Clinical Trial: Study of Tofacitinib in Refractory Dermatomyositis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Study of Tofacitinib in Refractory Dermatomyositis (STIR): Proof of Concept, Open-Label Study of 10 Patients

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Johns Hopkins University

Current Primary Outcome: Number of participants who achieve International Myositis Assessment and Clinical Studies (IMACS) Definition of Improvement (DOI) [ Time Frame: Up to 12 weeks ]

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)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome: Same as current

Information By: Johns Hopkins University

Dates:
Date Received: December 20, 2016
Date Started: January 2017
Date Completion: December 2018
Last Updated: January 25, 2017
Last Verified: January 2017