Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05569759 | A Study of Zetomipzomib (KZR-616) in Patients With Autoimmune Hepatitis (PORTOLA) | PHASE2 | COMPLETED | 24 | — | — | May 23, 2023 | Apr 30, 2025 | Jan 13, 2026 | 24 | United States |
The number of patients who achieve complete biochemical response (CR), defined as normal ALT, AST, and IgG values (if IgG level is elevated at Baseline) with glucocorticoid dose not higher than starting dose (at Baseline), by Week 24 of the Double-Blind Treatment Period. Analyses were also conducted at Week 12, Week 16, and Week 20.
Proportion of participants who experience AEs (adverse events) and SAEs (serious adverse events) during the double-blind treatment period (DBTP) and the open-label extension (OLE).
Proportion of participants experiencing a disease flare among the participants who achieved a complete biochemical response (CR) during the double-blind treatment period.
| Arm | Type | Description |
|---|---|---|
| zetomipzomib + standard-of-care (glucocorticoids) | EXPERIMENTAL | 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_COMPARATOR | 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 | EXPERIMENTAL | 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 up to a total of 24 additional weeks of treatment. |
| Name | Type | Description |
|---|---|---|
| zetomipzomib | DRUG | Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly |
| placebo | DRUG | Subcutaneous injection of placebo |
| zetomipzomib in open-label extension | DRUG | Subcutaneous injection of zetomipzomib with a target dose of 60 mg weekly |
Key Inclusion Criteria for the Double-blind Treatment Period: * Must be aged ≥18 years. * 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,...