NCT02821689

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
57

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Jul 2016

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 21, 2016

Completed
10 days until next milestone

First Posted

Study publicly available on registry

July 1, 2016

Completed
Same day until next milestone

Study Start

First participant enrolled

July 1, 2016

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2017

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2018

Completed
Last Updated

July 1, 2016

Status Verified

June 1, 2016

Enrollment Period

11 months

First QC Date

June 21, 2016

Last Update Submit

June 30, 2016

Conditions

DermatopolymyositisInterstitial Lung Disease

Keywords

DermatopolymyositisInterstitial lung diseasePirfenidone

Outcome Measures

Primary Outcomes (1)

  • changes of 12-month survival from the onset of ILD

    the effect of pirfenidone on improving the survival rate

    12 months

Secondary Outcomes (2)

  • changes of high-resolution computed tomography (HRCT) scores from baseline at each visit

    one year

  • changes of pulmonary function test from baseline at each visit

    one year

Study Arms (2)

pirfenidone

EXPERIMENTAL

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

Blank

NO INTERVENTION

Eligible participants for clinical trial were randomized in a 2:1 ratio to pirfenidone/blank add-on.

Interventions

PirfenidoneDRUG

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.

pirfenidone

Eligibility Criteria

Age16 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Shanghai, Shanghai Municipality, 200001, China

Location

MeSH Terms

Conditions

DermatomyositisLung Diseases, Interstitial

Interventions

pirfenidone

Condition Hierarchy (Ancestors)

PolymyositisMyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin DiseasesLung DiseasesRespiratory Tract Diseases

Study Officials

  • Shuang Ye, MD

    RenJi Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Shuang Ye, MD

CONTACT

+8613817615871ye_shuang2000@163.com

Zhiwei Chen, MS

CONTACT

+8613621621736zwchen88@gmail.com

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Executive Director, Dept. Rheumatology, Renji Hospital South Campus

Study Record Dates

First Submitted

June 21, 2016

First Posted

July 1, 2016

Study Start

July 1, 2016

Primary Completion

June 1, 2017

Study Completion

June 1, 2018

Last Updated

July 1, 2016

Record last verified: 2016-06

Locations

CN(1)