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

Study Purpose

"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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04033926
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Kezar Life Sciences, Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Kezar
Principal Investigator Affiliation Kezar Life Sciences, Inc.
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Czechia, Germany, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Polymyositis, Dermatomyositis
Arms & Interventions

Arms

Other: 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

Other: 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

Subcutaneous 30 mg weekly for 2 weeks, then 45 mg weekly for 14 weeks

Drug: - Placebo

Subcutaneous injection for 16 weeks

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

KZR Research Site, Irvine, California

Status

Recruiting

Address

KZR Research Site

Irvine, California, 92698

KZR Research Site, Miami, Florida

Status

Recruiting

Address

KZR Research Site

Miami, Florida, 33136

KZR Research Site, Kansas City, Kansas

Status

Recruiting

Address

KZR Research Site

Kansas City, Kansas, 66160

KZR Research Site, New Orleans, Louisiana

Status

Not yet recruiting

Address

KZR Research Site

New Orleans, Louisiana, 70115

KZR Research Site, Baltimore, Maryland

Status

Not yet recruiting

Address

KZR Research Site

Baltimore, Maryland, 21224

KZR Research Site, Ann Arbor, Michigan

Status

Recruiting

Address

KZR Research Site

Ann Arbor, Michigan, 48109

KZR Research Site, Great Neck, New York

Status

Recruiting

Address

KZR Research Site

Great Neck, New York, 11021

KZR Research Site, Duncansville, Pennsylvania

Status

Recruiting

Address

KZR Research Site

Duncansville, Pennsylvania, 16635

KZR Research Site, Pittsburgh, Pennsylvania

Status

Not yet recruiting

Address

KZR Research Site

Pittsburgh, Pennsylvania, 15213

KZR Research Site, Austin, Texas

Status

Recruiting

Address

KZR Research Site

Austin, Texas, 78756

KZR Research Site, Webster, Texas

Status

Recruiting

Address

KZR Research Site

Webster, Texas, 77598

International Sites

KZR Research Site, Prague, Czechia

Status

Not yet recruiting

Address

KZR Research Site

Prague, ,

KZR Research Site, Göttingen, Germany

Status

Not yet recruiting

Address

KZR Research Site

Göttingen, ,