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Trial record 4 of 4 for:    KZR-616

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 : Active, not recruiting
First Posted : July 26, 2019
Last Update Posted : August 24, 2021
Sponsor:
Information provided by (Responsible Party):
Kezar Life Sciences, Inc.

Tracking Information
First Submitted Date  ICMJE July 23, 2019
First Posted Date  ICMJE July 26, 2019
Last Update Posted Date August 24, 2021
Actual Study Start Date  ICMJE January 14, 2020
Estimated Primary Completion Date February 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 24, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 26, 2019)
  • Proportion of patients with an increase ≥20 points on the TIS from start to end of KZR-616 treatment. [ Time Frame: 16 weeks ]
  • 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 ]
  • Absolute change from start to end of KZR-616 treatment in the IMACS individual core set activity measures (CSAMs) [ Time Frame: 16 weeks ]
  • Percent change from start to end of KZR-616 treatment in the IMACS individual CSAMs [ Time Frame: 16 weeks ]
  • 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 ]
  • 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.
  • 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 ]
  • Peak plasma concentration (Cmax) following KZR-616 injection [ Time Frame: Day 1 ]
  • Time to peak plasma concentration (Tmax) following KZR-616 injection [ Time Frame: Day 1 ]
  • Area under the plasma concentration versus time curve (AUC) following KZR-616 injection [ Time Frame: Day 1 ]
  • Half life (T1/2) following KZR-616 injection [ Time Frame: Day 1 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 24, 2019)
  • Proportion of patients with an increase ≥20 points on the TIS from start to end of KZR-616 treatment. [ Time Frame: 16 weeks ]
  • 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 ]
  • Absolute change from start to end of KZR-616 treatment in the IMACS individual CSAMs [ Time Frame: 16 weeks ]
  • Percent change from start to end of KZR-616 treatment in the IMACS individual CSAMs [ Time Frame: 16 weeks ]
  • 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 ]
  • 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.
  • 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: Baseline through 40 weeks ]
  • Treatment Period 1: Peak plasma concentration (Cmax) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 1 ]
  • Treatment Period 1: Time to peak plasma concentration (Tmax) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 1 ]
  • Treatment Period 1: Area under the plasma concentration versus time curve (AUC) for KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 1 ]
  • Treatment Period 1: Half-life (T1/2) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 1 ]
  • Treatment Period 2: Peak plasma concentration (Cmax) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 113 ]
  • Treatment Period 2: Time to peak plasma concentration (Tmax) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 113 ]
  • Treatment Period 2: Area under the plasma concentration versus time curve (AUC) for KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 113 ]
  • Treatment Period 2: Half-life (T1/2) of KZR-616 and KZR-59587 following KZR-616 injection [ Time Frame: Day 113 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis
Official Title  ICMJE 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
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Polymyositis
  • Dermatomyositis
Intervention  ICMJE
  • Drug: KZR-616
    Subcutaneous 30 mg weekly for 2 weeks, then 45 mg weekly for 14 weeks
  • Drug: Placebo
    Subcutaneous injection for 16 weeks
Study Arms  ICMJE
  • 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
    Interventions:
    • Drug: KZR-616
    • Drug: Placebo
  • 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
    Interventions:
    • Drug: KZR-616
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: July 24, 2019)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2022
Estimated Primary Completion Date February 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Czechia,   Germany,   Poland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04033926
Other Study ID Numbers  ICMJE KZR-616-003
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Kezar Life Sciences, Inc.
Study Sponsor  ICMJE Kezar Life Sciences, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Kezar Kezar Life Sciences, Inc.
PRS Account Kezar Life Sciences, Inc.
Verification Date August 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP