A Pilot Study of Etanercept in Dermatomyositis
Condition: Dermatomyositis
MeSH terms: Dermatomyositis
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of the study is to obtain preliminary data regarding the safety and tolerability
      of etanercept in DM. In addition, we will use the study to assess the variability,
      reliability, and responsiveness of the core set of outcome measures recommended by IMACS. The
      study will be performed under the aegis of the Muscle Study Group (MSG), consisting of
      experienced investigators with an avid interest in myopathies. The ultimate goal of this
      pilot study will be to obtain necessary, prerequisite information important in designing
      future therapeutic trials of etanercept and other agents in patients with DM. The specific
      aims of the study are:

      Aim 1: To preliminarily assess the safety and tolerability of etanercept in patients with DM.

      Aim 2: To assess the safety and tolerability of prednisone in the dosing schedule we propose
      to use.

      Aim 3: To evaluate outcome measures recommended by IMACS and assess their variability,
      reliability, and responsiveness in order to facilitate the design of future therapeutic
      trials in the inflammatory myopathies.
    

Detailed description: 
      Dermatomyositis (DM) is one of the major subtypes of idiopathic inflammatory myopathy.
      Prednisone is the initial treatment of choice in most patients with DM. However, because of
      the high rate of patients with disabling weakness despite treatment with prednisone, the
      long-term side effects of prednisone, and the many side effects associated with other
      second-line immunosuppressive agents (e.g., methotrexate, azathioprine), better treatment
      options are needed. There is evidence that tumor necrosis factor-a (TNF-a) plays a role in
      the pathogenesis of DM. Thus, etanercept, which blocks TNF-a, is a logical drug to assess in
      DM. Etanercept has been associated with a number of side effects including an increased risk
      of infection, inducing other autoimmune diseases, and perhaps cancer. These risks may be
      further enhanced in DM in which the frequency of other autoimmune disorders (e.g., connective
      tissue disease) and malignancy are already increased.

      The goal of this pilot study will be to assess the safety and tolerability of etanercept in
      DM.We will perform a double-blind, placebo-controlled pilot study of etanercept in 40
      patients with DM randomized in a 3:1 ratio to receive etanercept or placebo. All newly
      diagnosed and untreated patients will be started on a standard dose of prednisone and
      tapering schedule. Refractory patients who have been or are currently being treated with
      prednisone, IVIG, or methotrexate can also participate. Subjects will be followed for 1 year
      and we will assess various outcome variables recommended by the The International Myositis
      Assessment Clinical Study Group (IMACS). The primary aim of the study is to preliminarily
      assess the safety and tolerability of etanercept in patients with DM. We hypothesize that
      etanercept will be safe and well tolerated in this population. The second aim is to assess
      the safety and tolerability of prednisone in the dosing schedule we propose to use. We
      hypothesize that most patients will be able to tolerate the reduction of the prednisone
      dosage but most will not be able to be completely weaned off the medication. We believe we
      will find a relationship between prednisone dosage and its related side effects. The third
      aim of the study is to assess the variability, reliability, and responsiveness of the outcome
      measures recommended by IMACS using this pilot study of etanercept as the vehicle. The
      information gained from this study is necessary in order to design larger therapeutic trials
      of etanercept and other drugs in dermatomyositis.
    

Intervention name(s): Etanercept; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Etanercept 50 mg will be injected subcutaneously once per week for 52 weeks; Placebo, contained in 50mg syringes, will be injected subcutaneously once per week for 52 weeks.
Other intervention name(s): Saline
Intervention MeSH terms: Etanercept
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
Keywords: dermatomyositis; etanercept; tumor necrosis factor alpha