NCT05569759

A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA)

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Kezar Life Sciences, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a Phase 2a, multi-center, placebo-controlled study in which patients with autoimmune hepatitis received zetomipzomib or placebo in addition to standard-of-care for 24 weeks; an optional open-label extension period allowed participants to receive zetomipzomib (KZR-616) for an additional 24 weeks of treatment.

Detailed description

This was 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. Zetomipzomib or placebo were 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 was administered subcutaneously (SC) once weekly. At the end of the 24-week treatment period, eligible participants from both the zetomipzomib- and placebo-treated arms who completed the double-blind treatment period could enroll in the open-label extension period to receive up to an additional 24 weeks of treatment with zetomipzomib.

Conditions

• Autoimmune Hepatitis

Interventions

Type	Name	Description
DRUG	zetomipzomib	Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly
DRUG	placebo	Subcutaneous injection of placebo
DRUG	zetomipzomib in open-label extension	Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly

Timeline

• Start date: 2023-05-23
• Primary completion: 2025-04-30
• Completion: 2025-04-30
• First posted: 2022-10-06
• Last updated: 2026-01-13
• Results posted: 2026-01-13

Locations

24 sites across 1 country: United States

Regulatory

• FDA-regulated drug study