Clinical trial 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.
Official 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Primary Biliary Cholangitis
Lead sponsor: Kowa Research Institute, Inc.
Sponsor class: INDUSTRY
First posted: 2024-02-08
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
measure: Percent change from baseline in serum alkaline phosphatase (ALP)
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Achievement of normalization of ALP level
timeFrame: Baseline to Week 12
measure: Achievement of target levels of ALP and total bilirubin (TB)
timeFrame: Baseline to Weeks 12 and 64
measure: Change from baseline in liver function parameters
timeFrame: Baseline to Weeks 12 and 64
measure: Change from baseline in GLOBE risk score
timeFrame: Baseline to Weeks 12 and 64
measure: Change from baseline in UK-PBC score
timeFrame: Baseline to Weeks 12 and 64
measure: Incidence of Treatment Emergent Adverse Events (TEAEs)
timeFrame: Baseline to Week 68
Other outcomes:
Locations:
city: Tucson
country: United States
facility: UA Thomas D. Boyer Liver Institute
state: Arizona
city: Coronado
country: United States
facility: Southern California Research Center - Coronado
state: California
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Santa Ana
country: United States
facility: Velocity Clinical Research
state: California
city: Colorado Springs
country: United States
facility: Peak Gastroenterology Associates Colorado Springs
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Hepatology Research at CTRB
state: Florida
city: Lakewood Ranch
country: United States
facility: Florida Research Institute
state: Florida
city: Miami
country: United States
facility: University of Miami Leonard M. Miller School of Medicine
state: Florida
city: Springfield
country: United States
facility: Springfield Clinic
state: Illinois
city: Baltimore
country: United States
facility: Mercy Medical Center - Mcauley Plaza
state: Maryland
city: Omaha
country: United States
facility: CommonSpirit Health Research Institute
state: Nebraska
city: New York
country: United States
facility: New York University Hepatology Associates
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest University Baptist Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Philadelphia
country: United States
facility: Einstein Medical Center
state: Pennsylvania
city: Rapid City
country: United States
facility: Rapid City Medical Center
state: South Dakota
city: Nashville
country: United States
facility: Vanderbilt Digestive Disease Center
state: Tennessee
city: Houston
country: United States
facility: Pioneer Research Solutions
state: Texas
city: Charlottesville
country: United States
facility: UVA Health - University of Virginia Health System
state: Virginia
city: Norfolk
country: United States
facility: Gastrointestinal and Liver Specialists of Tidewater - Digestive and Liver Disease Specialists
state: Virginia
city: Seattle
country: United States
facility: Liver Institute Northwest
state: Washington
city: Vancouver
country: Canada
facility: (G.I,R,I) GI Research Institute
state: British Columbia
city: North Bay
country: Canada
facility: Office of Dr. Gauthier
state: Ontario
city: Toronto
country: Canada
facility: Toronto General Hospital
state: Ontario
city: Montreal
country: Canada
facility: Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CRCHUM)
state: Quebec
city: Fukui
country: Japan
facility: 306
city: Fukuoka
country: Japan
facility: 313
city: Hamamatsu
country: Japan
facility: 305
city: Hirakata
country: Japan
facility: 308
city: Hiroshima
country: Japan
facility: 309
city: Isehara
country: Japan
facility: 304
city: Itabashi-ku
country: Japan
facility: 303
city: Kanazawa
country: Japan
facility: 312
city: Matsumoto
country: Japan
facility: 307
city: Omura
country: Japan
facility: 311
city: Sapporo
country: Japan
facility: 302
city: Sapporo
country: Japan
facility: Teine Keijinkai Hospital
city: Yokohama
country: Japan
facility: 314
Study officials:
affiliation: Kowa Pharma Development Co.
name: Andre Belous, MD, PhD
role: STUDY_DIRECTOR
References: