Trial · NCT04033926
A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis
NCT04033926, Phase 2, Completed, Kezar Life Sciences, Inc.; results first posted 2024-01-03
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis. Trials, record NCT04033926. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04033926
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis
- NCT number
- NCT04033926
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- Kezar Life Sciences, Inc.
- Conditions
- Polymyositis, Dermatomyositis
- Interventions
- KZR-616, Placebo
- First posted
- 2019-07-26
- Last updated
- 2025-11-19
- Results first posted
- 2024-01-03
- Limitations and caveats
* This study was not powered to detect statistical differences between treatment arms.~* Crossover study design with no washout period may have confounded assessments.~* Limited information from the safety follow-up as most patients elected to join the OLE study (NCT04628936).
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | 16 weeks in each Treatment Period (32 weeks total) | 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. |
| Secondary | Proportion of Patients With TIS Response | 16 weeks in each Treatment Period (32 weeks total) | The proportion of patients with an increase of ≥ 20 points on the TIS from start to end of zetomipzomib (KZR-616) treatment. TIS response is categorized by the following improvement thresholds: * Minimal response = TIS ≥ 20 * Moderate response = TIS ≥ 40 * Major response = TIS ≥ 60 This endpoint was assessed by comparing Week 16 versus Week 0 for patients allocated to Arm A and Week 32 versus Week 16 for patients allocated to Arm B. This re-baselining approach was utilized to maximize the precision for assessment of zetomipzomib effect in Arm B. |
| Secondary | Number of Patients Meeting the International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI) | 16 weeks in each Treatment Period (32 weeks total) | The IMACS DOI is ≥ 20% improvement in at least 3 of 6 core set activity measures, with no more than 2 core set activity measures (CSAMs) worsening by ≥ 25% (Manual Muscle Testing-8 Muscle Groups \[MMT-8\] could not be a worsening measure). |
| Secondary | Mean Percent Change From Baseline From Start to End of Treatment in the IMACS Individual CSAMs | 16 weeks in each Treatment Period (32 weeks total) | Mean percent change from baseline of the IMACS CSAMs consisting of: * Physician Global Assessment: physician assessment of patient's overall disease activity at present, high numbers indicate more severe disease activity \[0-10\] * Patient Global Assessments of Disease Activity: patient assessment of their overall disease activity at present, high numbers indicate more severe disease activity \[0-100\] * Manual Muscle Testing-8 Muscle Groups: scores range from 0 - 150, high scores are better * Health Assessment Questionnaire-Disability Index: scores range from 0 - 3, high scores are worse * Extramuscular Global Assessment of the Myositis Disease Activity Assessment Tool (2005 version): scores range from 0 - 10, high scores are worse * Muscle enzymes (clinical laboratory assessments \[CLA\]): Summarize the most abnormal CLA (creatine kinase \[CK\], aldolase, lactate dehydrogenase \[LDH\], alanine aminotransferase \[ALT\], or aspartate aminotransferase \[AST\]) at baseline, lower scores are better |
| Secondary | Mean Change in CDASI From Start to End of Zetomipzomib (KZR-616) Treatment | 16 weeks in each Treatment Period (32 weeks total) | Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) is a clinician scored single-page instrument that separately measures activity and damage, which consists of three (3) activity measures and two (2) damage measures which are assessed over 15 body areas. Scores range from 0-100 for activity and from 0-32 for damage, with higher scores indicating more severe disease. |
