Ignite Creation Date:
2025-12-24 @ 2:51 PM
Ignite Modification Date:
2025-12-26 @ 9:54 PM
Study NCT ID:
NCT05569759
Status:
COMPLETED
Last Update Posted:
2025-05-30
First Post:
2022-09-29
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-11-24', 'mcpReleaseN': 16, 'releaseDate': '2025-11-12'}, {'resetDate': '2025-12-18', 'mcpReleaseN': 17, 'releaseDate': '2025-12-03'}, {'releaseDate': '2025-12-19'}], 'estimatedResultsFirstSubmitDate': '2025-11-12'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D019693', 'term': 'Hepatitis, Autoimmune'}], 'ancestors': [{'id': 'D006521', 'term': 'Hepatitis, Chronic'}, {'id': 'D006505', 'term': 'Hepatitis'}, {'id': 'D008107', 'term': 'Liver Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000712054', 'term': 'KZR-616'}, {'id': 'D007267', 'term': 'Injections'}], 'ancestors': [{'id': 'D004333', 'term': 'Drug Administration Routes'}, {'id': 'D004358', 'term': 'Drug Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}]}}, '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': 24}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2023-05-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-09', 'completionDateStruct': {'date': '2025-04-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-05-28', 'studyFirstSubmitDate': '2022-09-29', 'studyFirstSubmitQcDate': '2022-10-03', 'lastUpdatePostDateStruct': {'date': '2025-05-30', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-10-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-04-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Plasma concentrations of zetomipzomib and its metabolites', 'timeFrame': 'Baseline through Week 16', 'description': 'Maximum plasma concentration \\[Cmax\\]'}, {'measure': 'Plasma concentrations of zetomipzomib and its metabolites', 'timeFrame': 'Baseline through Week 16', 'description': 'Time of maximum plasma concentration \\[Tmax\\]'}, {'measure': 'Plasma concentrations of zetomipzomib and its metabolites', 'timeFrame': 'Baseline through Week 16', 'description': 'Area under the concentration-time curve \\[AUC\\]'}], 'primaryOutcomes': [{'measure': 'To evaluate the efficacy of zetomipzomib', 'timeFrame': 'Week 24', 'description': 'Proportion of patients who achieve complete biochemical remission (CR) by Week 24.'}, {'measure': 'To evaluate the safety and tolerability of zetomipzomib', 'timeFrame': 'Baseline through 28 weeks.', 'description': 'Proportion of patients who experience AEs (adverse events) and SAEs (serious adverse events), including incidence and severity of AEs and SAEs, incidence of AEs leading to drug discontinuation, and changes in laboratory parameters and vital signs.'}, {'measure': 'To evaluate the efficacy of zetomipzomib during the open-label extension period', 'timeFrame': 'Start of open-label extension (OLE) period through End of Study (EOS) at OLE Week 29', 'description': 'Proportion of patients experiencing a disease flare among the patients who achieved a complete biochemical remission (CR) during the double-blind treatment period.'}], 'secondaryOutcomes': [{'measure': 'Alanine aminotransferase (ALT)', 'timeFrame': 'Weeks 12, 16, 20, and 24', 'description': 'Changes from baseline in alanine aminotransferase (ALT)'}, {'measure': 'Partial Remission', 'timeFrame': 'Weeks 12, 16, 20, and 24', 'description': 'Proportion of patients who achieve a partial remission (PR)'}, {'measure': 'Time to complete remission', 'timeFrame': 'Baseline through Week 24', 'description': 'Time to complete remission (CR)'}, {'measure': 'Time to partial remission', 'timeFrame': 'Baseline through Week 24', 'description': 'Time to partial remission (PR)'}, {'measure': 'Disease flare after CR', 'timeFrame': 'Week 24', 'description': 'Proportion of patients experiencing a disease flare after complete remission (CR)'}, {'measure': 'Treatment failures', 'timeFrame': 'Week 24', 'description': 'Proportion of patients who are treatment failures'}, {'measure': 'Successful glucocorticoid taper', 'timeFrame': 'Week 24', 'description': 'Proportion of patients who achieve CR or PR with successful glucocorticoid taper by Week 24'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['immunoproteasome inhibition', 'selective proteasome inhibition', 'disease flare', 'Liver enzymes', 'ALT (alanine aminotransferase)', 'AST (aspartate