Clinical trial NCT02821689: Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
Official title: Randomized Controlled Trial of Pirfenidone in Patients With Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Dermatopolymyositis
Interstitial Lung Disease
Keywords: Dermatopolymyositis
Interstitial lung disease
Pirfenidone
Lead sponsor: RenJi Hospital
Sponsor class: OTHER
First posted: 2016-07-01
Enrollment: 57
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: changes of 12-month survival from the onset of ILD
timeFrame: 12 months
Secondary outcomes:
measure: changes of high-resolution computed tomography (HRCT) scores from baseline at each visit
timeFrame: one year
measure: changes of pulmonary function test from baseline at each visit
timeFrame: one year
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Department of Rheumatology, Ren Ji Hospital South Campus, School of Medicine, Shanghai JiaoTong University
state: Shanghai
Study officials:
affiliation: RenJi Hospital
name: Shuang Ye, MD
role: PRINCIPAL_INVESTIGATOR
References: