Pemafibrate for Primary Biliary Cirrhosis
Recruiting in Palo Alto (17 mi)
+37 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Kowa Research Institute, Inc.
Disqualifiers: Hepatitis C, Hepatitis B, Alcoholic liver disease, Autoimmune hepatitis, NASH, others
Prior Safety Data
Approved in 1 Jurisdiction
Trial Summary
What is the purpose of this trial?Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
How is the drug pemafibrate different from other treatments for primary biliary cirrhosis?
Pemafibrate is unique because it is specifically studied for its effects on primary biliary cholangitis (a condition similar to primary biliary cirrhosis) in patients with dyslipidemia (abnormal cholesterol levels), which may offer benefits beyond standard treatments like ursodeoxycholic acid.
12345Eligibility Criteria
This trial is for people with Primary Biliary Cholangitis (PBC) who haven't had enough improvement with standard treatments like Ursodeoxycholic Acid or Obeticholic Acid. Specific eligibility details are not provided, but typically participants must meet certain health standards and may be excluded based on other medical conditions.Inclusion Criteria
Participant has ALP ≥1.5 × ULN
Participant meets all other eligibility criteria outlined in the Clinical Study Protocol
Male or female participant with a PBC diagnosis 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
+1 more
Exclusion Criteria
Participant has Gilbert's Syndrome
I have been diagnosed with Alpha-1-antitrypsin deficiency, cystic fibrosis, Wilson's disease, or hemochromatosis.
I have a liver condition known as sclerosing cholangitis.
+10 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive either K-808 (Pemafibrate) or placebo for 12 weeks
12 weeks
Extension
Participants who received placebo switch to K-808 (Pemafibrate) for 52 weeks
52 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study is testing the effectiveness and safety of two different doses of a drug called K-808 (pemafibrate) compared to a placebo. Participants will randomly receive either one of the K-808 doses or a placebo to see if there's an improvement in their PBC symptoms.
4Treatment groups
Experimental Treatment
Placebo Group
Group I: K-808 Group BExperimental Treatment1 Intervention
K-808 (Dose B) for 64 Weeks
Group II: K-808 Group AExperimental Treatment1 Intervention
K-808 (Dose A) for 64 Weeks
Group III: Placebo + K-877 (Group B)Placebo Group2 Interventions
Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks
Group IV: Placebo + K-877 (Group A)Placebo Group2 Interventions
Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks
Pemafibrate is already approved in Japan for the following indications:
🇯🇵 Approved in Japan as Parmodia for:
- Hyperlipidaemia
- Familial hyperlipidaemia
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Southern California Research Center - CoronadoCoronado, CA
Peak Gastroenterology Associates Colorado SpringsColorado Springs, CO
University of Florida Hepatology Research at CTRBGainesville, FL
Florida Research InstituteLakewood Ranch, FL
More Trial Locations
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Who Is Running the Clinical Trial?
Kowa Research Institute, Inc.Lead Sponsor
References
Effects of pemafibrate on primary biliary cholangitis with dyslipidemia. [2022]The purpose of this study was to examine the effect of pemafibrate (PEM) in primary biliary cholangitis (PBC) patients with dyslipidemia.
Bezafibrate normalizes alkaline phosphatase in primary biliary cirrhosis patients with incomplete response to ursodeoxycholic acid. [2022]Ursodeoxycholic acid (UDCA) is the standard treatment for primary biliary cirrhosis (PBC) but excellent response is not observed in all cases. Since potential favourable effects of fibrates have been reported in short series with inconclusive results, we have carried out a pilot study to analyse the effects of bezafibrate in patients with suboptimal response to UDCA.
Seladelpar: an investigational drug for the treatment of early-stage primary biliary cholangitis (PBC). [2023]Generic fibrates are used off-label as add-in therapy for the management of primary biliary cholangitis (PBC) but with unproven long-term liver-related survival benefits. The recently developed fibrate, seladelpar, has shown promising results in clinical trials, but these outcomes have been previously marred by safety concerns.
Bezafibrate treatment: a new medical approach for PBC patients? [2022]A new medical approach to primary biliary cirrhosis (PBC) has been desired. We investigated the feasibility of using combination ursodeoxycholic acid (UDCA)-bezafibrate therapy in patients with PBC nonresponsive to UDCA monotherapy.
Biochemical responses to bezafibrate improve long-term outcome in asymptomatic patients with primary biliary cirrhosis refractory to UDCA. [2021]Bezafibrate is reported to have biochemical efficacy for patients with primary biliary cirrhosis (PBC) refractory to ursodeoxycholic acid (UDCA), yet the long-term effect is still unknown. In Japan, nationwide surveys of PBC have been performed since 1980. In the current study, we retrospectively examined whether response to bezafibrate treatment is associated with the long-term outcomes using this large-scale database.