| Secondary | Mean Change in PP-NRS From Start to End of Zetomipzomib (KZR-616) Treatment | 16 weeks in each Treatment Period (32 weeks total) | The Peak Pruritus Numeric Rating Scale (PP-NRS) is used to evaluate severity of itch in DM patients. Scores range from 0-10, with zero (0) representing no itch and ten (10) representing the worst itch imaginable within a 24-hour recall period. |
| Secondary | PK of Zetomipzomib [KZR-616] (Cmax) | Up to 5 hours | This is the maximum observed plasma concentration (Cmax) observed after administration of the first dose of KZR-616 (either Week 0 or Week 16). The pharmacokinetic (PK) parameters were calculated using all timepoints at which the concentration was measured, ie. pre-dose and 30 minutes, and 4 hours post-dose, with an additional sample obtained at 0.25, 1, or 2 hours post-dose. |
| Secondary | PK of Zetomipzomib [KZR-616] (Tmax) | Up to 5 hours | This is the time to maximum observed plasma concentration (tmax) observed after administration of the first dose of KZR-616 (either Week 0 or Week 16). The PK parameters were calculated using all timepoints at which the concentration was measured, ie. predose and 30 minutes, and 4 hours postdose, with an additional sample obtained at 0.25, 1, or 2 hours postdose. |
| Secondary | PK of Zetomipzomib [KZR-616] (AUC) | Up to 5 hours | This is the area under the curve (AUC) from predose through 4 hour postdose observed after administration of the first dose of KZR-616 (either Week 0 or Week 16). The PK parameters were calculated using all timepoints at which the concentration was measured, ie. predose and 30 minutes, and 4 hours postdose, with an additional sample obtained at 0.25, 1, or 2 hours postdose. |
| Secondary | PK of KZR-59587 (Cmax) | Up to 5 hours | This is the maximum observed plasma concentration of KZR-59587 (Cmax) observed after administration of the first dose of KZR-616 (either Week 0 or Week 16). The pharmacokinetic (PK) parameters were calculated using all timepoints at which the concentration was measured, ie. pre-dose and 30 minutes, and 4 hours post-dose, with an additional sample obtained at 0.25, 1, or 2 hours post-dose. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | 16 weeks in each Treatment Period (32 weeks total) | For the first 16 weeks, analysis was done for Treatment Period 1. For the second 16 weeks, analysis was done for Treatment Period 2. | score on a scale | MEAN |
| SECONDARY | Proportion of Patients With TIS Response | 16 weeks in each Treatment Period (32 weeks total) | participants | NUMBER | |
| SECONDARY | Number of Patients Meeting the International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI) | 16 weeks in each Treatment Period (32 weeks total) | participants | NUMBER | |
| SECONDARY | Mean Percent Change From Baseline From Start to End of Treatment in the IMACS Individual CSAMs | 16 weeks in each Treatment Period (32 weeks total) | Some values were not collected for participants. | percent change | MEAN |
| SECONDARY | Mean Change in CDASI From Start to End of Zetomipzomib (KZR-616) Treatment | 16 weeks in each Treatment Period (32 weeks total) | This measure was only performed for patients with DM. | score on a scale | MEAN |
| SECONDARY | Mean Change in PP-NRS From Start to End of Zetomipzomib (KZR-616) Treatment | 16 weeks in each Treatment Period (32 weeks total) | This measure was only performed for patients with DM. | score on a scale | MEAN |
| SECONDARY | PK of Zetomipzomib [KZR-616] (Cmax) | Up to 5 hours | ng/mL | GEOMETRIC_MEAN | |
| SECONDARY | PK of Zetomipzomib [KZR-616] (Tmax) | Up to 5 hours | hours | GEOMETRIC_MEAN | |
| SECONDARY | PK of Zetomipzomib [KZR-616] (AUC) | Up to 5 hours | ng*h/mL | GEOMETRIC_MEAN | |
| SECONDARY | PK of KZR-59587 (Cmax) | Up to 5 hours | ng/mL | GEOMETRIC_MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group68
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | Arm A: Period 1 (Zetomipzomib) | 25.5 | Standard Deviation 18.6 | score on a scale | |
| Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | Arm B: Period 1 (Placebo) | 25.0 | Standard Deviation 19.9 | score on a scale | |
| Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | Arm A: Period 2 (Placebo) | 33.1 | Standard Deviation 17.6 | score on a scale | |
| Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period | Arm B: Period 2 (Zetomipzomib) | 33.5 | Standard Deviation 22.9 | score on a scale | |
| Proportion of Patients With TIS Response | Arm A: Period 1 (Zetomipzomib) | Minimal (TIS ≥ 20) | 6 | participants | |
| Proportion of Patients With TIS Response | Arm B: Period 1 (Placebo) | Minimal (TIS ≥ 20) | 7 | participants | |
| Proportion of Patients With TIS Response | Arm A: Period 2 (Placebo) | Minimal (TIS ≥ 20) | 1 | participants | |
| Proportion of Patients With TIS Response | Arm B: Period 2 (Zetomipzomib) | Minimal (TIS ≥ 20) | 6 | participants | |
| Proportion of Patients With TIS Response | Arm A: Period 1 (Zetomipzomib) | Moderate (TIS ≥ 40) | 2 | participants | |
| Proportion of Patients With TIS Response | Arm B: Period 1 (Placebo) | Moderate (TIS ≥ 40) | 2 | participants |
1 to 10 of 68Source ClinicalTrials.gov
See all 68 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1 | STARTED | Zetomipzomib First, Then Placebo (Arm A) | 13 | |
| Treatment Period 1 | STARTED | Placebo First, Then Zetomipzomib (Arm B) | 12 | |
| Treatment Period 1 | COMPLETED | Zetomipzomib First, Then Placebo (Arm A) | 10 | |
| Treatment Period 1 | COMPLETED | Placebo First, Then Zetomipzomib (Arm B) | 12 | |
| Treatment Period 1 | NOT COMPLETED | Zetomipzomib First, Then Placebo (Arm A) | 3 | |
| Treatment Period 1 | NOT COMPLETED | Placebo First, Then Zetomipzomib (Arm B) | 0 | |
| Treatment Period 2 | STARTED | Zetomipzomib First, Then Placebo (Arm A) | 10 | |
| Treatment Period 2 | STARTED | Placebo First, Then Zetomipzomib (Arm B) | 12 | |
| Treatment Period 2 | COMPLETED | Zetomipzomib First, Then Placebo (Arm A) | 8 | |
| Treatment Period 2 | COMPLETED | Placebo First, Then Zetomipzomib (Arm B) | 12 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events400
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Palpitations | Cardiac disorders | Arm A: Period 1 (Zetomipzomib) | 1 | 13 | 2 |
| other | Palpitations | Cardiac disorders | Arm A: Period 2 (Placebo) | 0 | 10 | 0 |
| other | Palpitations | Cardiac disorders | Arm B: Period 1 (Placebo) | 0 | 12 | 0 |
| other | Palpitations | Cardiac disorders | Arm B: Period 2 (Zetomipzomib) | 0 | 12 | 0 |
| other | Sinus tachycardia | Cardiac disorders | Arm A: Period 1 (Zetomipzomib) | 2 | 13 | 2 |
| other | Sinus tachycardia | Cardiac disorders | Arm A: Period 2 (Placebo) | 0 | 10 | 0 |
| other | Sinus tachycardia | Cardiac disorders | Arm B: Period 1 (Placebo) | 0 | 12 | 0 |
| other | Sinus tachycardia | Cardiac disorders | Arm B: Period 2 (Zetomipzomib) | 0 | 12 | 0 |
| other | Keratitis | Eye disorders | Arm A: Period 1 (Zetomipzomib) | 0 | 13 | 0 |
| other | Keratitis | Eye disorders | Arm A: Period 2 (Placebo) | 0 | 10 | 0 |
1 to 10 of 400Source ClinicalTrials.gov
See all 400 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm A: Period 1 (Zetomipzomib) | 0 | 13 |
| Total, all-cause mortality | Arm A: Period 2 (Placebo) | 0 | 10 |
| Total, all-cause mortality | Arm B: Period 1 (Placebo) | 0 | 12 |
| Total, all-cause mortality | Arm B: Period 2 (Zetomipzomib) | 0 | 12 |
| Total, other adverse events | Arm A: Period 1 (Zetomipzomib) | 12 | 13 |
| Total, other adverse events | Arm A: Period 2 (Placebo) | 8 | 10 |
| Total, other adverse events | Arm B: Period 1 (Placebo) | 7 | 12 |
| Total, other adverse events | Arm B: Period 2 (Zetomipzomib) | 10 | 12 |
| Total, serious adverse events | Arm A: Period 1 (Zetomipzomib) | 1 | 13 |
| Total, serious adverse events | Arm A: Period 2 (Placebo) | 0 | 10 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04033926
GET https://conductscience.org/api/trials/v1/NCT04033926/results?page=2