Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis

Overview

Interstitial lung disease (ILD) is a common complication of dermatomyositis (DM) with prevalence up to 65%, and is considered to be one of the determining factors of prognosis. Clinical amyopathic dermatomyositis (CADM), which is a special phenotype of DM, with characteristic cutaneous manifestations but no or only subclinical myopathy. Many studies, mainly from Asia, including ours, have demonstrated that these patients with CADM tend to develop a rapidly progressive ILD (RPILD) and have a poor response to conventional therapy, such as high-dose corticosteroids and immunosuppressants, leading to lethal outcome with a 6-month survival rate of less than 50%.

Pirfenidone, a new oral antifibrotic agent, has been approved for the treatment of idiopathic pulmonary fibrosis (IPF). Randomized controlled trials of pirfenidone in patients with IPF suggested that it could ameliorate pulmonary function decline and improve the progression-free survival. Its utility in connective tissue disease (CTD) related ILD has been implicated, but no evidence has yet demonstrated its efficacy. Therefore, the investigators conduct this study to evaluate the possible therapeutic effects of pirfenidone on RPILD associated with CADM.

Full Title of Study: “Randomized Controlled Trial of Pirfenidone in Patients With Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2017

Interventions

  • Drug: Pirfenidone
    • Pirfenidone was administered in three divided doses (200mg tid), and increased to the manufacturer’s instructed target dose (600mg tid) over a 2-week period. The maximum dose was maintained throughout the study in patients who tolerated it.

Arms, Groups and Cohorts

  • Experimental: pirfenidone
    • Eligible participants for clinical trial were randomized in a 2:1 ratio to pirfenidone/blank add-on. Pirfenidone was administered in three divided doses (200mg tid), and increased to the manufacturer’s instructed target dose (600mg tid) over a 2-week period. Investigators were allowed to adjust the dose according to the participants’ tolerance.
  • No Intervention: Blank
    • Eligible participants for clinical trial were randomized in a 2:1 ratio to pirfenidone/blank add-on.

Clinical Trial Outcome Measures

Primary Measures

  • changes of 12-month survival from the onset of ILD
    • Time Frame: 12 months
    • the effect of pirfenidone on improving the survival rate

Secondary Measures

  • changes of high-resolution computed tomography (HRCT) scores from baseline at each visit
    • Time Frame: one year
    • the influence of pirfenidone on pulmonary interstitial changes
  • changes of pulmonary function test from baseline at each visit
    • Time Frame: one year
    • the influence of pirfenidone on pulmonary function changes

Participating in This Clinical Trial

Inclusion Criteria

  • Willingness of the subject to participate in the study, proven by signing the informed consent;
  • All participants fulfilled the provisional diagnosis of CADM according to the modified Sontheimer's criteria.
  • The course of ILD is longer than 3 months, but shorter than 6 months, presenting as increase in level of dyspnea, and worsening of fibrosis on pulmonary HRCT with >10% increase of HRCT score, and/or decrease in %FVC by >10% absolute value.

Exclusion Criteria

  • Participants who are unwilling to sign the inform consent;
  • The course of participants ever treated with biologics including basiliximab, or malignancy-associated CADM or overlapped with other CTD, or with alanine transaminase more than 2 times the upper normal limits;
  • Pregnancy or lactation.

Gender Eligibility: All

Minimum Age: 16 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • RenJi Hospital
  • Provider of Information About this Clinical Study
    • Principal Investigator: Shuang Ye, Executive Director, Dept. Rheumatology, Renji Hospital South Campus – RenJi Hospital
  • Overall Official(s)
    • Shuang Ye, MD, Principal Investigator, RenJi Hospital
  • Overall Contact(s)
    • Shuang Ye, MD, +8613817615871, ye_shuang2000@163.com

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