
  • Drug: K-808 (Dose A)
    Administered orally once daily
    Other names:
    • Pemafibrate
  • Drug: Placebo
    Administered orally once daily
Placebo Comparator
Placebo + K-877 (Group B)
Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks
  • Drug: K-808 (Dose B)
    Administered orally once daily
    Other names:
    • Pemafibrate
  • Drug: Placebo
    Administered orally once daily
Experimental
K-808 Group A
K-808 (Dose A) for 64 Weeks
  • Drug: K-808 (Dose A)
    Administered orally once daily
    Other names:
    • Pemafibrate
Experimental
K-808 Group B
K-808 (Dose B) for 64 Weeks
  • Drug: K-808 (Dose B)
    Administered orally once daily
    Other names:
    • Pemafibrate

Recruiting Locations

Vanderbilt Digestive Disease Center
Nashville, Tennessee 37232

More Details

Status
Recruiting
Sponsor
Kowa Research Institute, Inc.

Study Contact

Director, Clinical Operations
919-433-1600
Clinical@KowaUS.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.