Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis

Interstitial Lung Disease Clinical Trial

Official title:

Randomized Controlled Trial of Pirfenidone in Patients With Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02821689
• Other study ID #: RenJi-ADM
• Status: Not yet recruiting
• Phase: Phase 4
• First received: June 21, 2016
• Last updated: June 30, 2016
• Start date: July 2016
• Est. completion date: June 2018

Study information

• Verified date: June 2016
• Source: RenJi Hospital
• Contact: Shuang Ye, MD
• Phone: +8613817615871
• Email: ye_shuang2000[@]163.com
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 57
• Est. completion date: June 2018
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 16 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dermatomyositis
• Dermatopolymyositis
• Interstitial Lung Disease
• Lung Diseases
• Lung Diseases, Interstitial

Intervention

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.

Locations

Country	Name	City	State
China	Department of Rheumatology, Ren Ji Hospital South Campus, School of Medicine, Shanghai JiaoTong University	Shanghai	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
RenJi Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	changes of 12-month survival from the onset of ILD	the effect of pirfenidone on improving the survival rate	12 months	No
Secondary	changes of high-resolution computed tomography (HRCT) scores from baseline at each visit	the influence of pirfenidone on pulmonary interstitial changes	one year	No
Secondary	changes of pulmonary function test from baseline at each visit	the influence of pirfenidone on pulmonary function changes	one year	No