Clinical Research Studies

This is a Phase 2a, multi-center, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and efficacy of zetomipzomib in patients with AIH who have failed standard-of-care treatment, had an incomplete biochemical response to =3 months of standard-of-care treatment, or had a disease flare after standard-of-care treatment. Zetomipzomib or placebo will be administered weekly for a 24-week treatment period, followed by a 4-week off-treatment safety follow-up period. A screening period of up to 4 weeks will precede Day 1. Efficacy will be assessed at Weeks 12, 16, 20, and 24; assessments are discussed in Section 7.2.2. Safety will be assessed throughout the study by monitoring of vital signs, clinical laboratory tests, and physical examinations, and by recording and analyzing all adverse events (AEs) and serious AEs (SAEs). Eligible patients will be randomized in a 2:1 ratio on Day 1 (Visit 2) to receive either standard-of-care (glucocorticoids) with zetomipzomib (Active Treatment group), or standard-of-care with Placebo (Control group). Zetomipzomib and placebo (ie, sterile water for injection [sWFI] in an equivalent volume to the reconstituted zetomipzomib dose), will be administered subcutaneously (SC) once weekly. The first dose of investigational medicinal product (IMP) will be 30 mg zetomipzomib or placebo, followed by weekly doses of 60 mg zetomipzomib or placebo for the remaining 23 weeks of the treatment period.