Pirfenidone in Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis

• ClinicalTrials.gov Identifier: NCT02821689
• Recruitment Status: Unknown
• First Posted: July 1, 2016
• Last Update Posted: July 1, 2016
• Sponsor: RenJi Hospital
• Information provided by (Responsible Party): Shuang Ye, RenJi Hospital

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Dermatopolymyositis; Interstitial Lung Disease	Drug: Pirfenidone	Phase 4

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 57 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Randomized Controlled Trial of Pirfenidone in Patients With Progressive Interstitial Lung Disease Associated With Clinically Amyopathic Dermatomyositis
• Study Start Date: July 2016
• Estimated Primary Completion Date: June 2017
• Estimated Study Completion Date: June 2018

Arms and Interventions

Arm	Intervention/treatment
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.	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.
No Intervention: Blank; Eligible participants for clinical trial were randomized in a 2:1 ratio to pirfenidone/blank add-on.

Outcome Measures

Primary Outcome Measures :

1. changes of 12-month survival from the onset of ILD [ Time Frame: 12 months ]
   the effect of pirfenidone on improving the survival rate

Secondary Outcome Measures :

1. 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
2. changes of pulmonary function test from baseline at each visit [ Time Frame: one year ]
   the influence of pirfenidone on pulmonary function changes

Eligibility Criteria

• Ages Eligible for Study: 16 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Contacts

Contact: Shuang Ye, MD; +8613817615871; ye_shuang2000@163.com

Contact: Zhiwei Chen, MS; +8613621621736; zwchen88@gmail.com

Locations

China, Shanghai

Department of Rheumatology, Ren Ji Hospital South Campus, School of Medicine, Shanghai JiaoTong University

Shanghai, Shanghai, China, 200001

Sponsors and Collaborators

RenJi Hospital

Investigators

• Principal Investigator: Shuang Ye, MD; RenJi Hospital

More Information

• Responsible Party: Shuang Ye, Executive Director, Dept. Rheumatology, Renji Hospital South Campus, RenJi Hospital
• ClinicalTrials.gov Identifier: NCT02821689
• Other Study ID Numbers: RenJi-ADM
• First Posted: July 1, 2016
• Last Update Posted: July 1, 2016
• Last Verified: June 2016

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

Keywords provided by Shuang Ye, RenJi Hospital:

Dermatopolymyositis; Interstitial lung disease; Pirfenidone

Additional relevant MeSH terms:

Dermatomyositis; Lung Diseases; Lung Diseases, Interstitial; Respiratory Tract Diseases; Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin Diseases; Pirfenidone; Analgesics; Sensory System Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Antineoplastic Agents