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

• Register: ClinicalTrials.gov
• Last refreshed on: 8 January 2024
• Main ID: NCT04033926
• Date of registration: 23/07/2019
• Prospective Registration: Yes
• Primary sponsor: Kezar Life Sciences, Inc.
• Public title: A Phase 2 Study of KZR-616 to Evaluate Safety and Efficacy in Patients With Active Polymyositis or Dermatomyositis PRESIDIO
• Scientific 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
• Date of first enrolment: January 14, 2020
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04033926
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Czechia, Germany, Poland, United States

Contacts

• Name: Kezar
• Affiliation: Kezar Life Sciences, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Adult patients at least 18 years of age

2. Body Mass Index (BMI) of 18 to 40 kg/m^2

3. Diagnosis of probable or definite DM or PM by the 2017 European League Against
Rheumatism (EULAR)/American College of Rheumatology (ACR) Classification Criteria

4. Must have their data reviewed by an adjudication committee to 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 × 10^9/L

4. Absolute neutrophil count (ANC) <1.5 × 10^9/L (1500/mm^3)

5. Platelet count <100 × 10^9/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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Polymyositis

Dermatomyositis

Intervention(s)

Drug: Placebo

Drug: KZR-616

Primary Outcome(s)

Mean Change in the Total Improvement Score (TIS) From Start to End of Zetomipzomib (KZR-616) Treatment Period [Time Frame: 16 weeks in each Treatment Period (32 weeks total)]

Secondary ID(s)

KZR-616-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/01/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04033926