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Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.

Primary Purpose

Polymyositis, Dermatomyositis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
KZR-616
Sponsored by
Kezar Life Sciences, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polymyositis focused on measuring Myositis, Idiopathic inflammatory myopathies, Polymyositis, Dermatomyositis, Musculoskeletal Diseases, Muscular Diseases

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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:

  • Have clinical evidence of significant unstable or uncontrolled diseases other than the disease under study, that in the opinion of the Investigator or Sponsor/designee, could confound the results of the study, put the patient at undue risk, or interfere with protocol adherence.
  • 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.

Sites / Locations

  • KZR Research Site
  • KZR Research Site
  • KZR Research Site
  • KZR Research Site
  • KZR Research Site
  • KZR Research Site
  • KZR Research Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

KZR-616 45 mg + standard therapy (open-label)

Arm Description

All patients will receive 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 will end for all patients in Study KZR-616-003E when the last patient enrolled has completed 48 weeks of dosing.

Outcomes

Primary Outcome Measures

Total Improvement Score (TIS)
Mean change in TIS over time for all patients, for patients with DM only, for patients with PM only, and for patients with a myositis-associated antibody or myositis-specific antibody.

Secondary Outcome Measures

Total Improvement Score (TIS) response
Proportion of patients by TIS response (minimal response = TIS ≥ 20, moderate response = TIS ≥ 40, major response = TIS ≥ 60)
International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI)
Proportion of patients meeting IMACS definition of improvement (DOI) over time for patients with baseline core set measures.
International Myositis Assessment and Clinical Studies Group (IMACS) individual Core Set Activity Measures (CSAMs)
Mean change and mean percentage change over time in the IMACS individual CSAMs and CSDMs, stratified by all patients and patients with myositis-associated or myositis-specific antibody.
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
Mean change over time in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) for all patients with DM, and for patients with DM who have active skin manifestations at baseline of Study KZR-616-003E.
Corticosteroid and non-corticosteroid immunosuppressants
Change in proportion and dose of corticosteroid and non-corticosteroid immunosuppressants during Study KZR-616-003E for all patients, and for patients taking corticosteroids or non-corticosteroid immunosuppressants at baseline of Study KZR-616-003E.

Full Information

First Posted
November 2, 2020
Last Updated
July 14, 2023
Sponsor
Kezar Life Sciences, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04628936
Brief Title
Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
November 4, 2020 (Actual)
Primary Completion Date
March 10, 2023 (Actual)
Study Completion Date
June 12, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kezar Life Sciences, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
This is an open-label study to evaluate the long-term efficacy and safety of KZR-616 in patients with active PM or 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 is 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, up to and including the Week 32 Visit, prior to the first dose of open-label KZR-616. Patients will be 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 will receive 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 will end for all patients in Study KZR-616-003E when the last patient enrolled has completed 48 weeks of dose. Patients will have 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polymyositis, Dermatomyositis
Keywords
Myositis, Idiopathic inflammatory myopathies, Polymyositis, Dermatomyositis, Musculoskeletal Diseases, Muscular Diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
All patients will receive 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 will end for all patients when the last patient enrolled has completed 48 weeks of dosing.
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
KZR-616 45 mg + standard therapy (open-label)
Arm Type
Experimental
Arm Description
All patients will receive 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 will end for all patients in Study KZR-616-003E when the last patient enrolled has completed 48 weeks of dosing.
Intervention Type
Drug
Intervention Name(s)
KZR-616
Other Intervention Name(s)
KZR-616 Lyophile
Intervention Description
KZR-616 subcutaneous injection
Primary Outcome Measure Information:
Title
Total Improvement Score (TIS)
Description
Mean change in TIS over time for all patients, for patients with DM only, for patients with PM only, and for patients with a myositis-associated antibody or myositis-specific antibody.
Time Frame
80 weeks
Secondary Outcome Measure Information:
Title
Total Improvement Score (TIS) response
Description
Proportion of patients by TIS response (minimal response = TIS ≥ 20, moderate response = TIS ≥ 40, major response = TIS ≥ 60)
Time Frame
80 weeks
Title
International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI)
Description
Proportion of patients meeting IMACS definition of improvement (DOI) over time for patients with baseline core set measures.
Time Frame
80 weeks
Title
International Myositis Assessment and Clinical Studies Group (IMACS) individual Core Set Activity Measures (CSAMs)
Description
Mean change and mean percentage change over time in the IMACS individual CSAMs and CSDMs, stratified by all patients and patients with myositis-associated or myositis-specific antibody.
Time Frame
80 weeks
Title
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)
Description
Mean change over time in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) for all patients with DM, and for patients with DM who have active skin manifestations at baseline of Study KZR-616-003E.
Time Frame
60 weeks
Title
Corticosteroid and non-corticosteroid immunosuppressants
Description
Change in proportion and dose of corticosteroid and non-corticosteroid immunosuppressants during Study KZR-616-003E for all patients, and for patients taking corticosteroids or non-corticosteroid immunosuppressants at baseline of Study KZR-616-003E.
Time Frame
60 weeks
Other Pre-specified Outcome Measures:
Title
Peak Pruritus Numeric Rating Scale Pruritis Numerical Rating Scale [NRS]
Description
For DM patients, the Peak Pruritus Numeric Rating Scale ranges from a score of 0 to 10, with 0 representing no itch and 10 representing the worst itch imaginable during the worst moment within a 24-hour recall period.
Time Frame
60 weeks
Title
Incidence, nature, and severity of treatment-emergent adverse events (TEAEs) with the causal relationship assessed by the investigator graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE).
Description
Safety
Time Frame
60 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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: Have clinical evidence of significant unstable or uncontrolled diseases other than the disease under study, that in the opinion of the Investigator or Sponsor/designee, could confound the results of the study, put the patient at undue risk, or interfere with protocol adherence. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kezar Study Director
Organizational Affiliation
Kezar Life Sciences, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
KZR Research Site
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
KZR Research Site
City
Irvine
State/Province
California
ZIP/Postal Code
92698
Country
United States
Facility Name
KZR Research Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
KZR Research Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21224
Country
United States
Facility Name
KZR Research Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
KZR Research Site
City
Duncansville
State/Province
Pennsylvania
ZIP/Postal Code
16635
Country
United States
Facility Name
KZR Research Site
City
Austin
State/Province
Texas
ZIP/Postal Code
78756
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://www.kezarlifesciences.com/
Description
Kezar Life Sciences, Inc. corporate website

Learn more about this trial

Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.

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