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Pirfenidone as Treatment of Skin Fibrosis in Systemic Sclerosis

Primary Purpose

Systemic Sclerosis

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Pirfenidone
Placebo oral capsule
Steroids
Sponsored by
RenJi Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Systemic Sclerosis

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • RenJi Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Pirfenidone group

Control group

Arm Description

The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose 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.

Outcomes

Primary Outcome Measures

Modified Rodnan Skin Score (mRSS）
A semi-quantitative score for skin fibrosis of every subjects

Secondary Outcome Measures

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

Full Information

First Posted
February 16, 2017
Last Updated
February 24, 2017
Sponsor
RenJi Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT03068234
Brief Title
Pirfenidone as Treatment of Skin Fibrosis in Systemic Sclerosis
Official Title
A Randomized, Double-blinded, Placebo Controlled Study to Evaluate Clinical Efficacy and Safety of Pirfenidone for Skin Fibrosis in Systemic Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Unknown status
Study Start Date
May 2017 (Anticipated)
Primary Completion Date
April 2019 (Anticipated)
Study Completion Date
December 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
RenJi Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Sclerosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Pirfenidone group
Arm Type
Experimental
Arm Description
The subjects will receive pirfenidone from 200mg three times a day, oral administrated, with low dose steroids.
Arm Title
Control group
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Pirfenidone
Intervention Description
A chemical synthesized agent showing anti-fibrotic effects in idiopathic pulmonary fibrosis.
Intervention Type
Drug
Intervention Name(s)
Placebo oral capsule
Intervention Description
Placebo for pirfenidone capsule
Intervention Type
Drug
Intervention Name(s)
Steroids
Intervention Description
Low dose of glucocorticoids, a basic treatment for patients with systemic sclerosis
Primary Outcome Measure Information:
Title
Modified Rodnan Skin Score (mRSS）
Description
A semi-quantitative score for skin fibrosis of every subjects
Time Frame
Week 52
Secondary Outcome Measure Information:
Title
Modified Rodnan Skin Score
Description
A semi-quantitative score for skin fibrosis of every subjects
Time Frame
Week 24
Title
Assessment of chest CT
Description
Semi-quantitative scale for fibrotic changes in chest CT images from an individual patient
Time Frame
Week 52
Title
Assessment of chest CT
Description
Semi-quantitative scale for fibrotic changes in chest CT images from an individual patient
Time Frame
Week 24
Title
Forced vital capacity
Description
A marker for lung function that may decrease with pulmonary fibrosis
Time Frame
Week 52
Title
Forced vital capacity
Description
A marker for lung function that may decrease with pulmonary fibrosis
Time Frame
Week 24
Title
Diffusing capacity
Description
Another marker for lung function that may decrease with pulmonary fibrosis
Time Frame
Week 52
Title
Diffusing capacity
Description
Another marker for lung function that may decrease with pulmonary fibrosis
Time Frame
Week 24
Title
6 minute walk test
Description
A simple, well-accepted and quantitative clinical exam to test heart and lung function
Time Frame
Week 52
Title
6 minute walk test
Description
A simple, well-accepted and quantitative clinical exam to test heart and lung function
Time Frame
Week 24
Title
Hand function assessment
Description
Hand function will be measured by Cochin Hand Function Scale
Time Frame
Week 52
Title
Hand function assessment
Description
Hand function will be measured by Cochin Hand Function Scale
Time Frame
Week 24
Title
Proportion of subjects with increased mRSS
Description
Proportion of subjects with ∆mRSS≥5
Time Frame
Week 52
Title
Proportion of subjects with increased mRSS
Description
Proportion of subjects with ∆mRSS≥5
Time Frame
Week 24
Title
Quality of life
Description
Assessed by the health assessment questionnaire disability index
Time Frame
Week 52
Title
Quality of life
Description
Assessed by the health assessment questionnaire disability index
Time Frame
Week 24
Title
Safety and Tolerability
Description
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame
Week 52
Title
Safety and Tolerability
Description
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame
Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
RenJi Hospital
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200001
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Pirfenidone as Treatment of Skin Fibrosis in Systemic Sclerosis

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