Clinical Characteristics and Mechanism Research of Inhibitors of Janus Kinase in the Idiopathic Inflammatory Myopathies

Real-world Experience With Inhibitors of Janus Kinase for the Treatment of Idiopathic Inflammatory Myopathies -- a Prospective Observational Study

This study aims to explore the clinical characteristics and mechanism of inhibitors of janus kinase in the treatment of idiopathic inflammatory myopathies

Study Overview

• Status: Recruiting
• Conditions: Idiopathic Inflammatory Myopathies
• Intervention / Treatment: Drug: tofacitinib

Detailed Description

The investigators designed a single center, open-label, prospective study. Adults with active idiopathic inflammatory myopathies will be enrolled, meeting the Bohan & Peter Dermatomyositis/Polymyositis(DM/PM) or Rheumatology(ACR) & European allance of associations for rheumatology(EULAR)(2017) diagnostic criteria. Inhibitors of janus kinase including tofacitinib 5 mg once a day or twice a day and baricitinib 2mg once a day or 4mg once a day was administered for 6 months to explore its efficacy and safety, which could help to evaluate inhibitors of janus kinase's clinical characteristics and mechanism. Patients would be evaluated the improvement of clinical and laboratory indexes. Changes of symptoms, immune cell subsets and cytokines were monitored. Symptoms were evaluated by Visual Analogue Scale (VAS) of patient global and physician global, manual muscle testing(MMT-8), the Health Assessment Questionnaire(HAQ), Creatine kinase, Myositis Disease Activity Assessment Tool(MDAAT).

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Xiaoyan Xing; Phone Number: +86 18518732225; Email: 1119745376@qq.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China
      • Recruiting
      • Department of Rheumatology and Immunology, Peking University People's Hospital
      • Contact: Jing He; Phone Number: +8618611707347; Email: hejing1105@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Adults between 18 years and 75 years of age.
• Adults with active idiopathic inflammatory myopathies will be enrolled, meeting the Bohan & Peter DM/PM or American College of Rheumatology(ACR) & European allance of associations for rheumatology(EULAR)(2017) diagnostic criteria.
• Written informed consent should be obtained from each study subject.
• Concomitant immunosuppressive agents or glucocorticoids were allowed, but subjects should have been on these therapies at least 4 weeks and on a stable dose for ≥4 weeks(glucocorticoid doses are lower than <0.5mg/kg/d).
• Consent to use effective contraception during the study (women of childbearing age).

Exclusion Criteria:

Any subject meeting either of the following criteria should be excluded:

• Laboratory abnormality: Hb<8 g/dl or platelet<60*10^9/L, or combined with severe hepatic, renal and cardiac insufficiency;
• Myositis in overlap with another systemic autoimmune rheumatic disorder, cancer-associated myositis, inclusion body myositis or any other non-immune mediated myopathy.
• Biologics such as rituximab are not allowed in the 3-month before enrollment.
• Patients with malignancy within 3 years of screening.
• Patients with hypersensitivity to study drug.
• Active infections(including but not limited to hepatitis, HIV).
• Uncontrolled mental or emotional disorders.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: tofacitinib or baricitinib; Tofacitinib 5mg was taken orally once or twice a day and baricitinib 2mg or 4mg was taken orally once a day for 6 months.	Drug: tofacitinib; Tofacitinib 5mg was taken orally once or twice a day and baricitinib 2mg or 4mg was taken orally once a day for 6 months.; Other Names: Baricitinib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Immunological Responses	Changes of follicular helper T cells (TFH) before and after tofacitinib treatment are analysed to evaluate efficacy.	week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Myositis Disease Activity Assessment Tool(MDAAT)	Change of MDAAT(Myositis Disease Activity Assessment Tool) is measurement of effectiveness. MDAAT ranges from 0 to 10cm. The more severe the disease activity, the higher the score. We define response as an improvement of MDAAT at least 5%.	week 24

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2022
• Primary Completion (Anticipated): March 1, 2023
• Study Completion (Anticipated): June 1, 2023

Study Registration Dates

• First Submitted: May 16, 2022
• First Submitted That Met QC Criteria: May 27, 2022
• First Posted (Actual): June 2, 2022

Study Record Updates

• Last Update Posted (Actual): October 12, 2022
• Last Update Submitted That Met QC Criteria: October 9, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Muscular Diseases; Myositis; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; Tofacitinib
• Other Study ID Numbers: 20220420 JAKi-IIM

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No