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A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis (PRESIDIO)

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ClinicalTrials.gov Identifier: NCT04033926
Recruitment Status : Recruiting
First Posted : July 26, 2019
Last Update Posted : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
Kezar Life Sciences, Inc.

Study Description
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Brief Summary:

"This is a Phase 2 randomized, double-blind, placebo-controlled, crossover, multicenter study to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK) and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Polymyositis (PM) or Dermatomyositis (DM). Patients will be evaluated for eligibility during the Screening Period. Eligible patients will be randomized 1:1 to Arm A or Arm B of the study.

During the 32-week treatment period, patients will receive study drug subcutaneously (SC) once weekly with 2 treatment periods of 16 weeks each.

This study will be conducted on an outpatient basis.


Condition or disease Intervention/treatment Phase
Polymyositis Dermatomyositis Drug: KZR-616 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A 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
Actual Study Start Date : January 14, 2020
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Arm A
  • Treatment Period 1: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks
  • Treatment Period 2: Placebo SC weekly for 16 weeks
 Drug: KZR-616
Subcutaneous 30 mg weekly for 2 weeks, then 45 mg weekly for 14 weeks

Drug: Placebo
Subcutaneous injection for 16 weeks
Arm B
  • Treatment Period 1: Placebo SC weekly for 16 weeks
  • Treatment Period 2: KZR-616 30 mg SC weekly for 2 weeks, then 45 mg SC weekly for 14 weeks
 Drug: KZR-616
Subcutaneous 30 mg weekly for 2 weeks, then 45 mg weekly for 14 weeks

Drug: Placebo
Subcutaneous injection for 16 weeks


Outcome Measures
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Primary Outcome Measures :
  1. Primary Outcome Measure [ Time Frame: From start to end of KZR-616 treatment for both sequence arms combined. ]
    Mean change from start to end of KZR-616 treatment in the Total Improvement Score (TIS), which ranges from 0 to 100.


Secondary Outcome Measures :
  1. Proportion of patients with an increase ≥20 points on the TIS from start to end of KZR-616 treatment. [ Time Frame: 16 weeks ]
  2. Proportion of patients from start to end of KZR-616 treatment meeting International Myositis Assessment and Clinical Studies Group (IMACS) Definition of Improvement (DOI). [ Time Frame: 16 weeks ]
  3. Absolute change from start to end of KZR-616 treatment in the IMACS individual core set activity measures (CSAMs) [ Time Frame: 16 weeks ]
  4. Percent change from start to end of KZR-616 treatment in the IMACS individual CSAMs [ Time Frame: 16 weeks ]
  5. For patients with DM, the mean change from start to end of KZR-616 treatment in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) [ Time Frame: 16 weeks ]
  6. For patients with DM, the mean change from start to end of KZR-616 treatment in the Peak Pruritus Numeric Rating Scale. [ Time Frame: 16 weeks ]
    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.

  7. Safety and tolerability of KZR-616 in patients with PM or DM as assessed by monitoring incidence and severity of adverse events (AEs) [ Time Frame: 40 weeks ]
  8. Peak plasma concentration (Cmax) following KZR-616 injection [ Time Frame: Day 1 ]
  9. Time to peak plasma concentration (Tmax) following KZR-616 injection [ Time Frame: Day 1 ]
  10. Area under the plasma concentration versus time curve (AUC) following KZR-616 injection [ Time Frame: Day 1 ]
  11. Half life (T1/2) following KZR-616 injection [ Time Frame: Day 1 ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Adult patients at least 18 years of age
  2. Body Mass Index (BMI) of 18 to 40 kg/m2
  3. Diagnosis of probable or definite DM or PM

  4. Must confirm eligibility unless at least 1 of the following is present:

    1. Muscle biopsy with evidence of active myositis within the last 6 months prior to or at Screening
    2. Electromyography or magnetic resonance imaging with evidence of active myositis within the last 6 months prior to Screening
    3. A creatine kinase (CK) ≥4 × upper limit of normal (ULN).

