Clinical trial NCT03068234: 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
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Systemic Sclerosis
Lead sponsor: RenJi Hospital
Sponsor class: OTHER
First posted: 2017-03-01
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Modified Rodnan Skin Score (mRSS）
timeFrame: Week 52
Secondary outcomes:
measure: Modified Rodnan Skin Score
timeFrame: Week 24
measure: Assessment of chest CT
timeFrame: Week 52
measure: Assessment of chest CT
timeFrame: Week 24
measure: Forced vital capacity
timeFrame: Week 52
measure: Forced vital capacity
timeFrame: Week 24
measure: Diffusing capacity
timeFrame: Week 52
measure: Diffusing capacity
timeFrame: Week 24
measure: 6 minute walk test
timeFrame: Week 52
measure: 6 minute walk test
timeFrame: Week 24
measure: Hand function assessment
timeFrame: Week 52
measure: Hand function assessment
timeFrame: Week 24
measure: Proportion of subjects with increased mRSS
timeFrame: Week 52
measure: Proportion of subjects with increased mRSS
timeFrame: Week 24
measure: Quality of life
timeFrame: Week 52
measure: Quality of life
timeFrame: Week 24
measure: Safety and Tolerability
timeFrame: Week 52
measure: Safety and Tolerability
timeFrame: Week 24
Other outcomes:
Locations:
city: Shanghai
country: China
facility: RenJi Hospital
state: Shanghai
Study officials:
References: