PolymyositisPhase 2NCT04628936
Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.
A Phase 2 study testing KZR-616 in people with Polymyositis and Dermatomyositis, run by Kezar Life Sciences, Inc. at 10 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polymyositis, Dermatomyositis
- What is being tested?
- KZR-616
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 4 Nov 2020to 12 Jun 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States9 sites
KZR Research Site
Ann Arbor, Michigan, 48109
KZR Research Site
Austin, Texas, 78756
KZR Research Site
Baltimore, Maryland, 21224
KZR Research Site
Beverly Hills, California, 90211
KZR Research Site
Duncansville, Pennsylvania, 16635
KZR Research Site
Kansas City, Kansas, 66160
KZR Research Site
Miami, Florida, 33136
KZR Research Site
Orange, California, 92868
KZR Research Site
Pittsburgh, Pennsylvania, 15213
About this study
Official title: An Open-label Extension to the Phase 2 Randomized, Double-blind, Placebo-controlled, Crossover Multicenter Study to Evaluate the Safety and Efficacy of KZR-616 in the Treatment of Patients With Active Polymyositis or Dermatomyositis
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.
Read the 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.
- Conditions
- Polymyositis, Dermatomyositis
- Being tested
- KZR-616
- Keywords
- Myositis, Idiopathic inflammatory myopathies, Polymyositis, Dermatomyositis, Musculoskeletal Diseases, Muscular Diseases
- Medical subject headings
- Polymyositis, Dermatomyositis, Myositis, Musculoskeletal Diseases, Muscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KZR-616Drug
Also called KZR-616 Lyophile, zetomipzomib
Subcutaneous 30 mg for 1 week, then 45 mg weekly
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: KZR-616 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2020
- Study start4 Nov 2020
- Primary completion10 Mar 2023
- Study completion12 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for PolymyositisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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.
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kezar Life Sciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kezar Study Director, Study director, Kezar Life Sciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04628936
- Sponsor's protocol ID
- KZR-616-003E
- Status last verified
- Nov 2025
- First submitted
- 2 Nov 2020
- Last updated
- 19 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension to the Phase 2 Randomized, Double-blind, Placebo-controlled, Crossover Multicenter Study to Evaluate the Safety and Efficacy of KZR-616 in the Treatment of Patients With Active Polymyositis or Dermatomyositis
- ClinicalTrials.gov ID
- NCT04628936
- Acronym / sponsor ID
- KZR-616-003E
- Conditions
- Polymyositis, Dermatomyositis
- Keywords
- Myositis, Idiopathic inflammatory myopathies, Polymyositis, Dermatomyositis, Musculoskeletal Diseases, Muscular Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Kezar Life Sciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Nov 2020 (actual)
- Primary completion
- 10 Mar 2023 (actual)
- Study completion
- 12 Jun 2023 (actual)
- First posted
- 16 Nov 2020 (actual)
- Results
- Posted 5 Jun 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| KZR-616 45 mg + standard therapy (open-label) | Experimental | Drug: KZR-616All 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KZR-616Also: KZR-616 Lyophile, zetomipzomibSubcutaneous 30 mg for 1 week, then 45 mg weekly | Drug | KZR-616 45 mg + standard therapy (open-label) |
Primary outcome
Mean Total Improvement Score (TIS) at OLE Week 48
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Kezar Life Sciences, Inc. corporate websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kezar Study Director study director
Kezar Life Sciences, Inc.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KZR-616 in people with Polymyositis and Dermatomyositis, run by Kezar Life Sciences, Inc. at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Total Improvement Score (TIS) at OLE Week 48 (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Nov 2025.