Primary Biliary CholangitisPhase 2NCT06247735
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.
A Phase 2 study testing K-808 (Dose A) and K-808 (Dose B) in people with Primary Biliary Cholangitis, run by Kowa Research Institute, Inc. at 38 sites in 3 countries.
Trial summary
Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- K-808 (Dose A) and K-808 (Dose B)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 46 participants
- Where
- 38 sitesin 3 countries
- Study dates
- 7 Feb 2024to 23 Jul 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 14Show fewer
- 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.
Trial locations and contacts
38 sites in 3 countries.
38 sites in 3 countries
United States21 sites
Mercy Medical Center - Mcauley Plaza
Baltimore, Maryland, 21202
UVA Health - University of Virginia Health System
Charlottesville, Virginia, 22908
University of Cincinnati
Cincinnati, Ohio, 45267
Peak Gastroenterology Associates Colorado Springs
Colorado Springs, Colorado, 80921
Southern California Research Center - Coronado
Coronado, California, 92118
University of Florida Hepatology Research at CTRB
Gainesville, Florida, 32610
Pioneer Research Solutions
Houston, Texas, 77099
Florida Research Institute
Lakewood Rch, Florida, 34211
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Miami Leonard M. Miller School of Medicine
Miami, Florida, 33136
Vanderbilt Digestive Disease Center
Nashville, Tennessee, 37232
New York University Hepatology Associates
New York, New York, 10016
Gastrointestinal and Liver Specialists of Tidewater - Digestive and Liver Disease Specialists
Norfolk, Virginia, 23502
CommonSpirit Health Research Institute
Omaha, Nebraska, 68124
Einstein Medical Center
Philadelphia, Pennsylvania, 19141
Rapid City Medical Center
Rapid City, South Dakota, 57701
Velocity Clinical Research
Santa Ana, California, 92704
Liver Institute Northwest
Seattle, Washington, 98105
Springfield Clinic
Springfield, Illinois, 62702
UA Thomas D. Boyer Liver Institute
Tucson, Arizona, 85724
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, 27157
Japan13 sites
306
Fukui
313
Fukuoka
305
Hamamatsu
308
Hirakata
309
Hiroshima
304
Isehara
312
Kanazawa
307
Matsumoto
311
Ōmura
302
Sapporo
Teine Keijinkai Hospital
Sapporo
303
tabashi City
314
Yokohama
Canada4 sites
Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CRCHUM)
Montreal, Quebec, H2X 0A9
Office of Dr. Gauthier
North Bay, Ontario, P1B 2H3
Toronto General Hospital
Toronto, Ontario, M5G 2C4
(G.I,R,I) GI Research Institute
Vancouver, British Columbia, V6Z 2K5
About this study
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 to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.
- Conditions
- Primary Biliary Cholangitis
- Being tested
- K-808 (Dose A), K-808 (Dose B), Placebo
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
K-808 (Dose A)Drug
Also called Pemafibrate
Administered orally once daily
K-808 (Dose B)Drug
Also called Pemafibrate
Administered orally once daily
PlaceboDrug
Administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| Placebo + K-808 (Group A) | Placebo comparator | Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks Receives: Drug: K-808 (Dose A); Drug: Placebo |
| Placebo + K-808 (Group B) | Placebo comparator | Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks Receives: Drug: K-808 (Dose B); Drug: Placebo |
| K-808 Group A | Experimental | K-808 (Dose A) for 64 Weeks Receives: Drug: K-808 (Dose A) |
| K-808 Group B | Experimental | K-808 (Dose B) for 64 Weeks Receives: Drug: K-808 (Dose B) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change from baseline in serum alkaline phosphatase (ALP)
Two doses of K-808 compared to placebo after 12 weeks of treatment
Measured: Baseline to Week 12
Secondary outcomes (6)
Achievement of normalization of ALP level
ALP ≤1 × upper limit of normal (ULN)
Measured: Baseline to Week 12
Achievement of target levels of ALP and total bilirubin (TB)
After two doses of K-808
Measured: Baseline to Weeks 12 and 64
Change from baseline in liver function parameters
liver function test results including ALP, total and conjugated bilirubin, albumin, international normalized ratio [INR], γ-GT, ALT, AST, albumin, platelets count
Measured: Baseline to Weeks 12 and 64
Change from baseline in GLOBE risk score
calculated by GLOBE scoring system which is calculated based on serum values of bilirubin, ALP, albumin and platelet count.
Measured: Baseline to Weeks 12 and 64
Change from baseline in UK-PBC score
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.
Measured: Baseline to Weeks 12 and 64
Incidence of Treatment Emergent Adverse Events (TEAEs)
Coded using the most recent version of Medical Dictionary of Regulatory Activities (MedDRA).
Measured: Baseline to Week 68
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2024
- Study start7 Feb 2024
- Primary completion26 Jun 2025
- Study completion23 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kowa Research Institute, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andre Belous, MD, PhD, Study director, Kowa Pharma Development Co.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06247735
- Sponsor's protocol ID
- K-808-2.01
- Status last verified
- Oct 2026
- First submitted
- 22 Jan 2024
- Last updated
- 7 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06247735
- Acronym / sponsor ID
- K-808-2.01
- Conditions
- Primary Biliary Cholangitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Kowa Research Institute, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Feb 2024 (actual)
- Primary completion
- 26 Jun 2025 (actual)
- Study completion
- 23 Jul 2026 (actual)
- First posted
- 8 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo + K-808 (Group A) | Placebo comparator | Drug: K-808 (Dose A), Drug: PlaceboPlacebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks |
| Placebo + K-808 (Group B) | Placebo comparator | Drug: K-808 (Dose B), Drug: PlaceboPlacebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks |
| K-808 Group A | Experimental | Drug: K-808 (Dose A)K-808 (Dose A) for 64 Weeks |
| K-808 Group B | Experimental | Drug: K-808 (Dose B)K-808 (Dose B) for 64 Weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female participant who has a PBC diagnosis as demonstrated by the presence of ≥2 of the following three diagnostic criteria:
- Participant has the following qualifying biochemistry value at Screening:
- Participant is ≥18 years of age at consent.
- Participant meets all other eligibility criteria outlined in the Clinical Study Protocol.
Key exclusion criteria (14 listed)
- Participant meets any one of the following criteria at Screening:
- Participant meets any other exclusion criteria outlined in the Clinical Study Protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| K-808 (Dose A)Also: PemafibrateAdministered orally once daily | Drug | K-808 Group A, Placebo + K-808 (Group A) |
| K-808 (Dose B)Also: PemafibrateAdministered orally once daily | Drug | K-808 Group B, Placebo + K-808 (Group B) |
| PlaceboAdministered orally once daily | Drug | Placebo + K-808 (Group A), Placebo + K-808 (Group B) |
Primary outcome
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
6 secondary outcomes
Achievement of normalization of ALP level
Time frame: Baseline to Week 12
Achievement of target levels of ALP and total bilirubin (TB)
Time frame: Baseline to Weeks 12 and 64
Change from baseline in liver function parameters
Time frame: Baseline to Weeks 12 and 64
Change from baseline in GLOBE risk score
Time frame: Baseline to Weeks 12 and 64
Change from baseline in UK-PBC score
Time frame: Baseline to Weeks 12 and 64
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline to Week 68
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andre Belous, MD, PhD study director
Kowa Pharma Development Co.
38 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing K-808 (Dose A) and K-808 (Dose B) in people with Primary Biliary Cholangitis, run by Kowa Research Institute, Inc. at 38 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, Japan, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change from baseline in serum alkaline phosphatase (ALP) (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Oct 2026.