Ignite Creation Date:
2025-12-24 @ 10:06 PM
Ignite Modification Date:
2025-12-27 @ 11:29 AM
Study NCT ID:
NCT06247735
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-09-10
First Post:
2024-01-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
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.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008105', 'term': 'Liver Cirrhosis, Biliary'}], 'ancestors': [{'id': 'D002780', 'term': 'Cholestasis, Intrahepatic'}, {'id': 'D002779', 'term': 'Cholestasis'}, {'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D008103', 'term': 'Liver Cirrhosis'}, {'id': 'D005355', 'term': 'Fibrosis'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C540740', 'term': '(R)-2-(3-((benzoxazol-2-yl-d4 (3-(4-methoxyphenoxy-d7)propyl)amino)methyl)phenoxy) butanoic acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 46}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-02-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2026-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-09', 'studyFirstSubmitDate': '2024-01-22', 'studyFirstSubmitQcDate': '2024-01-30', 'lastUpdatePostDateStruct': {'date': '2025-09-10', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-02-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-06-26', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percent change from baseline in serum alkaline phosphatase (ALP)', 'timeFrame': 'Baseline to Week 12', 'description': 'Two doses of K-808 compared to placebo after 12 weeks of treatment'}], 'secondaryOutcomes': [{'measure': 'Achievement of normalization of ALP level', 'timeFrame': 'Baseline to Week 12', 'description': 'ALP ≤1 × upper limit of normal (ULN)'}, {'measure': 'Achievement of target levels of ALP and total bilirubin (TB)', 'timeFrame': 'Baseline to Weeks 12 and 64', 'description': 'After two doses of K-808'}, {'measure': 'Change from baseline in liver function parameters', 'timeFrame': 'Baseline to Weeks 12 and 64', 'description': 'liver function test results including ALP, total and conjugated bilirubin, albumin, international normalized ratio \\[INR\\], γ-GT, ALT, AST, albumin, platelets count'}, {'measure': 'Change from baseline in GLOBE risk score', 'timeFrame': 'Baseline to Weeks 12 and 64', 'description': 'calculated by GLOBE scoring system which is calculated based on serum values of bilirubin, ALP, albumin and platelet count.'}, {'measure': 'Change from baseline in UK-PBC score', 'timeFrame': 'Baseline to Weeks 12 and 64', 'description': '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.'}, {'measure': 'Incidence of Treatment Emergent Adverse Events (TEAEs)', 'timeFrame': 'Baseline to Week 68', 'description': 'Coded using the most recent version of Medical Dictionary of Regulatory Activities (MedDRA).'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Primary Biliary Cholangitis']}, 'descriptionModule': {'briefSummary': 'Study to investigate the efficacy and safety of two doses of K-808 (pemafribate) in subjects with PBC.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male or female participant who has a PBC diagnosis as demonstrated by the presence of ≥2 of the following three diagnostic criteria:\n\n * History of ALP above ULN for at least 6 months\n * History of positive antimitochondrial antibody (AMA) titer or positive PBC-specific antinuclear antibody (ANA) titer\n * Historical liver biopsy consistent with PBC\n* Participant has the following qualifying biochemistry value at Screening:\n\n * ALP ≥1.5 × ULN\n* Participant is ≥18 years of age at consent.\n* Participant meets all other eligibility criteria outlined in the Clinical Study Protocol.\n\nExclusion Criteria:\n\n* Participant meets any one of the following criteria at Screening:\n\n * ALP\\>10 × ULN\n * ALT or AST \\>5 × ULN\n * 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.\n * Primary sclerosing cholangitis and secondary sclerosing cholangitis (eg, due to cholangiolithiasis, ischemia, telangiectasia, vasculitis, infectious diseases)\n * Alcoholic liver disease\n * 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\n * Nonalcoholic steatohepatitis (NASH)\n * Gilbert's Syndrome\n * Alpha-1-antitrypsin deficiency, cystic fibrosis, Wilson's disease, hemochromatosis based on historically established diagnosis\n * Drug-induced liver injury (DILI) as defined by typical exposure and history\n * 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\n * Hepatocellular carcinoma\n* Participant meets any other exclusion criteria outlined in the Clinical Study Protocol."}, 'identificationModule': {'nctId': 'NCT06247735', 'briefTitle': '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.', 'organization': {'class': 'INDUSTRY', 'fullName': 'Kowa Research Institute, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'K-808-2.01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo + K-877 (Group A)', 'description': 'Placebo for 12 Weeks followed by K-808 (Dose A) for 52 Weeks', 'interventionNames': ['Drug: K-808 (Dose A)', 'Drug: Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo + K-877 (Group B)', 'description': 'Placebo for 12 Weeks followed by K-808 (Dose B) for 52 Weeks', 'interventionNames': ['Drug: K-808 (Dose B)', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'K-808 Group A', 'description': 'K-808 (Dose A) for 64 Weeks', 'interventionNames': ['Drug: K-808 (Dose A)']}, {'type': 'EXPERIMENTAL', 'label': 'K-808 Group B', 'description': 'K-808 (Dose B) for 64 Weeks', 'interventionNames': ['Drug: K-808 (Dose B)']}], 'interventions': [{'name': 'K-808 (Dose A)', 'type': 'DRUG', 'otherNames': ['Pemafibrate'], 'description': 'Administered orally once daily', 'armGroupLabels': ['K-808 Group A', 'Placebo + K-877 (Group A)']}, {'name': 'K-808 (Dose B)', 'type': 'DRUG', 'otherNames': ['Pemafibrate'], 'description': 'Administered orally once daily', 'armGroupLabels': ['K-808 Group B', 'Placebo + K-877 (Group B)']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Administered orally once daily', 'armGroupLabels': ['Placebo + K-877 (Group A)', 'Placebo + K-877 (Group B)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'UA Thomas D. 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