Dermatomyositis, Polymyositis
Conditions
Keywords
Myositis, Idiopathic inflammatory myopathies, Polymyositis, Dermatomyositis, Musculoskeletal Diseases, Muscular Diseases
Brief summary
This was an open-label study to evaluate the long-term efficacy and safety of KZR-616 in patients with active polymyositis (PM) or dermatomyositis (DM) who completed the double-blind treatment period of Study KZR-616-003, up to and including the Week 32 Visit, prior to the first dose of open-label KZR-616.
Detailed description
This was an open-label study to evaluate the long-term efficacy and safety of KZR-616 in patients with active polymyositis or dermatomyositis who completed the double-blind treatment period of Study KZR-616-003 (NCT04033926), up to and including the Week 32 Visit, prior to the first dose of open-label KZR-616. Patients were evaluated for eligibility according to the entry criteria at, or within 8 weeks after, the Week 32 Visit (ie, the End of Treatment \[EOT\] Visit \[Visit 34\]) of Study KZR-616-003. All patients received a subcutaneous (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. Patients had a final follow-up visit 12 weeks after their last dose of KZR-616 (End of Study \[EOS\] Visit), for a maximum potential length of participation of 108 weeks.
Interventions
DRUGKZR-616
Subcutaneous 30 mg for 1 week, then 45 mg weekly
Sponsors
Kezar Life Sciences, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
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 when the last patient enrolled completed 48 weeks of dosing.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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 continue to use a highly effective method of birth control until completion of the study. * Male patients must continue to use an effective contraception method for 1 week following their last dose of KZR-616 or be congenitally or surgically sterile.
Exclusion criteria
* Has participated in any clinical study other than KZR-616-003 between the Week 32 Visit of Study KZR-616-003 and the first study visit of KZR-616-003E, if they are not on the same calendar day. * Are females who are breastfeeding or who plan to become pregnant during the study, or who are actively trying to conceive at the time of signing of the informed consent form. * Have hypersensitivity to KZR-616 or any of its excipients.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Total Improvement Score (TIS) at OLE Week 48 | 48 weeks | 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. |
Countries
Czechia, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| KZR-616 45 mg + Standard Therapy (Open-label) All patients received SC injections of KZR-616 QW at doses of 30 mg on day 1 and 45 mg thereafter for up to 96 weeks. Study drug administration ended when the last enrolled patient completed 48 weeks of dosing in the OLE. Patients had an end of study visit 12 weeks after the last dose of KZR-616.
KZR-616: zetomipzomib subcutaneous injection | 18 |
| Total | 18 |
Baseline characteristics
| Characteristic | KZR-616 45 mg + Standard Therapy (Open-label) |
|---|---|
| Age, Continuous | 52.7 years STANDARD_DEVIATION 14.3 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 5 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 13 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Sex: Female, Male Female | 13 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 18 |
| other Total, other adverse events | 17 / 18 |
| serious Total, serious adverse events | 1 / 18 |
Outcome results
Primary
Mean Total Improvement Score (TIS) at OLE Week 48
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.
Time frame: 48 weeks
Population: Nine (9) patients were enrolled in the OLE study at Week 48.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| KZR-616 45 mg + Standard Therapy (Open-label) | Mean Total Improvement Score (TIS) at OLE Week 48 | 36.4 score on a scale | Standard Deviation 23.2 |