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

NCT04628936 | Completed Phase 2 Results FDA Drug

• 1 other identifier: KZR-616-003E
• Study Type: interventional
• Target: 18
• Locations: 2 countries
• Sites: 10

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.

Conditions

Polymyositis; Dermatomyositis

Keywords

Myositis; Idiopathic inflammatory myopathies; Polymyositis; Dermatomyositis; Musculoskeletal Diseases; Muscular Diseases

Outcome Measures

Primary Outcomes (1)

• 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.

  48 weeks

Study Arms (1)

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.

Drug: KZR-616

Interventions

KZR-616 DRUG

Subcutaneous 30 mg for 1 week, then 45 mg weekly

Also known as: KZR-616 Lyophile, zetomipzomib

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Kezar Life Sciences, Inc.: lead

Study Sites (10)

KZR Research Site

Beverly Hills, California, 90211, United States

Location

KZR Research Site

Orange, California, 92868, United States

Location

KZR Research Site

Miami, Florida, 33136, United States

Location

KZR Research Site

Kansas City, Kansas, 66160, United States

Location

KZR Research Site

Baltimore, Maryland, 21224, United States

Location

KZR Research Site

Ann Arbor, Michigan, 48109, United States

Location

KZR Research Site

Duncansville, Pennsylvania, 16635, United States

Location

KZR Research Site

Pittsburgh, Pennsylvania, 15213, United States

Location

KZR Research Site

Austin, Texas, 78756, United States

Location

KZR Research Site

Prague, Czechia

Location

Related Links

• Kezar Life Sciences, Inc. corporate website

MeSH Terms

Conditions

Polymyositis; Dermatomyositis; Myositis; Musculoskeletal Diseases; Muscular Diseases

Interventions

KZR-616

Condition Hierarchy (Ancestors)

Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Regulatory Affairs
• Organization: Kezar Life Sciences, Inc

clinicaltrials@kezarbio.com

650-822-5600

Study Officials

• Kezar Study Director

  Kezar Life Sciences, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 2, 2020
• First Posted: November 16, 2020
• Study Start: November 4, 2020
• Primary Completion: March 10, 2023
• Study Completion: June 12, 2023
• Last Updated: November 19, 2025
• Results First Posted: June 5, 2024

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1); US (9)