aminotransferase)', 'glucocorticoids', 'steroids'], 'conditions': ['Autoimmune Hepatitis']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.kezarlifesciences.com', 'label': 'Corporate website'}]}, 'descriptionModule': {'briefSummary': 'This is a Phase 2a, multi-center, placebo-controlled study in which patients with autoimmune hepatitis will receive zetomipzomib or placebo in addition to standard-of-care for 24 weeks; an optional open-label extension period allows patients to receive zetomipzomib (KZR-616) for an additional 24 weeks of treatment.', 'detailedDescription': 'This is a Phase 2a, multi-center, randomized, double-blind, placebo-controlled study with an open-label extension to evaluate safety, tolerability, and efficacy of zetomipzomib in patients with autoimmune hepatitis (AIH) who have not benefited from standard-of-care treatment, had an incomplete response to ≥3 months of standard-of-care treatment, or had a disease flare after standard of care.\n\nZetomipzomib or placebo will be administered weekly for a 24-week treatment period in addition to standard-of-care (glucocorticoids), followed by a 4-week off-treatment safety follow-up period. Zetomipzomib and placebo will be administered subcutaneously (SC) once weekly.\n\nAt the end of the 24-week treatment period, eligible patients from both the zetomipzomib- and placebo-treated arms who complete the double-blind treatment period can enroll in the open-label extension period to receive an additional 24 weeks of treatment with zetomipzomib.\n\nPrimary completion date represents the anticipated completion date of the double-blind portion of the study. Study completion date represents the anticipated completion date of the open-label extension portion of the study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria for the Double-blind Treatment Period:\n\n* Must be aged ≥18 years.\n\n * Must have a clinical diagnosis of AIH and signs of active disease despite standard-of-care therapy for ≥3 months or disease flare after experiencing complete remission induced by standard-of-care treatment, including:\n\n * Screening ALT values that are 1.25 to 10 times the upper limit of the normal range (ULN)\n * Liver biopsy results with Ishak score (modified HAI) ≥5/18 indicating active AIH, from a biopsy performed at Screening, or within 6 months prior to Screening\n * Mild or no hepatic impairment (Child Pugh category A)\n * Must be willing to use and taper glucocorticoid therapy.\n * Must be willing to use effective contraception.\n\nKey Exclusion Criteria for the Double-blind Treatment Period:\n\n* Have a concomitant diagnosis of primary biliary sclerosis, primary sclerosing cholangitis, IgG 4 related cholangitis, drug related AIH (at Screening) or a history of drug-related AIH.\n* Have clinical evidence of significant unstable or uncontrolled diseases other than the disease under study.\n* Are receiving oral or injectable immunomodulating treatment for any other autoimmune disease prior to enrollment in the study. Patients who have been using such treatments must follow the specified washout periods.\n* Have an active infection (eg, acute hepatitis E, cytomegalovirus, or Epstein-Barr virus) requiring systemic therapy with antibiotic, antiviral, or antifungal treatment, or has had any febrile illness within 7 days prior to Day -1.\n* Have a history of thyroiditis, celiac disease, or other autoimmune disorder known to be associated with transaminitis.\n* Have liver cirrhosis with significant impairment of liver function (Child Pugh category B or C) or have decompensated cirrhosis.\n* Patients with histology confirmed coincident non-alcoholic steatohepatitis.\n\nKey Inclusion Criteria for the Open-label Extension Period:\n\n* Same as Double-blind Treatment Period inclusion criteria, except the following modifications:\n\n * ALT value can be normal or, if elevated, in the range of 1.25 to 10 times the upper limit of normal\n* Must have completed the Double-blind Period study visits through Week 24, including all Week 24 Visit assessments.\n* Must be willing to maintain glucocorticoid therapy or continue to taper glucocorticoid therapy.\n\nKey Exclusion Criteria for the Open-label Extension Period:\n\n• Same as Double-blind Treatment Period except no need to re-test for HIV, HBV, HCV, and TB.'