A study to evaluate the safety and efficacy of KZR-616 for the tretament of patients with active inflammations of the muscle and the skin (rheumatic disease)

Phase 1

• Conditions: Autoimmune Disorders Polymyositis and Dermatomyositis; MedDRA version: 20.0Level: HLGTClassification code 10003816Term: Autoimmune disordersSystem Organ Class: 10021428 - Immune system disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2019-002605-22-DE
• Lead Sponsor: Kezar Life Sciences, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-12-16
• Last Update Posted: 23 May 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

1. 18 years of age at the time of signing informed consent at Screening

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

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:
a. Muscle biopsy with evidence of active myositis within the last 6 months prior to or at Screening
b. Electromyography or magnetic resonance imaging (MRI) with evidence of active myositis within the last 6 months prior to Screening
c. A CK =4 × upper limit of normal (ULN).

5. Must have demonstrable muscle weakness as measured by the MMT-8 with a score =80/150 but =136/150 units and any 2 of the following:
a. MDGA visual analog scale (VAS) =2 cm
b. PtGADA VAS =2 cm
c. At least one muscle enzyme laboratory measurement (ie, CK, aldolase, LDH) =1.3 × ULN
d. MDAAT Extramuscular Global Activity VAS =1 cm.

6. Documented inadequate response to a 12-week trial of corticosteroids or at least 1 immunosuppressant (eg, methotrexate [MTX], mycophenolate mofetil [MMF], mycophenolate sodium [MPS], azathioprine [AZA], leflunomide [LEF], tacrolimus, cyclosporine [CyA]) OR have demonstrated significant
documented toxicity or intolerance to such 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

8. Female patients of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline and must agree to employ adequate birth control measures for the duration of the study.

9. Male patients with a partner of childbearing potential must be either congenitally sterile or surgically sterile (by vasectomy) or willing to use a condom in addition to having their female partner use another form of contraception (such as an intrauterine device, barrier method with spermicide, or hormonal contraceptive [eg, implant, injectable, patch, or oral]) from Screening until 12 weeks after the last dose of study drug, unless their partners are infertile or surgically sterile

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 6

Exclusion Criteria

1. Has significant muscle damage (eg, severe muscle atrophy, end-stage disease, postmyopathic DM) per Investigator opinion 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 (eg, metabolic or drug-induced myopathy, drug-induced myositis, juvenile PM or DM, inclusion body myositis, cancer-associated myositis [myositis diagnosed within 3 years, either before or after, of a diagnosis of any malignancy except for squamous or basal cell carcinoma of the skin or cervical carcinoma in situ], myositis in
overlap with another connective disease [eg, systemic lupus erythematosus{SLE}, systemic sclerosis,
rheumatoid arthritis {RA}], or muscular dystrophy); patients with secondary Sjogren’s syndrome, necrotizing myopathy, or antisynthetase syndrome are permitted to participate in this study

3. Any condition (eg, severe arthritis with limited range of motion or severe calcinosis) that, in the Investigator’s opinion, precludes the ability to quantitate muscle strength

4. Has severe interstitial lung disease per Investigator opinion or has a pulmonary damage VAS score =5 cm on the MDI

5. Presence of autoinflammatory disease (eg, psoriatic arthritis, axial spondyloarthropathy, inflammatory bowel disease)

Nota Bene: Exclusion Criteria one to five are listed. Exclusion criteria six to twenty are listed in the protocol (28May2021) from page 8-11.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified