Clinical Trial: A Pilot Study of Etanercept in Dermatomyositis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Pilot Study of Etanercept in Dermatomyositis

Brief 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 Summary:

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 do
Sponsor: Brigham and Women's Hospital

Current Primary Outcome:

  • Occurrence of at Least One Adverse Event [ Time Frame: at each visit during the 12 month study ]

    Adverse events (AEs) were assessed using the Common Terminology Criteria for Adverse Events version 3.0 (CTCAE). The grade of "mild", "moderate" or "severe" matches with the descriptions from the CTCAE dictionary.

    In general, a "Mild" AE is asymptomatic; clinical or diagnostic observations only; intervention not indicated.

    A "Moderate" AE is minimal, local or noninvasive intervention indicated; limiting activities of daily living.

    A "Severe" AE is medically significant but not immediately life-threatening; hospitalization or prolonged hospitalization indicated; disabling;

  • Tolerability [ Time Frame: At any point between Baseline (week 0) and the end of the study (Week 52) ]
    The reported tolerability measure was defined as the number of participants that completed the entire 52 week study on their originally assigned treatment.
  • Average Change in Oral Temperature From Baseline to Week 52 [ Time Frame: At Baseline (Week 0) and Week 52 ]

    The subject's oral temperature was measured in degrees Celsius. The average change was determined by subtracting the Baseline Visit (Week 0) results from the Week 52 results (Week 52- Baseline (Week 0)).

    This measure was also collected as part of th

    Original Primary Outcome: Safety and tolerability of etanercept

    Current Secondary Outcome:

    • Average Prednisone Dosage After Week 24 [ Time Frame: from week 24 to 52 ]
      We calculated the average dosage of prednisone from the week 24 visit until the end of the study (week 52).
    • Average Daily Dose of Prednisone From Baseline to Week 52 [ Time Frame: Baseline through Week 52 ]
      The average daily dose of prednisone from baseline to week 52 was calculated by treatment group.


    Original Secondary Outcome: Efficacy of etanercept

    Information By: Brigham and Women's Hospital

    Dates:
    Date Received: June 2, 2005
    Date Started: March 2006
    Date Completion:
    Last Updated: May 23, 2011
    Last Verified: May 2011