Pirfenidone as Treatment of Skin Fibrosis in Systemic Sclerosis

Phase 2

• Conditions: Systemic Sclerosis
• Interventions: Drug: Pirfenidone; Drug: Steroids; Drug: Placebo oral capsule

• Registration Number: NCT03068234
• Lead Sponsor: RenJi Hospital

Brief Summary

This study is to evaluate the efficacy and safety of an antifibrotic agent, pirfenidone as treatment of systemic sclerosis. The primary outcome of this study is improvement of skin fibrosis.

Detailed Description

All patients enrolled should suffer from moderate to severe skin fibrosis at the screening stage.

The study period is 52 week. The study contains two stages, a 24-week blind stage following with open stage for another 24-week period of time. Subjects will receive pirfenidone or placebo randomized in the first 24-week and will all receive pirfenidone treatment in the second 24-week, with of combination of low dose steroids.

The secondary outcomes of this study include but not limit to safety, improvement of lung fibrosis and digital ulcer burden.

Study Timeline

• Status Verified: 2017-02
• Study First Submitted: 2017-02-16
• Study First Submitted QC: 2017-02-24
• Last Update Submitted: 2017-02-24
• Study First Posted: 2017-03-01
• Last Update Posted: 2017-03-01
• Study Start: 2017-05
• Primary Completion: 2019-04
• Study Completion: 2019-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 72

Inclusion Criteria

• Fulfill EULAR/ACR classification criteria (2013) for systemic sclerosis (SSc).
• Subjects must have mRSS≥10 at screening stage
• Subjects must have signs of fibrosis in their chest CT at screening stage
• If a subject has received treatments before, it should be stable at least 3 months until screening and with no significant efficacy (ΔmRSS≤-2)

Exclusion Criteria

• Subjects with other connective tissue diseases overlapping with SSc
• Subjects with skin atrophy as the main cutaneous manifestation
• Subjects with active digital ulcers or gangrene
• Active severe SSc-driven renal disease or heart dysfunction at screening
• Subjects with significant hematologic abnormalities
• Abnormal liver function test at screening (ALT, AST or total bilirubin over 2 fold of upper normal level
• Clinically significant active infection including ongoing and chronic infections
• History of human immunodeficiency virus (HIV)
• Confirmed Positive tests for hepatitis B or positive test for hepatitis C
• Active tuberculosis
• Live or attenuated vaccine within 4 weeks prior to screening
• Subjects with significant hematologic abnormalities

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pirfenidone group	Steroids	The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose steroids.
Control group	Placebo oral capsule	The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
Control group	Pirfenidone	The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
Control group	Steroids	The subjects will receive placebo within first 24-week, and pirfenidone 200mg three times a day for the second 24-week, with low dose steroids.
Pirfenidone group	Pirfenidone	The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose steroids.

Primary Outcome Measures

Name	Time	Method
Modified Rodnan Skin Score (mRSS）	Week 52	A semi-quantitative score for skin fibrosis of every subjects

Secondary Outcome Measures

Name	Time	Method
Assessment of chest CT	Week 24	Semi-quantitative scale for fibrotic changes in chest CT images from an individual patient
Hand function assessment	Week 24	Hand function will be measured by Cochin Hand Function Scale
Proportion of subjects with increased mRSS	Week 24	Proportion of subjects with ∆mRSS≥5
Modified Rodnan Skin Score	Week 24	A semi-quantitative score for skin fibrosis of every subjects
Forced vital capacity	Week 24	A marker for lung function that may decrease with pulmonary fibrosis
Diffusing capacity	Week 24	Another marker for lung function that may decrease with pulmonary fibrosis
6 minute walk test	Week 24	A simple, well-accepted and quantitative clinical exam to test heart and lung function
Quality of life	Week 24	Assessed by the health assessment questionnaire disability index
Safety and Tolerability	Week 24	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Trial Locations

Locations (1)

RenJi Hospital; 🇨🇳 Shanghai, Shanghai, China