The purpose of this study is to obtain preliminary data regarding the safety and efficacy of Janus kinase (JAK) inhibitor, tofacitinib, in adults with active, treatment-refractory dermatomyositis.
Dermatomyositis (DM) is a rare, progressively debilitating disorder that affects the muscle (causing weakness) and skin (causing a rash) in most affected patients. DM can also involve multiple body systems including the lungs, joints, gut and heart. Therapy for DM involves administering corticosteroids, typically with an immunosuppressive agent, but treatment options for refractory DM are very limited. This research is being done to determine the safety and effectiveness of a Janus kinase (JAK) inhibitor called Tofacitinib in adults with active, treatment-refractory dermatomyositis. The investigators will also look for specific biomolecular changes in blood, skin, and muscle when it is exposed to Tofacitinib. Tofacitinib is approved by the Food and Drug Administration (FDA) for the treatment of rheumatoid arthritis. The study aim is to conduct a 12-week, open-label, pilot study with up to 10 patients who have refractory DM to assess whether a JAK inhibitor effectively and safely reduces the symptoms of DM in both the skin and muscle. This study consists of up to 9 study visits over 6 months. There is an optional treatment extension period of 4 weeks. All subjects will undergo follow-up assessments 4 weeks after stopping treatment. Results from this study will contribute to the design of future trials that will further define the role of JAK inhibitors in the treatment of patients with dermatomyositis.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Tofacitinib comes as an extended-release (XR) (long-acting) tablet to take by mouth. The extended-release tablet is usually taken with or without food once daily.
Johns Hopkins University
Baltimore, Maryland, United States
Number of participants who achieve International Myositis Assessment and Clinical Studies (IMACS) Definition of Improvement (DOI)
IMACS DOI is 3 of any of the 6 core set measures (CSM) improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (worsening measure cannot include the manual muscle testing)
Time frame: Up to 12 weeks
Change from baseline in CDASI activity score
Time frame: Up to 16 weeks
Safety and tolerability of tofacitinib as assessed by frequency of adverse events reported and observed
Time frame: Up to 16 weeks
Safety and tolerability of tofacitinib as assessed by incidence of adverse events reported and observed
Time frame: Up to 16 weeks
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