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.
Purpose
Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.
Condition
- Primary Biliary Cholangitis
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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator Placebo + K-877 (Group A) |
Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks |
|
|
Placebo Comparator Placebo + K-877 (Group B) |
Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks |
|
|
Experimental K-808 Group A |
K-808 (Dose A) for 64 Weeks |
|
|
Experimental K-808 Group B |
K-808 (Dose B) for 64 Weeks |
|
Recruiting Locations
Vanderbilt Digestive Disease Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Kowa Research Institute, Inc.