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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04628936 | Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM. | PHASE2 | COMPLETED | 18 | — | — | Nov 4, 2020 | Jun 12, 2023 | Nov 19, 2025 | 10 | United States, Czechia |
| NCT04033926 | A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis | PHASE2 | COMPLETED | 25 | — | — | Jan 14, 2020 | Apr 6, 2022 | Nov 19, 2025 | 14 | United States, Czechia +1 |
The mean Total Improvement Score (TIS) at OLE Week 48, which ranges from 0 to 100 \[low of 0 to high of 100, where higher scores are better\]. The timeframe of 48 weeks was selected because it represented the maximum timeframe of dosing for the last patient enrolled as the study drug administration ended when the last patient enrolled completed 48 weeks of dosing.
The primary efficacy endpoint was mean change from start to end of zetomipzomib (KZR-616) Treatment Periods in the Total Improvement Score (TIS), which ranges from 0 to 100 \[low of 0 to high of 100, where higher scores are better\]. Mean change in TIS was calculated by comparing the Baseline and post Baseline observations for patients in both KZR-616 treatment periods combined. Note: TIS scores for placebo treatment periods are presented in this outcome measure but were not included in the primary outcome measure analysis.
| Arm | Type | Description |
|---|---|---|
| KZR-616 45 mg + standard therapy (open-label) | EXPERIMENTAL | All patients received a SC injection of 30 mg KZR-616 at Visit 1 (Day 1), followed by weekly SC injections of 45 mg KZR-616 up to a maximum of 96 weeks. Study drug administration ended for all patients in Study KZR-616-003E when the last patient enrolled completed 48 weeks of dosing. |
| Arm A | OTHER | * Treatment Period 1: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks * Treatment Period 2: Placebo SC weekly for 16 weeks |
| Arm B | OTHER | * Treatment Period 1: Placebo SC weekly for 16 weeks * Treatment Period 2: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks |
| Name | Type | Description |
|---|---|---|
| KZR-616 | DRUG | Subcutaneous 30 mg for 1 week, then 45 mg weekly |
| Placebo | DRUG | Subcutaneous injection for 16 weeks |
Inclusion Criteria: * Must have successfully completed Study KZR-616-003 through Week 32, including the Week 32 Visit assessments * Women of childbearing potential (WOCBP) must have a negative urine or serum pregnancy test prior to the first dose of KZR-616 in KZR-616-003E, and must agree to contin...