A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)
- Conditions
- Autoimmune Hepatitis
- Interventions
- Registration Number
- NCT05569759
- Lead Sponsor
- Kezar Life Sciences, Inc.
- Brief Summary
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.
- Detailed Description
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 โฅ3 months of standard-of-care treatment, or had a disease flare after standard of care.
Zetomipzomib 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.
At 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.
Primary 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 24
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description zetomipzomib + standard-of-care (glucocorticoids) zetomipzomib Initial 30 mg dose of zetomipzomib, followed by weekly 60 mg doses of zetomipzomib, for the remaining 23 weeks of the treatment period. placebo + standard-of-care (glucocorticoids) placebo 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. zetomipzomib + standard-of care (glucocorticoids) open-label extension period zetomipzomib in open-label extension 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.
- Primary Outcome Measures
Name Time Method To evaluate the efficacy of zetomipzomib Week 24 Proportion of patients who achieve complete biochemical remission (CR) by Week 24.
To evaluate the safety and tolerability of zetomipzomib Baseline through 28 weeks. 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.
To evaluate the efficacy of zetomipzomib during the open-label extension period Start of open-label extension (OLE) period through End of Study (EOS) at OLE Week 29 Proportion of patients experiencing a disease flare among the patients who achieved a complete biochemical remission (CR) during the double-blind treatment period.
- Secondary Outcome Measures
Name Time Method Alanine aminotransferase (ALT) Weeks 12, 16, 20, and 24 Changes from baseline in alanine aminotransferase (ALT)
Partial Remission Weeks 12, 16, 20, and 24 Proportion of patients who achieve a partial remission (PR)
Time to complete remission Baseline through Week 24 Time to complete remission (CR)
Time to partial remission Baseline through Week 24 Time to partial remission (PR)
Disease flare after CR Week 24 Proportion of patients experiencing a disease flare after complete remission (CR)
Treatment failures Week 24 Proportion of patients who are treatment failures
Successful glucocorticoid taper Week 24 Proportion of patients who achieve CR or PR with successful glucocorticoid taper by Week 24
Trial Locations
- Locations (24)
Northwell Health Center for Liver Disease and Transplantation
๐บ๐ธManhasset, New York, United States
University of Pennsylvania
๐บ๐ธPhiladelphia, Pennsylvania, United States
Beth Israel Deaconess Medical Center
๐บ๐ธBoston, Massachusetts, United States
Mayo Clinic Arizona
๐บ๐ธPhoenix, Arizona, United States
University of California, San Francisco
๐บ๐ธSan Francisco, California, United States
California Pacific Medical Center
๐บ๐ธSan Francisco, California, United States
University of Colorado
๐บ๐ธAurora, Colorado, United States
University of Miami
๐บ๐ธMiami, Florida, United States
University of California, Los Angeles
๐บ๐ธLos Angeles, California, United States
Keck School of Medicine of USC
๐บ๐ธLos Angeles, California, United States
Stanford Medicine
๐บ๐ธRedwood City, California, United States
Mayo Clinic Florida
๐บ๐ธJacksonville, Florida, United States
Northwestern University
๐บ๐ธChicago, Illinois, United States
Rush University
๐บ๐ธChicago, Illinois, United States
Indiana University
๐บ๐ธIndianapolis, Indiana, United States
Massachusetts General Hospital
๐บ๐ธBoston, Massachusetts, United States
New York University Langone Health/Grossman School of Medicine
๐บ๐ธNew York, New York, United States
Duke University Medical Center
๐บ๐ธDurham, North Carolina, United States
Washington University School of Medicine
๐บ๐ธSaint Louis, Missouri, United States
University Hospitals Cleveland Medical Center
๐บ๐ธCleveland, Ohio, United States
University of Michigan Medical Center
๐บ๐ธAnn Arbor, Michigan, United States
Yale University
๐บ๐ธNew Haven, Connecticut, United States
Ochsner Clinic Foundation
๐บ๐ธNew Orleans, Louisiana, United States
Virginia Commonwealth University
๐บ๐ธRichmond, Virginia, United States
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