}, 'identificationModule': {'nctId': 'NCT05569759', 'briefTitle': 'A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Kezar Life Sciences, Inc.'}, 'officialTitle': 'A Randomized, Double-blind, Placebo-controlled, Phase 2a Study With Open-label Extension to Evaluate the Safety and Efficacy of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis', 'orgStudyIdInfo': {'id': 'KZR-616-208'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'zetomipzomib + standard-of-care (glucocorticoids)', 'description': 'Initial 30 mg dose of zetomipzomib, followed by weekly 60 mg doses of zetomipzomib, for the remaining 23 weeks of the treatment period.', 'interventionNames': ['Drug: zetomipzomib']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'placebo + standard-of-care (glucocorticoids)', 'description': 'Initial 30 mg dose of placebo (sterile water for injection), followed by weekly 60 mg doses of placebo, for the remaining 23 weeks of the treatment period.', 'interventionNames': ['Drug: placebo']}, {'type': 'EXPERIMENTAL', 'label': 'zetomipzomib + standard-of care (glucocorticoids) open-label extension period', 'description': 'Initial 30 mg dose of zetomipzomib at the open-label extension (OLE) Week 1 visit, followed by weekly doses of 60 mg of zetomipzomib, for a total of 24 additional weeks of treatment.', 'interventionNames': ['Drug: zetomipzomib in open-label extension']}], 'interventions': [{'name': 'zetomipzomib', 'type': 'DRUG', 'otherNames': ['KZR-616'], 'description': 'Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly', 'armGroupLabels': ['zetomipzomib + standard-of-care (glucocorticoids)']}, {'name': 'placebo', 'type': 'DRUG', 'otherNames': ['sterile water for injection'], 'description': 'Subcutaneous injection of placebo', 'armGroupLabels': ['placebo + standard-of-care (glucocorticoids)']}, {'name': 'zetomipzomib in open-label extension', 'type': 'DRUG', 'otherNames': ['KZR-616'], 'description': 'Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly', 'armGroupLabels': ['zetomipzomib + standard-of care (glucocorticoids) open-label extension period']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85054', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Mayo Clinic Arizona', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90033', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Keck School of Medicine of USC', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94063', 'city': 'Redwood City', 'state': 'California', 'country': 'United States', 'facility': 'Stanford Medicine', 'geoPoint': {'lat': 37.48522, 'lon': -122.23635}}, {'zip': '94109', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'California Pacific Medical Center', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '94132', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06510', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'Mayo Clinic Florida', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '70121', 'city': 'New Orleans', 'state': 'Louisiana', 'country': 'United States', 'facility': 'Ochsner Clinic Foundation', 'geoPoint': {'lat': 29.95465, 'lon': -90.07507}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Beth Israel Deaconess Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University of Michigan Medical Center', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '11030', 'city': 'Manhasset', 'state': 'New York', 'country': 'United States', 'facility': 'Northwell Health Center for Liver Disease and Transplantation', 'geoPoint': {'lat': 40.79788, 'lon': -73.69957}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University Langone Health/Grossman School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'University Hospitals Cleveland Medical Center', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '23298', 'city': 'Richmond', 'state': 'Virginia', 'country': 'United States', 'facility': 'Virginia Commonwealth University', 'geoPoint': {'lat': 37.55376, 'lon': -77.46026}}], 'overallOfficials': [{'name': 'Craig Lammert, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Indiana University'}, {'name': 'Ethan Weinberg, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Pennsylvania'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kezar Life Sciences, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}