  5. Must have demonstrable muscle weakness as measured by the Manual Muscle Testing-8 muscle Groups (MMT-8) with a score ≥80/150 but ≤136/150 units and any 2 of the following:

    1. Physician Global Assessment (MDGA) visual analog scale (VAS) ≥2 cm
    2. Patient Global Assessment of Disease Activity (PtGADA) VAS ≥2 cm
    3. At least one muscle enzyme laboratory measurement ≥1.3 × ULN
    4. Myositis Disease Activity Assessment Tool (MDAAT) Extramuscular Global Activity VAS ≥1 cm.
  6. Documented inadequate response OR have demonstrated documented toxicity or intolerance to prior standard of care therapies
  7. Has had age-appropriate cancer screening that is up to date and negative for evidence of malignancy as per local standard of care

Exclusion Criteria:

  1. Has significant muscle damage or has a muscle damage VAS score ≥5 cm on the MDI
  2. Any other form of myositis or myopathy other than PM or DM
  3. Any condition that precludes the ability to quantitate muscle strength
  4. Has severe interstitial lung disease or has a pulmonary damage VAS score ≥5 cm on the Myositis Damage Index (MDI)
  5. Presence of autoinflammatory disease
  6. Use of nonpermitted medications or treatments within the specified washout periods prior to screening
  7. Patient has had recent serious or ongoing infection, or risk for serious infection

  8. Any of the following laboratory values at Screening:

    1. Estimated glomerular filtration rate <45 mL/min
    2. Hemoglobin <10 g/dL
    3. White blood cell (WBC) count <3.0 × 109/L
    4. Absolute neutrophil count (ANC) <1.5 × 109/L (1500/mm3)
    5. Platelet count <100 × 109/L
    6. Serum AST or serum ALT >2.5 × ULN (unless considered consistent with muscle origin)
    7. Serum alkaline phosphatase >2.5 × ULN
    8. Total bilirubin >1.5 × ULN (3 × ULN for patients with documented Gilbert's syndrome)
    9. Thyroid stimulating hormone outside of the central laboratory normal range
    10. Immunoglobulin G (IgG) <500 mg/dL.
  9. Presence of New York Heart Association Class III or IV heart failure, or uncontrolled blood pressure, or prolonged QT interval
  10. Major surgery within 12 weeks before Screening or planned during the study period
  11. Clinical evidence of significant unstable or uncontrolled diseases
  12. Any active or suspected malignancy, including myeloproliferative or lymphoproliferative disorder, or history of documented malignancy within the last 5 years before Screening or within 3 years of diagnosis of myositis, except appropriately excised and cured cervical carcinoma in situ or basal or squamous cell carcinoma of the skin
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04033926


Contacts
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Contact: Clinical Operations (650)822-5600 clinicaltrials@kezarbio.com

Locations
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United States, California
KZR Research Site Recruiting
Irvine, California, United States, 92698
United States, Florida
KZR Research Site Not yet recruiting
Miami, Florida, United States, 33136
KZR-616 Research Site Not yet recruiting
Panama City, Florida, United States, 32405
United States, Kansas
KZR Research Site Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
KZR Research Site Not yet recruiting
Baltimore, Maryland, United States, 21224
United States, Michigan
KZR Research Site Recruiting
Ann Arbor, Michigan, United States, 48109
United States, New York
KZR Research Site Recruiting
Great Neck, New York, United States, 11021
United States, Pennsylvania
KZR-616 Research Site Recruiting
Duncansville, Pennsylvania, United States, 16635
KZR Research Site Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
KZR-616 Research Site Recruiting
Austin, Texas, United States, 78756
KZR Research Site Recruiting
Webster, Texas, United States, 77598
Sponsors and Collaborators
Kezar Life Sciences, Inc.
Investigators
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Study Director: Kezar Kezar Life Sciences, Inc.
More Information
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Responsible Party: Kezar Life Sciences, Inc.
ClinicalTrials.gov Identifier: NCT04033926    
Other Study ID Numbers: KZR-616-003
First Posted: July 26, 2019    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Kezar Life Sciences, Inc.:
Myositis
Idiopathic inflammatory myopathies
Polymyositis
 Dermatomyositis
Musculoskeletal Diseases
Muscular Diseases
Additional relevant MeSH terms:
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Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases